Vertex Pharmaceuticals Incorporated

United States of America

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[Owner] Vertex Pharmaceuticals Incorporated 1,583
Vertex Pharmaceuticals (europe) Limited 17
Virochem Pharma Inc. 7
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2025 March (MTD) 5
2025 February 21
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IPC Class
C07D 405/12 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings linked by a chain containing hetero atoms as chain links 176
C07D 471/04 - Ortho-condensed systems 147
C07D 405/14 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing three or more hetero rings 143
A61P 35/00 - Antineoplastic agents 137
C07D 401/14 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings 133
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05 - Pharmaceutical, veterinary and sanitary products 185
42 - Scientific, technological and industrial services, research and design 35
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services 32
36 - Financial, insurance and real estate services 11
35 - Advertising and business services 10
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Pending 298
Registered / In Force 1,310
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1.

PHARMACEUTICAL COMPOSITIONS FOR TREATING CYSTIC FIBROSIS

      
Application Number 18643207
Status Pending
Filing Date 2024-04-23
First Publication Date 2025-03-27
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Chu, Cathy
  • Dhamankar, Varsha
  • Dokou, Eleni
  • Haseltine, Eric L.
  • Moskowitz, Samuel
  • Robertson, Sarah
  • Waltz, David
  • Chen, Weichao George

Abstract

A pharmaceutical composition comprising Compound I: A pharmaceutical composition comprising Compound I: A pharmaceutical composition comprising Compound I: Methods of treating cystic fibrosis comprising administering one or more of such pharmaceutical compositions to a patient.

IPC Classes  ?

  • A61K 31/4045 - Indole-alkylaminesAmides thereof, e.g. serotonin, melatonin
  • A61K 31/404 - Indoles, e.g. pindolol
  • A61K 31/443 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with oxygen as a ring hetero atom
  • A61K 31/4439 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
  • A61K 31/47 - QuinolinesIsoquinolines
  • A61P 11/00 - Drugs for disorders of the respiratory system
  • A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia

2.

Precise Excisions of Portions of Exon 44, 50, and 53 for Treatment of Duchenne Muscular Dystrophy

      
Application Number 18826808
Status Pending
Filing Date 2024-09-06
First Publication Date 2025-03-20
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Fulga, Tudor
  • Xin, Yurong
  • Min, Yi-Li
  • Ashar, Foram
  • Wang, Su
  • Liu, Jianming
  • Guo, Yang
  • Nelson, D'Anna

Abstract

Compositions and methods for treating Duchenne Muscular Dystrophy (DMD) and excising small portions of exons 44, 50, and 53 of the DMD gene are encompassed.

IPC Classes  ?

  • A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
  • A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
  • C12N 9/22 - Ribonucleases
  • C12N 15/11 - DNA or RNA fragmentsModified forms thereof
  • C12N 15/86 - Viral vectors

3.

PROCESS FOR PREPARING (2S,4S,4'S,6S)-2-METHYL-6-(1-METHYL-1H-1,2,3-TRIAZOL-4-YL)-2',-(TRIELUOROMETHYL)-4',5'-DIHYDROSPIRO[PIPERIDINE-4,7'-THIENO [2,3-C] PYRAN] -4'-OL

      
Application Number US2024046013
Publication Number 2025/059054
Status In Force
Filing Date 2024-09-10
Publication Date 2025-03-20
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor Rose, Peter Jamison

Abstract

The disclosure provides processes of preparing (2S,4S,4'S,6S)-2-methyl-6-(1-methyl-1H-1,2,3-triazol-4-yl)-2'-(trifluoromethyl)-4',5'-dihydrospiro[piperidine-4,7'-thieno[2,3-c]pyran]-4'-ol (Compound (I)), stereoisomers of Compound (I), deuterated derivatives of Compound (I), and deuterated derivatives of stereoisomers of Compound (I).

IPC Classes  ?

  • C07D 495/20 - Spiro-condensed systems
  • A61K 31/4353 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
  • A61K 31/438 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom the ring being spiro-condensed with carbocyclic or heterocyclic ring systems
  • A61P 35/00 - Antineoplastic agents
  • A61P 43/00 - Drugs for specific purposes, not provided for in groups

4.

CRYSTALLINE FORMS OF C21H22Cl2N4O2

      
Application Number 18960604
Status Pending
Filing Date 2024-11-26
First Publication Date 2025-03-13
Owner
  • BIOMED VALLEY DISCOVERIES, INC. (USA)
  • VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Decrescenzo, Gary
  • Welsch, Dean
  • Vlahova, Petinka I.
  • Boerrigter, Stephan X.M.
  • Aronov, Alexander
  • Keshavarz-Shokri, Ali
  • Scangas, Alexander N.
  • Stavropoulos, Kathy
  • Littler, Benjamin
  • Kadiyala, Irina Nikolaevna
  • Alargova, Rossitza Gueorguieva

Abstract

The present invention provides crystalline forms of a compound of formula (I): The present invention provides crystalline forms of a compound of formula (I): The present invention provides crystalline forms of a compound of formula (I): Also provided are pharmaceutical compositions that include the provided crystalline forms and methods of using the provided crystalline forms and pharmaceutical compositions for the treatment of cancer.

IPC Classes  ?

  • C07D 401/04 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings directly linked by a ring-member-to-ring- member bond
  • A61K 31/4439 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
  • A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
  • A61P 35/00 - Antineoplastic agents

5.

WHERE PAIN STARTS JOURNAVX BEGINS

      
Serial Number 99075786
Status Pending
Filing Date 2025-03-10
Owner Vertex Pharmaceuticals Incorporated ()
NICE Classes  ?
  • 35 - Advertising and business services
  • 44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services

Goods & Services

Providing medical information, namely, consumer product information regarding medical products to patients and healthcare professionals Providing medical information, namely, information regarding medical conditions, treatments to patients and healthcare professionals; medical consultation, namely, providing advice to healthcare professionals regarding medical conditions and treatment

6.

Materials and Methods for Treatment of Duchenne Muscular Dystrophy

      
Application Number 18760930
Status Pending
Filing Date 2024-07-01
First Publication Date 2025-02-27
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Kabadi, Ami Meda
  • Cowan, Chad Albert
  • Lundberg, Ante Sven

Abstract

The present application provides materials and methods for treating a patient with Duchenne Muscular Dystrophy (DMD) both ex vivo and in vivo. In addition, the present application provides materials and methods for editing a dystrophin gene in a cell by genome editing.

IPC Classes  ?

  • A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
  • A61K 38/46 - Hydrolases (3)
  • A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
  • A61P 25/14 - Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
  • C12N 9/22 - Ribonucleases
  • C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
  • C12N 15/90 - Stable introduction of foreign DNA into chromosome

7.

GCN2 INHIBITORS AND USES THEREOF

      
Application Number 18775203
Status Pending
Filing Date 2024-07-17
First Publication Date 2025-02-20
Owner
  • Merck Patent GmbH (Germany)
  • Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Bleich, Matthew
  • Charrier, Jean-Damien
  • Dong, Huijun
  • Durrant, Steven
  • Eno, Meredith Suzanne
  • Etxebarria I Jardi, Gorka
  • Everitt, Simon
  • Fraysse, Damien
  • Knegtel, Ronald
  • Mochalkin, Igor
  • North, Kiri
  • Porichis, Filippos
  • Qiu, Hui
  • Pullin, Robert
  • Storck, Pierre-Henri
  • Twin, Heather Clare
  • Xiao, Yufang

Abstract

The present invention provides compounds, compositions thereof, and methods of using the same.

IPC Classes  ?

  • C07D 471/04 - Ortho-condensed systems
  • A61P 35/00 - Antineoplastic agents
  • C07D 519/00 - Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups or

8.

CELL THERAPY FOR DIABETES

      
Application Number 18786200
Status Pending
Filing Date 2024-07-26
First Publication Date 2025-02-20
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Bruinsma, Bote
  • Vergani, Andrea
  • Pagliuca, Felicia
  • Harb, George
  • Gaglia, Jason

Abstract

Disclosed herein are methods of treating a subject having diabetes with a population of cells expressing ISL1 and one or more immunosuppressive reagents, such as an anti-thymocyte globulin binding moiety.

IPC Classes  ?

  • A61K 35/12 - Materials from mammalsCompositions comprising non-specified tissues or cellsCompositions comprising non-embryonic stem cellsGenetically modified cells
  • A61K 31/365 - Lactones
  • A61K 31/453 - Non-condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a six-membered ring with oxygen as a ring hetero atom
  • A61K 31/56 - Compounds containing cyclopenta[a]hydrophenanthrene ring systemsDerivatives thereof, e.g. steroids
  • A61P 3/10 - Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics

9.

CRYSTALLINE FORMS OF CFTR MODULATORS

      
Application Number 18493667
Status Pending
Filing Date 2023-10-24
First Publication Date 2025-02-13
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Shi, Yi
  • Gagnon, Kevin J.
  • Li, Jicong
  • Lu, Jennifer
  • Medek, Ales
  • Shrestha, Muna
  • Waldo, Michael
  • Zhang, Beili
  • Zwicker, Carl L.
  • Anderson, Corey Don
  • Clemens, Jeremy J.
  • Cleveland, Thomas
  • Coon, Timothy Richard
  • Frieman, Bryan
  • Grootenhuis, Peter
  • Hadida Ruah, Sara Sabina
  • Mccartney, Jason
  • Miller, Mark Thomas
  • Paraselli, Prasuna
  • Pierre, Fabrice
  • Swift, Sara E.
  • Zhou, Jinglan

Abstract

Crystalline forms of Compound I: Crystalline forms of Compound I: pharmaceutically acceptable salts thereof, and solvates and hydrates thereof are disclosed. Pharmaceutical compositions comprising the same, methods of treating cystic fibrosis using the same, and methods for making the same are also disclosed.

IPC Classes  ?

10.

AMKADY

      
Serial Number 99031073
Status Pending
Filing Date 2025-02-06
Owner Vertex Pharmaceuticals Incorporated ()
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceuticals and medicines for the treatment of cystic fibrosis, pain, sickle cell disease, beta thalassemia, muscular dystrophy, diabetes, respiratory diseases, liver disease, kidney disease, alpha-1 antitrypsin deficiency and APOL1-mediated diseases

11.

ANKESDRY

      
Serial Number 99031093
Status Pending
Filing Date 2025-02-06
Owner Vertex Pharmaceuticals Incorporated ()
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceuticals and medicines for the treatment of cystic fibrosis, pain, sickle cell disease, beta thalassemia, muscular dystrophy, diabetes, respiratory diseases, liver disease, kidney disease, alpha-1 antitrypsin deficiency and APOL1-mediated diseases

12.

APOLMEYA

      
Serial Number 99031096
Status Pending
Filing Date 2025-02-06
Owner Vertex Pharmaceuticals Incorporated ()
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceuticals and medicines for the treatment of cystic fibrosis, pain, sickle cell disease, beta thalassemia, muscular dystrophy, diabetes, respiratory diseases, liver disease, kidney disease, alpha-1 antitrypsin deficiency and APOL1-mediated diseases

13.

VOCEMEO

      
Serial Number 99031155
Status Pending
Filing Date 2025-02-06
Owner Vertex Pharmaceuticals Incorporated ()
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceuticals and medicines for the treatment of cystic fibrosis, pain, sickle cell disease, beta thalassemia, muscular dystrophy, diabetes, respiratory diseases, liver disease, kidney disease, alpha-1 antitrypsin deficiency and APOL1-mediated kidney disease

14.

VOCEMIVY

      
Serial Number 99031163
Status Pending
Filing Date 2025-02-06
Owner Vertex Pharmaceuticals Incorporated ()
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceuticals and medicines for the treatment of cystic fibrosis, pain, sickle cell disease, beta thalassemia, muscular dystrophy, diabetes, respiratory diseases, liver disease, kidney disease, alpha-1 antitrypsin deficiency and APOL1-mediated kidney disease

15.

CRISPR-CAS9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells and Uses Thereof

      
Application Number 18929289
Status Pending
Filing Date 2024-10-28
First Publication Date 2025-02-06
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Morawa, Ewelina
  • Chakraborty, Tirtha
  • Lundberg, Ante Sven
  • Ho, Tony
  • Sandler, Laura
  • Eustace, Brenda
  • Rossert, Jerome
  • Kauffman, Robert

Abstract

Provided herein, in some embodiments, are methods and compositions for treatment of subjects with β-thalassemia and subjects with severe sickle cell disease using autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells.

IPC Classes  ?

  • A61K 35/28 - Bone marrowHaematopoietic stem cellsMesenchymal stem cells of any origin, e.g. adipose-derived stem cells
  • A61K 31/255 - Esters, e.g. nitroglycerine, selenocyanates of sulfoxy acids or sulfur analogues thereof
  • A61K 31/395 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
  • A61K 35/18 - Erythrocytes
  • A61K 38/19 - CytokinesLymphokinesInterferons
  • A61P 7/00 - Drugs for disorders of the blood or the extracellular fluid
  • C12N 5/0789 - Stem cellsMultipotent progenitor cells
  • C12N 9/22 - Ribonucleases
  • C12N 15/11 - DNA or RNA fragmentsModified forms thereof

16.

AMKYDRI

      
Serial Number 99031079
Status Pending
Filing Date 2025-02-06
Owner Vertex Pharmaceuticals Incorporated ()
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceuticals and medicines for the treatment of cystic fibrosis, pain, sickle cell disease, beta thalassemia, muscular dystrophy, diabetes, respiratory diseases, liver disease, kidney disease, alpha-1 antitrypsin deficiency and APOL1-mediated diseases

17.

AMKESDI

      
Serial Number 99031086
Status Pending
Filing Date 2025-02-06
Owner Vertex Pharmaceuticals Incorporated ()
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceuticals and medicines for the treatment of cystic fibrosis, pain, sickle cell disease, beta thalassemia, muscular dystrophy, diabetes, respiratory diseases, liver disease, kidney disease, alpha-1 antitrypsin deficiency and APOL1-mediated diseases

18.

KIFAMKA

      
Serial Number 99031102
Status Pending
Filing Date 2025-02-06
Owner Vertex Pharmaceuticals Incorporated ()
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceuticals and medicines for the treatment of cystic fibrosis, pain, sickle cell disease, beta thalassemia, muscular dystrophy, diabetes, respiratory diseases, liver disease, kidney disease, alpha-1 antitrypsin deficiency and APOL1-mediated diseases

19.

AMKADENZ

      
Serial Number 99031105
Status Pending
Filing Date 2025-02-06
Owner Vertex Pharmaceuticals Incorporated ()
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceuticals and medicines for the treatment of cystic fibrosis, pain, sickle cell disease, beta thalassemia, muscular dystrophy, diabetes, respiratory diseases, liver disease, kidney disease, alpha-1 antitrypsin deficiency and APOL1-mediated diseases

20.

LIFVOCIO

      
Serial Number 99031143
Status Pending
Filing Date 2025-02-06
Owner Vertex Pharmaceuticals Incorporated ()
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceuticals and medicines for the treatment of cystic fibrosis, pain, sickle cell disease, beta thalassemia, muscular dystrophy, diabetes, respiratory diseases, liver disease, kidney disease, alpha-1 antitrypsin deficiency and APOL1-mediated kidney disease

21.

MYVOCIO

      
Serial Number 99031149
Status Pending
Filing Date 2025-02-06
Owner Vertex Pharmaceuticals Incorporated ()
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceuticals and medicines for the treatment of cystic fibrosis, pain, sickle cell disease, beta thalassemia, muscular dystrophy, diabetes, respiratory diseases, liver disease, kidney disease, alpha-1 antitrypsin deficiency and APOL1-mediated kidney disease

22.

VOCEMBLIO

      
Serial Number 99031152
Status Pending
Filing Date 2025-02-06
Owner Vertex Pharmaceuticals Incorporated ()
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceuticals and medicines for the treatment of cystic fibrosis, pain, sickle cell disease, beta thalassemia, muscular dystrophy, diabetes, respiratory diseases, liver disease, kidney disease, alpha-1 antitrypsin deficiency and APOL1-mediated kidney disease

23.

VOXFINLY

      
Serial Number 99031165
Status Pending
Filing Date 2025-02-06
Owner Vertex Pharmaceuticals Incorporated ()
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceuticals and medicines for the treatment of cystic fibrosis, pain, sickle cell disease, beta thalassemia, muscular dystrophy, diabetes, respiratory diseases, liver disease, kidney disease, alpha-1 antitrypsin deficiency and APOL1-mediated kidney disease

24.

VOXJEMI

      
Serial Number 99031170
Status Pending
Filing Date 2025-02-06
Owner Vertex Pharmaceuticals Incorporated ()
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceuticals and medicines for the treatment of cystic fibrosis, pain, sickle cell disease, beta thalassemia, muscular dystrophy, diabetes, respiratory diseases, liver disease, kidney disease, alpha-1 antitrypsin deficiency and APOL1-mediated kidney disease

25.

INHIBITORS OF APOL1 AND METHODS OF USING SAME

      
Application Number 18745762
Status Pending
Filing Date 2024-06-17
First Publication Date 2025-02-06
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Cao, Jingrong
  • Come, Jon H.
  • Dakin, Leslie A.
  • Denis, Francois
  • Dorsch, Warren A.
  • Fortier, Anne
  • Hamel, Martine
  • Krueger, Elaine B.
  • Ledford, Brian
  • Nanthakumar, Suganthini S.
  • Nicolas, Olivier
  • Sayegh, Camil
  • Senter, Timothy J.
  • Wang, Tiansheng
  • Brodney, Michael
  • Hu, Kan-Nian
  • Rose, Peter
  • Gagnon, Kevin
  • Shi, Yi
  • Shrestha, Muna
  • Medek, Ales
  • Witkos, Faith

Abstract

The disclosure provides at least one entity chosen from compounds of formula (I), solid state forms of the same, compositions comprising the same, and methods of using the same, including use in treating focal segmental glomerulosclerosis (FSGS) and/or non-diabetic kidney disease (NDKD).

IPC Classes  ?

  • C07D 403/12 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group containing two hetero rings linked by a chain containing hetero atoms as chain links
  • C07D 405/14 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing three or more hetero rings

26.

CELL THERAPY FOR DIABETES

      
Application Number US2024039866
Publication Number 2025/029671
Status In Force
Filing Date 2024-07-26
Publication Date 2025-02-06
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Bruinsma, Bote
  • Pagliuca, Felicia
  • Harb, George
  • Gaglia, Jason
  • Vergani, Andrea

Abstract

Disclosed herein are methods of treating a subject having diabetes with a population of cells expressing ISL1 and one or more immunosuppressive reagents, such as an anti-thymocyte globulin binding moiety.

IPC Classes  ?

  • A61K 35/14 - BloodArtificial blood
  • A61K 35/28 - Bone marrowHaematopoietic stem cellsMesenchymal stem cells of any origin, e.g. adipose-derived stem cells
  • A61P 37/02 - Immunomodulators
  • C12N 5/07 - Animal cells or tissues
  • A61P 3/10 - Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
  • A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
  • C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants

27.

JOURNAVX+YOU

      
Serial Number 99023402
Status Pending
Filing Date 2025-01-30
Owner Vertex Pharmaceuticals Incorporated ()
NICE Classes  ? 44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services

Goods & Services

Providing health information to patients, caregivers and health care professionals; providing health care information; providing patient education support services relating to medications, cell therapy and genetic therapy, in the nature of providing information about healthcare

28.

MACROENCAPSULATION DEVICE IMPLANTATION AND TRANSPORT SYSTEM AND METHODS

      
Application Number 18777686
Status Pending
Filing Date 2024-07-19
First Publication Date 2025-01-23
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Billings, Megan
  • Mills, John
  • Mills, John Fraser
  • Nguyen, Noah
  • Thanos, Christopher

Abstract

Macroencapsulation implantation devices and containers for storing a macroencapsulation device disposed within a receptacle of a macroencapsulation implantation device as well as related methods are described. In some embodiments, a macroencapsulation implantation device may include a handle that is configured to be selectively engaged with a receptacle including a recess in which a macroencapsulation device may be disposed. In some embodiments, a container may be configured to maintain a receptacle of a macroencapsulation implantation device, and a macroencapsulation device disposed therein, in a desired pose within an internal volume of the container.

IPC Classes  ?

  • A61B 17/34 - TrocarsPuncturing needles
  • A61J 1/03 - Containers specially adapted for medical or pharmaceutical purposes for pills or tablets
  • A61K 9/00 - Medicinal preparations characterised by special physical form

29.

COMPLEMENT FACTOR I-RELATED COMPOSITIONS AND METHODS

      
Application Number 18750921
Status Pending
Filing Date 2024-06-21
First Publication Date 2025-01-09
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Blouse, Grant E.
  • Jensen, Jan Kristian
  • Oldenburg, Emil
  • Schar, Christine René
  • Jendroszek, Agnieszka
  • Mcguire, James N.
  • Iyer, Shyam Rajan
  • Pelot, Kyle A.

Abstract

Provided herein are Complement Factor I (CFI) variants and CFI containing fusion constructs that exhibit at least one improved characteristic relative to a wild type CFI. The CFI variants and fusion constructs of the disclosure can exhibit tunable specificity and activity. The CFI variants and fusion constructs provided herein may be useful for treating a disease or condition associated with dysregulation of the complement system or a deficiency of CFI.

IPC Classes  ?

  • C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
  • A61K 47/60 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
  • A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection
  • C07K 14/76 - Albumins

30.

COMPLEMENT FACTOR-I FORMULATIONS

      
Application Number 18750927
Status Pending
Filing Date 2024-06-21
First Publication Date 2025-01-09
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Shan, Bing
  • Connolly, Brian David
  • Balan, Sibu
  • Chatterji, Anju

Abstract

Provided herein are pharmaceutically acceptable formulations comprising wild type Complement Factor I (CFI) and variants thereof; in some embodiments the wild type CFI and variants thereof are part of a fusion construct, e.g. a fusion construct comprising human serum albumin. The formulations stabilize CFI against acute stresses during storage in either a liquid or lyophilized state. Also provided are methods of making the formulations, and methods of using the formulations in the treatment of diseases.

IPC Classes  ?

  • A61K 38/48 - Hydrolases (3) acting on peptide bonds (3.4)

31.

DIFFERENTIATION OF PANCREATIC ENDOCRINE CELLS

      
Application Number 18824576
Status Pending
Filing Date 2024-09-04
First Publication Date 2025-01-09
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Harb, George
  • Xie, Chunhi

Abstract

Disclosed herein are compositions and methods related to differentiation of stem cells into pancreatic endocrine cells. In some aspects, the methods provided herein relate to generation of pancreatic β cell, α cell, δ cells, and EC cells in vitro. In some aspects, the disclosure provides pharmaceutical compositions including the cells generated according to the methods disclosed herein, as well as methods of treatment making use thereof.

IPC Classes  ?

  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues
  • A61K 35/39 - PancreasIslets of Langerhans

32.

UREA-CONTAINING AGONISTS OF OREXIN RECEPTOR TYPE 2

      
Application Number US2024036643
Publication Number 2025/010311
Status In Force
Filing Date 2024-07-03
Publication Date 2025-01-09
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Volgraf, Matthew
  • Ewing, Todd J.A.
  • Long, Daniel D.

Abstract

Disclosed are compounds, compositions, and methods useful for preventing or treating a disease which is at least partially mediated by orexin receptor type 2.

IPC Classes  ?

  • C07D 401/12 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings linked by a chain containing hetero atoms as chain links
  • C07D 401/14 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
  • C07D 403/12 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group containing two hetero rings linked by a chain containing hetero atoms as chain links
  • C07D 403/14 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group containing three or more hetero rings
  • C07D 413/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing three or more hetero rings
  • A61K 31/496 - Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
  • A61K 31/454 - Non-condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. pimozide, domperidone
  • A61P 25/00 - Drugs for disorders of the nervous system

33.

UREA-CONTAINING AGONISTS OF OREXIN RECEPTOR TYPE 2

      
Application Number US2024036648
Publication Number 2025/010314
Status In Force
Filing Date 2024-07-03
Publication Date 2025-01-09
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Ewing, Todd J.A.
  • Long, Daniel D.
  • Volgraf, Matthew

Abstract

Disclosed are compounds, compositions, and methods useful for preventing or treating a disease which is at least partially mediated by orexin receptor 2.

IPC Classes  ?

  • C07D 401/12 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings linked by a chain containing hetero atoms as chain links
  • C07D 401/14 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
  • C07D 413/12 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings linked by a chain containing hetero atoms as chain links
  • C07D 413/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing three or more hetero rings
  • A61K 31/435 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
  • A61K 31/4245 - Oxadiazoles
  • A61P 25/00 - Drugs for disorders of the nervous system

34.

Differentiation of pancreatic endocrine cells

      
Application Number 18824588
Grant Number 12227765
Status In Force
Filing Date 2024-09-04
First Publication Date 2024-12-26
Grant Date 2025-02-18
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Harb, George
  • Xie, Chunhi

Abstract

Disclosed herein are compositions and methods related to differentiation of stem cells into pancreatic endocrine cells. In some aspects, the methods provided herein relate to generation of pancreatic β cell, α cell, δ cells, and EC cells in vitro. In some aspects, the disclosure provides pharmaceutical compositions including the cells generated according to the methods disclosed herein, as well as methods of treatment making use thereof.

IPC Classes  ?

  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues
  • A61K 35/39 - PancreasIslets of Langerhans

35.

ENHANCED DIFFERENTIATION OF BETA CELLS

      
Application Number 18270314
Status Pending
Filing Date 2021-12-28
First Publication Date 2024-12-26
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Carey, Bryce
  • Szymaniak, Aleksander
  • Thanos, Christopher

Abstract

Provided herein are, inter alia, compositions and methods for improved production of SC-β cells in vitro. For example, provided are novel formulations and differentiation methods that result in higher cell yields and recoveries, increased numbers and relative percentages of SC-β cells, enhanced stability and shelf-life of SC-β cells, SC-islet clusters with advantageous characteristics such as reduced size and increased uniformity, improved function of the SC-β cells in vitro, and improved viability, function, and reduced immunogenicity after transplantation. The disclosed compositions and methods can be employed in the manufacture of SC-islets for human therapeutic use.

IPC Classes  ?

  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues
  • A61K 35/39 - PancreasIslets of Langerhans

36.

Precise Excisions of Portions of Exons for Treatment of Duchenne Muscular Dystrophy

      
Application Number 18826876
Status Pending
Filing Date 2024-09-06
First Publication Date 2024-12-19
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Fulga, Tudor
  • Min, Yi-Li
  • Ashar, Foram
  • Wang, Su
  • Liu, Jianming

Abstract

Compositions and methods for treating Duchenne Muscular Dystrophy (DMD) and excising small portions of exons of the DMD gene are encompassed.

IPC Classes  ?

  • A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
  • C12N 9/22 - Ribonucleases
  • C12N 15/11 - DNA or RNA fragmentsModified forms thereof
  • C12N 15/90 - Stable introduction of foreign DNA into chromosome

37.

N-(HYDROXYALKYL (HETERO)ARYL) TETRAHYDROFURAN CARBOXAMIDE ANALOGS AS MODULATORS OF SODIUM CHANNELS

      
Application Number 18566282
Status Pending
Filing Date 2022-06-03
First Publication Date 2024-12-05
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Beck, Elizabeth Mary
  • Pullin, Robert
  • Etxebarria Jardi, Gorka
  • Stamos, Dean
  • Schmidt, Yvonne
  • Pontillo, Joseph
  • Thomson, Stephen Andrew
  • Shaw, David Matthew
  • Ahmad, Nadia M.
  • Carvalho Meireles, Lidio Marx
  • Skerratt, Sarah
  • Hadida Ruah, Sara S.
  • Neubert, Timothy Donald
  • Hurley, Dennis James
  • Kintzer, Alexander
  • Durrant, Steven John
  • Wray, Christopher
  • Virani, Anisa Nizarali
  • North, Kiri
  • Jacques, Reece
  • Miller, Mark Thomas
  • Zhou, Jinglan
  • Knegtel, Ronald Marcellus
  • Chudyk, Ewa Iwona
  • Pinder, Joanne Louise
  • Sousa, Bruno Artur
  • Dodd, James
  • O'Donnell, Michael Edward
  • Galan, Bhairavi
  • Stevens, Rebecca E.

Abstract

Compounds of formula (I) and pharmaceutically acceptable salts thereof, useful as inhibitors of sodium channels are provided. Also provided are pharmaceutical compositions comprising the compounds or pharmaceutically acceptable salts and methods of using the compounds, pharmaceutically acceptable salts, and pharmaceutical compositions in the treatment of various disorders, including pain. Compounds of formula (I) and pharmaceutically acceptable salts thereof, useful as inhibitors of sodium channels are provided. Also provided are pharmaceutical compositions comprising the compounds or pharmaceutically acceptable salts and methods of using the compounds, pharmaceutically acceptable salts, and pharmaceutical compositions in the treatment of various disorders, including pain.

IPC Classes  ?

  • C07D 413/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing three or more hetero rings
  • A61K 31/4355 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having oxygen as a ring hetero atom
  • A61K 31/443 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with oxygen as a ring hetero atom
  • A61K 31/506 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
  • A61K 31/5377 - 1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
  • C07D 405/12 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings linked by a chain containing hetero atoms as chain links
  • C07D 405/14 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing three or more hetero rings
  • C07D 491/04 - Ortho-condensed systems

38.

METHODS FOR DIRECTING LIPID NANOPARTICLES IN VIVO

      
Application Number US2024031121
Publication Number 2024/249354
Status In Force
Filing Date 2024-05-24
Publication Date 2024-12-05
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Dokshin, Gregoriy, Aleksandrovich
  • Biswas, Souvik

Abstract

Compositions and methods for enhancing payload-based gene therapy by increasing the percentage of payload delivered to a non-liver target in a subject by blocking binding of LDL to LDL receptors (LDLR) in the liver, and then administering a payload-based therapy targeting a non-liver tissue.

IPC Classes  ?

  • C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
  • A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
  • A61K 47/69 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
  • A61K 9/127 - Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
  • A61K 9/51 - Nanocapsules

39.

AFFINITY PURIFICATION METHODS FOR ADENO ASSOCIATED VIRUSES (AAVS)

      
Application Number US2024031719
Publication Number 2024/249664
Status In Force
Filing Date 2024-05-30
Publication Date 2024-12-05
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Clark, Lars
  • Kerstetter, Randall

Abstract

Provided herein are isolated peptides that can bind an AAV (such as an AAV9). Methods of using the peptides in obtaining an AAV for use in a variety of gene transfer and/or other applications are also provided. The disclosed methods separate the AAV from all or a portion of other components of an AAV starting preparation

40.

Substituted tetrahydrofurans as modulators of sodium channels

      
Application Number 18524339
Grant Number 12247021
Status In Force
Filing Date 2023-11-30
First Publication Date 2024-11-28
Grant Date 2025-03-11
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Durrant, Steven John
  • Ahmad, Nadia M.
  • Beck, Elizabeth Mary
  • Carvalho Meireles, Lidio Marx
  • Chudyk, Ewa Iwona
  • Etxebarria Jardi, Gorka
  • Galan, Bhairavi
  • Hadida Ruah, Sara S.
  • Hurley, Dennis James
  • Knegtel, Ronald Marcellus
  • Neubert, Timothy Donald
  • Pinder, Joanne Louise
  • Pontillo, Joseph
  • Pullin, Robert
  • Schmidt, Yvonne
  • Shaw, David Matthew
  • Skerratt, Sarah
  • Stamos, Dean
  • Thomson, Stephen Andrew
  • Virani, Anisa Nizarali
  • Wray, Christopher

Abstract

Compounds, and pharmaceutically acceptable salts thereof, useful as inhibitors of sodium channels are provided. Also provided are pharmaceutical compositions comprising the compounds or pharmaceutically acceptable salts and methods of using the compounds, pharmaceutically acceptable salts, and pharmaceutical compositions in the treatment of various disorders, including pain.

IPC Classes  ?

  • C07D 405/12 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings linked by a chain containing hetero atoms as chain links
  • C07D 307/24 - Carbon atoms having three bonds to hetero atoms with at the most one bond to halogen
  • C07D 405/14 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing three or more hetero rings

41.

alyftrek

      
Application Number 1824178
Status Registered
Filing Date 2024-10-15
Registration Date 2024-10-15
Owner Vertex Pharmaceuticals Incorporated (USA)
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceutical preparations for the treatment of cystic fibrosis.

42.

COMPACT PROTEINS

      
Application Number US2024028693
Publication Number 2024/238296
Status In Force
Filing Date 2024-05-10
Publication Date 2024-11-21
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Dokshin, Gregoriy A.
  • Good, Michelle
  • Nelson, D'Anna M.
  • Takemoto, Darin Katsumi
  • Swift, Sara Elizabeth
  • Saha, Jishnu
  • Mcgaughey Nicholls, Georgia

Abstract

Disclosed herein are compositions of compact proteins such as compact nucleobase editors that may be used, in some embodiments, for genome editing technologies.

43.

Miscellaneous Design

      
Application Number 1824179
Status Registered
Filing Date 2024-10-15
Registration Date 2024-10-15
Owner Vertex Pharmaceuticals Incorporated (USA)
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceutical preparations for the treatment of cystic fibrosis.

44.

STEM CELL DERIVED PANCREATIC ISLET DIFFERENTIATION

      
Application Number 18647137
Status Pending
Filing Date 2024-04-26
First Publication Date 2024-11-21
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Harb, George
  • Xie, Chunhui
  • Carey, Bryce W.
  • Szymaniak, Aleksander
  • Thanos, Christopher
  • Thompson, Evrett
  • Chinn, Rebecca
  • Raj, Suyash

Abstract

Disclosed herein are compositions and methods related to differentiation of stem cells into pancreatic islet cells. In some aspects, the methods provided herein relate to generation of pancreatic β cell, α cell, δ cells, and EC cells in vitro. In some aspects, the disclosure provides pharmaceutical compositions including the cells generated according to the methods disclosed herein, as well as methods of treatment making use thereof.

IPC Classes  ?

  • A61K 35/39 - PancreasIslets of Langerhans
  • A61K 38/28 - Insulins
  • A61P 3/10 - Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues

45.

SUBSTITUTED TETRAHYDROFURAN-2-CARBOXAMIDES AS MODULATORS OF SODIUM CHANNELS

      
Application Number 18566297
Status Pending
Filing Date 2022-06-03
First Publication Date 2024-11-07
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Beck, Elizabeth Mary
  • Pullin, Robert
  • Etxebarria Jardi, Gorka
  • Stamos, Dean
  • Schmidt, Yvonne
  • Pontillo, Joseph
  • Thomson, Stephen Andrew
  • Shaw, David Matthew
  • Ahmad, Nadia M.
  • Carvalho Meireles, Lidio Marx
  • Skerratt, Sarah
  • Hadida Ruah, Sara S.
  • Miller, Mark Thomas
  • Neubert, Timothy Donald
  • Hurley, Dennis James
  • Zhou, Jinglan
  • Kintzer, Alexander
  • Durrant, Steven John
  • Wray, Christopher
  • Virani, Anisa Nizarali
  • North, Kiri
  • Dodd, James
  • Jacques, Reece
  • Geddis, Stephen Michael
  • Galan, Bhairavi
  • Knegtel, Ronald Marcellus
  • Chudyk, Ewa Iwona
  • Pinder, Joanne Louise
  • Sousa, Bruno Artur
  • Stevens, Rebecca E.
  • Wright, Miranda Adele
  • Twin, Heather Clare
  • Mui, James Jun Bon

Abstract

Compounds of formula I and pharmaceutically acceptable salts thereof, useful as inhibitors of sodium channels are provided. Also provided are pharmaceutical compositions comprising the compounds or pharmaceutically acceptable salts and methods of using the compounds, pharmaceutically acceptable salts, and pharmaceutical compositions in the treatment of various disorders, including pain. Compounds of formula I and pharmaceutically acceptable salts thereof, useful as inhibitors of sodium channels are provided. Also provided are pharmaceutical compositions comprising the compounds or pharmaceutically acceptable salts and methods of using the compounds, pharmaceutically acceptable salts, and pharmaceutical compositions in the treatment of various disorders, including pain.

IPC Classes  ?

  • C07D 405/12 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings linked by a chain containing hetero atoms as chain links
  • A61K 31/341 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide not condensed with another ring, e.g. ranitidine, furosemide, bufetolol, muscarine
  • A61K 31/351 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom not condensed with another ring
  • A61K 31/38 - Heterocyclic compounds having sulfur as a ring hetero atom
  • A61K 31/397 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having four-membered rings, e.g. azetidine
  • A61K 31/4025 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil not condensed and containing further heterocyclic rings, e.g. cromakalim
  • A61K 31/407 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil condensed with heterocyclic ring systems, e.g. ketorolac, physostigmine
  • A61K 31/4155 - 1,2-Diazoles not condensed and containing further heterocyclic rings
  • A61K 31/416 - 1,2-Diazoles condensed with carbocyclic ring systems, e.g. indazole
  • A61K 31/4178 - 1,3-Diazoles not condensed and containing further heterocyclic rings, e.g. pilocarpine, nitrofurantoin
  • A61K 31/422 - Oxazoles not condensed and containing further heterocyclic rings
  • A61K 31/4245 - Oxadiazoles
  • A61K 31/427 - Thiazoles not condensed and containing further heterocyclic rings
  • A61K 31/437 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
  • A61K 31/443 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with oxygen as a ring hetero atom
  • A61K 31/4433 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a six-membered ring with oxygen as a ring hetero atom
  • A61K 31/4439 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
  • A61K 31/4525 - Non-condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a five-membered ring with oxygen as a ring hetero atom
  • A61K 31/4545 - Non-condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring hetero atom, e.g. pipamperone, anabasine
  • A61K 31/4709 - Non-condensed quinolines containing further heterocyclic rings
  • A61K 31/496 - Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
  • A61K 31/4995 - Pyrazines or piperazines forming part of bridged ring systems
  • A61K 31/501 - PyridazinesHydrogenated pyridazines not condensed and containing further heterocyclic rings
  • A61K 31/506 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
  • A61K 31/5377 - 1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
  • A61K 31/5383 - 1,4-Oxazines, e.g. morpholine ortho- or peri-condensed with heterocyclic ring systems
  • A61K 31/55 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
  • A61K 31/551 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole having two nitrogens as ring hetero atoms, e.g. clozapine, dilazep
  • A61K 31/665 - Phosphorus compounds having oxygen as a ring hetero atom, e.g. fosfomycin
  • C07D 307/24 - Carbon atoms having three bonds to hetero atoms with at the most one bond to halogen
  • C07D 405/14 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing three or more hetero rings
  • C07D 407/12 - Heterocyclic compounds containing two or more hetero rings, at least one ring having oxygen atoms as the only ring hetero atoms, not provided for by group containing two hetero rings linked by a chain containing hetero atoms as chain links
  • C07D 409/12 - Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms containing two hetero rings linked by a chain containing hetero atoms as chain links
  • C07D 413/12 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings linked by a chain containing hetero atoms as chain links
  • C07D 413/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing three or more hetero rings
  • C07D 417/12 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group containing two hetero rings linked by a chain containing hetero atoms as chain links
  • C07D 471/04 - Ortho-condensed systems
  • C07D 487/04 - Ortho-condensed systems
  • C07D 487/08 - Bridged systems
  • C07D 491/048 - Ortho-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring the oxygen-containing ring being five-membered
  • C07D 491/107 - Spiro-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring
  • C07D 498/04 - Ortho-condensed systems
  • C07D 498/08 - Bridged systems
  • C07F 9/655 - Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom having oxygen atoms, with or without sulfur, selenium, or tellurium atoms, as the only ring hetero atoms

46.

Modulators of Cystic Fibrosis Transmembrane Conductance Regulator, Pharmaceutical Compositions, Methods of Treatment, and Process for Making the Modulators

      
Application Number 18484185
Status Pending
Filing Date 2023-10-10
First Publication Date 2024-11-07
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Clemens, Jeremy J.
  • Abela, Alexander Russell
  • Anderson, Corey Don
  • Busch, Brett B.
  • Chen, Weichao George
  • Cleveland, Thomas
  • Coon, Timothy Richard
  • Frieman, Bryan
  • Ghirmai, Senait G.
  • Grootenhuis, Peter
  • Gulevich, Anton V.
  • Hadida Ruah, Sara Sabina
  • Hsia, Clara Kuang-Ju
  • Kang, Ping
  • Khatuya, Haripada
  • Mccartney, Jason
  • Miller, Mark Thomas
  • Paraselli, Prasuna
  • Pierre, Fabrice
  • Swift, Sara E.
  • Termin, Andreas
  • Uy, Johnny
  • Vogel, Carl V.
  • Zhou, Jinglan

Abstract

Compounds of Formula (I): Compounds of Formula (I): Compounds of Formula (I): pharmaceutically acceptable salts thereof, deuterated derivatives of any of the foregoing, and metabolites of any of the foregoing are disclosed. Pharmaceutical compositions comprising the same, methods of treating cystic fibrosis using the same, and methods for making the same are also disclosed.

IPC Classes  ?

  • C07D 513/22 - Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for in groups , or in which the condensed system contains four or more hetero rings
  • A61K 31/404 - Indoles, e.g. pindolol
  • A61K 31/439 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom the ring forming part of a bridged ring system, e.g. quinuclidine
  • A61K 31/47 - QuinolinesIsoquinolines
  • A61P 11/00 - Drugs for disorders of the respiratory system
  • C07D 515/22 - Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen, oxygen, and sulfur atoms as the only ring hetero atoms, not provided for in groups , or in which the condensed system contains four or more hetero rings

47.

INHIBITORS OF APOL1 AND METHODS OF USING SAME

      
Application Number 18504559
Status Pending
Filing Date 2023-11-08
First Publication Date 2024-11-07
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Ahn, Jun Myun
  • Angle, Samantha
  • Brodney, Michael Aaron
  • Cao, Jingrong
  • Cochran, John E.
  • Come, Jon H.
  • Dakin, Leslie A.
  • Dolgikh, Elena
  • Maxwell, Brad D.
  • Nanthakumar, Suganthini S.
  • O'Dowd, Hardwin
  • Olsen, Jessica Howard
  • Senter, Timothy J.
  • Shimizu, Akira Joseph
  • Stone, Steven David
  • Wang, Haoxuan

Abstract

The disclosure provides at least one entity chosen from compounds of Formula I, a tautomer thereof, a deuterated derivative of that compound or tautomer, and a pharmaceutically acceptable salt of any of the foregoing, compositions comprising the same, and methods of using the same, including uses in treating APOL1-mediated diseases, including pancreatic cancer, focal segmental glomerulosclerosis (FSGS), and/or non-diabetic kidney disease (NDKD). The disclosure provides at least one entity chosen from compounds of Formula I, a tautomer thereof, a deuterated derivative of that compound or tautomer, and a pharmaceutically acceptable salt of any of the foregoing, compositions comprising the same, and methods of using the same, including uses in treating APOL1-mediated diseases, including pancreatic cancer, focal segmental glomerulosclerosis (FSGS), and/or non-diabetic kidney disease (NDKD).

IPC Classes  ?

  • C07D 495/20 - Spiro-condensed systems
  • C07D 519/00 - Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups or

48.

STEM CELL DIFFERENTIATION AND CHEMICAL COMPOUNDS

      
Application Number 18707756
Status Pending
Filing Date 2022-11-07
First Publication Date 2024-10-31
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Xie, Chunhui
  • Harb, George
  • Ryu, Elizabeth
  • Teceno, Nicholas
  • Farrar, Christopher

Abstract

Disclosed herein are compositions and methods related to differentiation of stem cells into pancreatic cells. In some aspects, the methods provided herein relate to generation of pancreatic β cell, α cell, δ cells, and EC cells in vitro in the presence of one or more chemical compounds that inhibit PI3K/Akt/mTOR signaling. In some aspects, the disclosure provides pharmaceutical compositions including the cells generated according to the methods disclosed herein, as well as methods of making use thereof.

IPC Classes  ?

  • A61K 35/39 - PancreasIslets of Langerhans
  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues

49.

MACROENCAPSULATION DEVICES

      
Application Number 18682738
Status Pending
Filing Date 2022-08-16
First Publication Date 2024-10-24
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Thanos, Christopher
  • Mills, John
  • Watson, Matthew
  • Rajendran, Rahul R.
  • Nguyen, Noah

Abstract

Macroencapsulation devices and related methods of manufacture are described where bonded membranes of a device may be mounted to an associated frame in an arrangement to provide stress relief between the frame and a seal perimeter of the bonded membranes. The seal perimeter may be arranged radially inward from an outer perimeter of the membranes such that when the membranes are mounted to a corresponding perimeter frame, the seal perimeter is spaced radially inwards from the frame with a unbonded portion of the one or more membranes disposed between the frame and seal perimeter.

IPC Classes  ?

  • C12M 1/12 - Apparatus for enzymology or microbiology with sterilisation, filtration, or dialysis means

50.

CELL HOUSING DEVICE

      
Application Number 18471622
Status Pending
Filing Date 2023-09-21
First Publication Date 2024-10-17
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Thanos, Christopher
  • Lavin, Danya M.
  • Bintz, Briannan E.
  • Bhatnagar, Divya
  • Mills, John
  • Billings, Megan

Abstract

The present disclosure provides a cell housing device and a method of manufacturing such a device that has an array of chemicals to increase the ratio of surface area to volume.

IPC Classes  ?

  • A61K 9/00 - Medicinal preparations characterised by special physical form
  • A61K 35/39 - PancreasIslets of Langerhans
  • A61K 47/32 - Macromolecular compounds obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. carbomers
  • A61K 47/34 - Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers

51.

Gene Editing of Regulatory Elements

      
Application Number 18435805
Status Pending
Filing Date 2024-02-07
First Publication Date 2024-10-17
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Ruan, Guoxiang
  • Liu, Jianming
  • Rao, Lingjun
  • Zhu, Jinjin

Abstract

Compositions and methods for disrupting and/or excising regulatory elements or portions thereof that are associated with genes that produce toxic proteins and transcripts are encompassed.

IPC Classes  ?

  • C12N 15/11 - DNA or RNA fragmentsModified forms thereof
  • C12N 9/22 - Ribonucleases
  • C12N 15/90 - Stable introduction of foreign DNA into chromosome

52.

ALYFTREK

      
Application Number 236363800
Status Pending
Filing Date 2024-10-15
Owner Vertex Pharmaceuticals Incorporated (USA)
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

(1) Pharmaceutical preparations for the treatment of cystic fibrosis.

53.

GEOMETRICAL FIGURES DESIGN

      
Application Number 236364200
Status Pending
Filing Date 2024-10-15
Owner Vertex Pharmaceuticals Incorporated (USA)
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

(1) Pharmaceutical preparations for the treatment of cystic fibrosis.

54.

MODULATORS OF ALPHA-1 ANTITRYPSIN

      
Application Number 18487690
Status Pending
Filing Date 2023-10-16
First Publication Date 2024-10-10
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Bandarage, Upul Keerthi
  • Bligh, Cavan Mckeon
  • Boucher, Diane
  • Boyd, Michael John
  • Brodney, Michael Aaron
  • Clark, Michael Philip
  • Damagnez, Veronique
  • Fanning, Lev Tyler Dewey
  • Fimognari, Robert Francis
  • Fleming, Gabrielle Simone
  • Gagnon, Kevin James
  • Garcia Barrantes, Pedro Manuel
  • Giacometti, Robert Daniel
  • Giroux, Simon
  • Grey, Jr., Ronald Lee
  • Guido, Samantha
  • Hall, Amy Beth
  • Hood, Sarah Carol
  • Hurley, Dennis James
  • Johnson, Jr., Mac Arthur
  • Jones, Peter
  • Kesavan, Sarathy
  • Lai, Mei-Hsiu
  • Liu, Siying
  • Looker, Adam
  • Maxwell, Brad
  • Maxwell, John Patrick
  • Medek, Ales
  • Nuhant, Philippe Marcel
  • Overhoff, Kirk Alan
  • Roday, Setu
  • Roeper, Stefanie
  • Ronkin, Steven M.
  • Sawant, Rupa
  • Shi, Yi
  • Shrestha, Muna
  • Sposato, Marisa
  • Stavropoulos, Kathy
  • Swett, Rebecca Jane
  • Tapley, Timothy Lewis
  • Tang, Qing
  • Thomson, Stephen
  • Xu, Jinwang
  • Zaky, Mariam
  • Cottrell, Kevin Michael

Abstract

The disclosure provides compounds useful for treating alpha-1 antitrypsin deficiency (AATD), according to formula (I): The disclosure provides compounds useful for treating alpha-1 antitrypsin deficiency (AATD), according to formula (I): The disclosure provides compounds useful for treating alpha-1 antitrypsin deficiency (AATD), according to formula (I): tautomers thereof, pharmaceutically acceptable salts of the compounds, pharmaceutically acceptable salts of the tautomers, deuterated derivatives of the compounds, deuterated derivatives of the tautomers, and deuterated derivatives of the salts, solid forms of those compounds and processes for making those compounds.

IPC Classes  ?

  • C07D 487/04 - Ortho-condensed systems
  • A61K 9/16 - AgglomeratesGranulatesMicrobeadlets
  • A61K 31/4162 - 1,2-Diazoles condensed with heterocyclic ring systems
  • C07D 491/18 - Bridged systems
  • C07D 513/04 - Ortho-condensed systems
  • C07D 519/00 - Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups or
  • C07F 9/6561 - Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings
  • C07H 15/26 - Acyclic or carbocyclic radicals, substituted by hetero rings

55.

ALYFTREK

      
Serial Number 98760764
Status Pending
Filing Date 2024-09-20
Owner Vertex Pharmaceuticals Incorporated ()
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceutical preparations for the treatment of cystic fibrosis

56.

Miscellaneous Design

      
Serial Number 98760768
Status Pending
Filing Date 2024-09-20
Owner Vertex Pharmaceuticals Incorporated ()
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceutical preparations for the treatment of cystic fibrosis

57.

HYDROXY AND (HALO)ALKOXY SUBSTITUTED TETRAHYDROFURANS AS MODULATORS OF SODIUM CHANNELS

      
Application Number 18566305
Status Pending
Filing Date 2022-06-03
First Publication Date 2024-09-05
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Beck, Elizabeth Mary
  • Pullin, Robert
  • Etxebarria Jardi, Gorka
  • Stamos, Dean
  • Schmidt, Yvonne
  • Pontillo, Joseph
  • Thomson, Stephen Andrew
  • Shaw, David Matthew
  • Ahmad, Nadia M.
  • Carvalho Meireles, Lidio Marx
  • Skerratt, Sarah
  • Hadida Ruah, Sara S.
  • Neubert, Timothy Donald
  • Hurley, Dennis James
  • Durrant, Steven John
  • Wray, Christopher
  • Virani, Anisa Nizarali
  • North, Kiri
  • Galan, Bhairavi
  • Knegtel, Ronald Marcellus
  • Chudyk, Ewa Iwona
  • Pinder, Joanne Louise
  • Sousa, Bruno Artur
  • Pierard, Francoise

Abstract

Compounds of formula I and pharmaceutically acceptable salts thereof, useful as inhibitors of sodium channels are provided. Also provided are pharmaceutical compositions comprising the compounds or pharmaceutically acceptable salts and methods of using the compounds, pharmaceutically acceptable salts, and pharmaceutical compositions in the treatment of various disorders, including pain. Compounds of formula I and pharmaceutically acceptable salts thereof, useful as inhibitors of sodium channels are provided. Also provided are pharmaceutical compositions comprising the compounds or pharmaceutically acceptable salts and methods of using the compounds, pharmaceutically acceptable salts, and pharmaceutical compositions in the treatment of various disorders, including pain.

IPC Classes  ?

  • C07D 405/12 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings linked by a chain containing hetero atoms as chain links
  • A61K 31/443 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with oxygen as a ring hetero atom
  • A61K 31/501 - PyridazinesHydrogenated pyridazines not condensed and containing further heterocyclic rings

58.

ISLET CELL MANUFACTURING COMPOSITIONS AND METHODS OF USE

      
Application Number 18581700
Status Pending
Filing Date 2024-02-20
First Publication Date 2024-09-05
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Thiel, Austin
  • Yasin, Jihad
  • Thompson, Evrett
  • Pagliuca, Felicia J.

Abstract

Disclosed herein are compositions and methods useful for manufacturing SC-β cell, and isolated populations of SC-β cells for use in various applications, such as cell therapy.

IPC Classes  ?

  • C07D 401/14 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
  • C07D 401/04 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings directly linked by a ring-member-to-ring- member bond
  • C07K 14/495 - Transforming growth factor [TGF]
  • C07K 14/50 - Fibroblast growth factor [FGF]
  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues

59.

STEM CELL DIFFERENTIATION AND POLYMERS

      
Application Number 18281154
Status Pending
Filing Date 2022-03-08
First Publication Date 2024-09-05
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Thompson, Evrett
  • Chinn, Rebecca
  • Raj, Suyash

Abstract

Disclosed herein are compositions and methods related to differentiation of stem cells into pancreatic cells. In some aspects, the methods provided herein relate to generation of pancreatic β cell, α cell, δ cells, and EC cells in vitro in the presence of a polymer, e.g., a water-soluble synthetic polymer. In some aspects, the disclosure provides pharmaceutical compositions including the cells generated according to the methods disclosed herein, as well as methods of treatment making use thereof.

IPC Classes  ?

  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues

60.

SOLID DOSAGE FORMS AND DOSING REGIMENS COMPRISING (2R,3S,4S,5R)-4-[[3-(3,4-DIFLUORO-2-METHOXY-PHENYL)-4,5-DIMETHYL-5-(TRIFLUOROMETHYL) TETRAHYDROFURAN-2-CARBONYL]AMINO]PYRIDINE-2-CARBOXAMIDE

      
Application Number 18566176
Status Pending
Filing Date 2022-06-03
First Publication Date 2024-08-29
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Karkare, Radhika
  • Chu, Cathy
  • Cirincione, Brenda
  • Correll, Darin J.
  • Delff, Philip Kaj Harder
  • Dinehart, Kirk Raymond
  • Benito Gallo, Paloma
  • Hay, Tanya Louise
  • Jiang, Licong
  • Jones, James B.
  • Mccarty, Katie L.
  • Metzler, Catherine P.
  • Miller, Jonathan M.
  • Peterson, Mark C.
  • Roopwani, Rahul
  • Staropoli, John F.

Abstract

Provided is a solid dispersion of (2R,3S,4S,5R)-4-[[3-(3,4-difluoro-2-methoxy-phenyl)-4,5-dimethyl-5-(trifluoromethyl) tetrahydrofuran-2-carbonyl]amino]pyridine-2-carboxamide (Compound 1), defined as described herein, or a pharmaceutically acceptable salt thereof and a tablet containing the solid dispersion for treating pain. Also disclosed herein is Compound 1 or a pharmaceutically acceptable salt thereof for use in a method of treating pain.

IPC Classes  ?

  • A61K 31/443 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with oxygen as a ring hetero atom
  • A61K 9/00 - Medicinal preparations characterised by special physical form
  • A61K 9/20 - Pills, lozenges or tablets
  • A61P 25/04 - Centrally acting analgesics, e.g. opioids

61.

HYPOIMMUNE CELLS

      
Application Number 18640564
Status Pending
Filing Date 2024-04-19
First Publication Date 2024-08-22
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Conway, Michael
  • Fulga, Tudor

Abstract

Disclosed herein are compositions and methods related to isolated cells (e.g., isolated stem cells) comprising a disruption in the 3′-UTR of an immunosuppressor, cells differentiated from such stem cells (e.g., pancreatic islet cells or immune cells) and methods of using the cells to treat diseases (e.g., diabetes or cancer). Methods of producing (i.e., genetically modifying) the isolated cells (e.g., isolated stem cells) are also provided.

IPC Classes  ?

  • A61K 35/545 - Embryonic stem cellsPluripotent stem cellsInduced pluripotent stem cellsUncharacterised stem cells
  • C07K 14/705 - ReceptorsCell surface antigensCell surface determinants
  • C12N 5/0735 - Embryonic stem cellsEmbryonic germ cells
  • C12N 9/22 - Ribonucleases
  • C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
  • C12N 15/90 - Stable introduction of foreign DNA into chromosome

62.

MACROENCAPSULATION DEVICES INCLUDING IMMUNOMODULATORY COMPOUNDS

      
Application Number US2024015452
Publication Number 2024/173283
Status In Force
Filing Date 2024-02-13
Publication Date 2024-08-22
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Thanos, Christopher
  • Song, Wei
  • Calaveiras Baltazar, Tania, Daniela
  • Nguyen, Thi, Hien, Luong
  • Hoffmann, Joseph, Charles
  • Furtado, Stacia

Abstract

Macroencapsulation devices and their methods of use are disclosed. In one embodiment, a macroencapsulation device may include one or more membrane layers configured to encapsulate a population of cells, and at least one immunomodulatory compound. The immunomodulatory compound may be contained in an immunomodulatory material including, for example, a matrix. The immunomodulatory material or compound, in certain cases, may mitigate undesirable foreign body responses during implantation of the device, gives rise to a low cytotoxicity, and/or the ability to promote cell viability.

IPC Classes  ?

  • A61K 9/48 - Preparations in capsules, e.g. of gelatin, of chocolate
  • A61K 35/39 - PancreasIslets of Langerhans
  • A61P 3/10 - Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
  • A61P 5/48 - Drugs for disorders of the endocrine system of the pancreatic hormones
  • A61P 29/00 - Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agentsNon-steroidal antiinflammatory drugs [NSAID]

63.

METHODS OF TREATING APOL-1 DEPENDENT FOCAL SEGMENTAL GLOMERULOSCLEROSIS

      
Application Number 18476131
Status Pending
Filing Date 2023-09-27
First Publication Date 2024-08-22
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Mallalieu, Navita
  • Egbuna, Ifeatu
  • Hare, Brian J.
  • Krug, Alexander Wolfgang
  • Wu, Shu-Pei

Abstract

This application describes methods of inhibiting APOL1 and treating APOL1-mediated diseases comprising administering Compound I and/or a pharmaceutically acceptable salt thereof. This application describes methods of inhibiting APOL1 and treating APOL1-mediated diseases comprising administering Compound I and/or a pharmaceutically acceptable salt thereof. This application describes methods of inhibiting APOL1 and treating APOL1-mediated diseases comprising administering Compound I and/or a pharmaceutically acceptable salt thereof. The application also describes pharmaceutical compositions comprising Compound I and/or a pharmaceutically acceptable salt thereof.

IPC Classes  ?

  • A61K 31/404 - Indoles, e.g. pindolol
  • A61K 9/00 - Medicinal preparations characterised by special physical form
  • A61K 9/20 - Pills, lozenges or tablets
  • A61K 31/573 - Compounds containing cyclopenta[a]hydrophenanthrene ring systemsDerivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
  • A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
  • A61P 13/12 - Drugs for disorders of the urinary system of the kidneys

64.

IN VITRO DIFFERENTIATION OF PANCREATIC ENDOCRINE CELLS

      
Application Number 18018787
Status Pending
Filing Date 2021-07-30
First Publication Date 2024-08-15
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Harb, George
  • Xie, Chunhui

Abstract

Disclosed herein are compositions and methods related to differentiation of stem cells into pancreatic endocrine cells. In some aspects, the methods provided herein relate to generation of pancreatic β cell, α cell, δ cells, and EC cells in vitro. In some aspects, the disclosure provides pharmaceutical compositions including the cells generated according to the methods disclosed herein, as well as methods of treatment making use thereof.

IPC Classes  ?

  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues

65.

BIARYL AMIDE-CONTAINING AGONISTS OF OREXIN RECEPTOR TYPE 2

      
Application Number US2024014749
Publication Number 2024/168004
Status In Force
Filing Date 2024-02-07
Publication Date 2024-08-15
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Volgraf, Matthew
  • Smith, Cameron Rees
  • Zhao, Liang
  • Larouche-Gauthier, Robin
  • Bouayad-Gervais, Samir
  • Ewing, Todd J.A.

Abstract

Disclosed are compounds, compositions, and methods useful for preventing or treating a disease which is at least partially mediated by orexin receptor 2.

IPC Classes  ?

  • C07D 207/12 - Oxygen or sulfur atoms
  • C07D 211/44 - Oxygen atoms attached in position 4
  • C07D 213/56 - Amides
  • C07D 213/61 - Halogen atoms or nitro radicals
  • C07D 295/135 - Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms with the ring nitrogen atoms and the substituent nitrogen atoms separated by carbocyclic rings or by carbon chains interrupted by carbocyclic rings
  • A61P 3/00 - Drugs for disorders of the metabolism
  • A61P 25/00 - Drugs for disorders of the nervous system
  • A61K 31/445 - Non-condensed piperidines, e.g. piperocaine
  • A61K 31/496 - Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
  • C07D 401/12 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings linked by a chain containing hetero atoms as chain links
  • C07D 401/14 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
  • C07D 413/12 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings linked by a chain containing hetero atoms as chain links
  • C07D 471/10 - Spiro-condensed systems
  • A61K 31/55 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
  • A61K 31/438 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom the ring being spiro-condensed with carbocyclic or heterocyclic ring systems
  • A61P 15/00 - Drugs for genital or sexual disordersContraceptives
  • A61P 27/00 - Drugs for disorders of the senses
  • A61P 35/00 - Antineoplastic agents

66.

Precise Excisions of Portions of Exon 51 for Treatment of Duchenne Muscular Dystrophy

      
Application Number 18598647
Status Pending
Filing Date 2024-03-07
First Publication Date 2024-08-01
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Fulga, Tudor
  • Xin, Yurong
  • Min, Yi-Li
  • Liu, Jianming
  • Bolukbasi, Mehmet Fatih

Abstract

Compositions and methods for treating Duchenne Muscular Dystrophy (DMD) and excising small portions of exon 51 of the DMD gene are encompassed.

IPC Classes  ?

  • A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
  • A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
  • C12N 9/22 - Ribonucleases
  • C12N 15/11 - DNA or RNA fragmentsModified forms thereof
  • C12N 15/86 - Viral vectors

67.

RE-AGGREGATION OF STEM CELL-DERIVED PANCREATIC BETA CELLS

      
Application Number 18391867
Status Pending
Filing Date 2023-12-21
First Publication Date 2024-07-18
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Poh, Yeh-Chuin
  • Harb, George
  • Pagliuca, Felicia J.

Abstract

The present application discloses cell clusters resembling the function and characteristics of endogenous pancreatic islets, and methods for making and using such cell clusters.

IPC Classes  ?

  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues
  • C12N 5/00 - Undifferentiated human, animal or plant cells, e.g. cell linesTissuesCultivation or maintenance thereofCulture media therefor

68.

Materials and Methods for Treatment of Hemoglobinopathies

      
Application Number 18612729
Status Pending
Filing Date 2024-03-21
First Publication Date 2024-07-11
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Cowan, Chad Albert
  • Lundberg, Ante Sven
  • Chakraborty, Tirtha
  • Lin, Michelle I-Ching
  • Mishra, Bibhu Prasad
  • Paik, Elizabeth
  • Kernytsky, Andrew
  • Borland, Todd Douglass

Abstract

Materials and methods for treating a patient with a hemoglobinopathy, both ex vivo and in vivo, and materials and methods for deleting modulating or inactivating a transcriptional control sequence of a BCL11A gene in a cell by genome editing.

IPC Classes  ?

  • A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
  • A61K 31/395 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
  • A61K 38/46 - Hydrolases (3)
  • A61P 7/00 - Drugs for disorders of the blood or the extracellular fluid
  • C12N 9/22 - Ribonucleases
  • C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
  • C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

69.

ZUNISLA

      
Application Number 1796285
Status Registered
Filing Date 2023-12-19
Registration Date 2023-12-19
Owner Vertex Pharmaceuticals Incorporated (USA)
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceutical preparations for treating diabetes; medical treatments in the nature of anti-diabetic pharmaceuticals; stem cell-derived cell therapy composed of cells for medical or veterinary use.

70.

PROCESS FOR THE SYNTHESIS OF SUBSTITUTED TETRAHYDROFURAN MODULATORS OF SODIUM CHANNELS

      
Application Number US2023082576
Publication Number 2024/123815
Status In Force
Filing Date 2023-12-05
Publication Date 2024-06-13
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Harrison, Cristian
  • Benito, Paloma, Gallo
  • Changi, Shujauddin, M.
  • Davis, Christopher, John
  • Everitt, Simon, Robert
  • Emer, Enrico
  • Gagnon, Kevin, J.
  • Lewandowski, Berenice, L.
  • Mctiernan, Andrew
  • Medek, Ales
  • O'Donnell, Michael, Edward
  • O'Neil, Simon, Adam
  • Panesar, Maninder
  • Sidera Portela, Mireia
  • Shanahan, Stephen, Edward
  • Sousa, Bruno, Artur
  • Stephens, David, E.
  • Wijethunga, Tharanga, K.

Abstract

Provided in this application is a process for making a compound of formula (I) and pharmaceutically acceptable salts thereof, which are useful as inhibitors of sodium channels. Also provided in this application is a process for making a compound of formula (II) and pharmaceutically acceptable salts thereof, which are useful as inhibitors of sodium channels. Processes for making various intermediate products, and suitable salts thereof, are also provided.

IPC Classes  ?

  • C07D 213/61 - Halogen atoms or nitro radicals
  • C07D 213/78 - Carbon atoms having three bonds to hetero atoms, with at the most one bond to halogen, e.g. ester or nitrile radicals
  • C07D 307/12 - Radicals substituted by oxygen atoms
  • C07D 307/20 - Oxygen atoms
  • C07D 307/32 - Oxygen atoms
  • C07D 405/02 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings
  • C07D 405/14 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing three or more hetero rings
  • A61P 29/00 - Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agentsNon-steroidal antiinflammatory drugs [NSAID]
  • A61K 31/443 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with oxygen as a ring hetero atom

71.

Compositions and Methods for Treatment of Myotonic Dystrophy Type 1 with CRISPR/SACAS9

      
Application Number 18456269
Status Pending
Filing Date 2023-08-25
First Publication Date 2024-06-06
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Ruan, Guoxiang
  • Liu, Jianming
  • Fulga, Tudor
  • Anderson, Eric Gunnar
  • Rao, Lingjun
  • Abdul-Manan, Norzehan
  • Heidenreich, Matthias
  • Dokshin, Gregoriy Aleksandrovich
  • Gromada, Jesper

Abstract

Compositions and methods for treating Myotonic Dystrophy Type 1 (DM1) are encompassed.

IPC Classes  ?

  • A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
  • C12N 9/22 - Ribonucleases
  • C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
  • C12N 15/86 - Viral vectors
  • C12N 15/90 - Stable introduction of foreign DNA into chromosome

72.

N-(hydroxyalkyl (hetero)aryl) tetrahydrofuran carboxamides as modulators of sodium channels

      
Application Number 18491021
Grant Number 12258333
Status In Force
Filing Date 2023-10-20
First Publication Date 2024-06-06
Grant Date 2025-03-25
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Beck, Elizabeth Mary
  • Durrant, Steven John
  • Skerratt, Sarah
  • Pullin, Robert
  • Etxebarria Jardi, Gorka
  • Shaw, David Matthew
  • Ahmad, Nadia M.
  • Wray, Christopher
  • Virani, Anisa Nizarali
  • North, Kiri
  • Dodd, James
  • O'Donnell, Michael Edward
  • Galan, Bhairavi
  • Knegtel, Ronald Marcellus
  • Chudyk, Ewa Iwona
  • Pinder, Joanne Louise
  • Thomson, Stephen Andrew
  • Carvalho Meireles, Lidio Marx
  • Stamos, Dean
  • Schmidt, Yvonne
  • Pontillo, Joseph
  • Hadida Ruah, Sara S.
  • Neubert, Timothy Donald
  • Hurley, Dennis James
  • Zhou, Jinglan

Abstract

Compounds, and pharmaceutically acceptable salts thereof, useful as inhibitors of sodium channels are provided. Also provided are pharmaceutical compositions comprising the compounds or pharmaceutically acceptable salts and methods of using the compounds, pharmaceutically acceptable salts, and pharmaceutical compositions in the treatment of various disorders, including pain.

IPC Classes  ?

  • C07D 405/12 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings linked by a chain containing hetero atoms as chain links

73.

Compositions and Methods for Treatment of Myotonic Dystrophy Type 1 with CRISPR/SluCas9

      
Application Number 18456288
Status Pending
Filing Date 2023-08-25
First Publication Date 2024-05-30
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Ruan, Guoxiang
  • Liu, Jianming
  • Fulga, Tudor
  • Bolukbasi, Mehmet Fatih
  • Anderson, Eric Gunnar
  • Rao, Lingjun
  • Abdul-Manan, Norzehan
  • Heidenreich, Matthias
  • Dokshin, Gregoriy Aleksandrovich
  • Gromada, Jesper

Abstract

Compositions and methods for treating Myotonic Dystrophy Type 1 (DM1) are encompassed.

IPC Classes  ?

  • A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
  • A61K 31/506 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
  • A61K 31/5377 - 1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
  • A61P 19/00 - Drugs for skeletal disorders
  • C12N 9/22 - Ribonucleases
  • C12N 15/11 - DNA or RNA fragmentsModified forms thereof

74.

SUBSTITUTED MACROCYCLIC AMINE MODULATORS OF OREXIN RECEPTOR 2

      
Application Number US2023080929
Publication Number 2024/112899
Status In Force
Filing Date 2023-11-22
Publication Date 2024-05-30
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Volgraf, Matthew
  • Zhao, Liang
  • Leblanc, Melissa
  • Larouche-Gauthier, Robin
  • Colwell, Curtis Eugene
  • Bouayad-Gervais, Samir
  • Ewing, Todd J.A.

Abstract

Disclosed are compounds, compositions, and methods useful for preventing or treating a disease which is at least partially mediated by orexin receptor 2.

IPC Classes  ?

75.

Processes for making modulators of cystic fibrosis transmembrane conductance regulator

      
Application Number 18211772
Grant Number 12024491
Status In Force
Filing Date 2023-06-20
First Publication Date 2024-05-23
Grant Date 2024-07-02
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Lemercier Lewandowski, Berenice
  • Lewis, Robert
  • Looker, Adam
  • Morgan, Adam
  • Roeper, Stefanie
  • Ryan, Michael
  • Solsona Rocabert, Juan Gabriel
  • Wilde, Nathan

Abstract

The disclosure provides processes for synthesizing compounds for use as CFTR modulators.

IPC Classes  ?

  • C07D 215/56 - Carbon atoms having three bonds to hetero atoms with at the most one bond to halogen attached in position 3 with oxygen atoms in position 4
  • C07B 59/00 - Introduction of isotopes of elements into organic compounds
  • C07C 39/06 - Alkylated phenols
  • C07C 69/96 - Esters of carbonic or haloformic acids
  • C07C 205/43 - Compounds containing nitro groups bound to a carbon skeleton the carbon skeleton being further substituted by esterified hydroxy groups having nitro groups or esterified hydroxy groups bound to carbon atoms of six-membered aromatic rings of the carbon skeleton to carbon atoms of the same non-condensed six-membered aromatic ring or to carbon atoms of six-membered aromatic rings being part of the same condensed ring system
  • C07C 219/34 - Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having amino groups and esterified hydroxy groups bound to carbon atoms of six-membered aromatic rings of the same carbon skeleton

76.

Pharmaceutical composition and administrations thereof

      
Application Number 18355475
Grant Number 12214083
Status In Force
Filing Date 2023-07-20
First Publication Date 2024-05-16
Grant Date 2025-02-04
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Dokou, Eleni
  • Jamzad, Shahla
  • Caesar, Jr., John P.
  • Fawaz, Majed
  • Das, Laura
  • Gu, Chong-Hui
  • Hurter, Patricia Nell
  • Israni, Meghna Jai
  • Johnston, Meghan M.
  • Knezic, Dragutin
  • Kuzmission, Andrew G.
  • Wang, Hongren

Abstract

The present invention relates to pharmaceutical compositions containing a solid dispersion of N-[2,4-Bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinoline-3-carboxamide including formulations of the solid dispersions into powders, granules and mini-tablets, methods for manufacturing and processing the powders, granules and mini-tablets, and methods for treating cystic fibrosis employing the pharmaceutical composition.

IPC Classes  ?

  • A61K 9/20 - Pills, lozenges or tablets
  • A61K 9/14 - Particulate form, e.g. powders
  • A61K 9/16 - AgglomeratesGranulatesMicrobeadlets
  • A61K 9/48 - Preparations in capsules, e.g. of gelatin, of chocolate
  • A61K 31/47 - QuinolinesIsoquinolines
  • A61K 47/02 - Inorganic compounds
  • A61K 47/12 - Carboxylic acidsSalts or anhydrides thereof
  • A61K 47/20 - Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing sulfur, e.g. dimethyl sulfoxide [DMSO], docusate, sodium lauryl sulfate or aminosulfonic acids
  • A61K 47/26 - Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharidesDerivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
  • A61K 47/38 - CelluloseDerivatives thereof
  • A61P 1/10 - Laxatives
  • A61P 1/16 - Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
  • A61P 1/18 - Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
  • A61P 3/00 - Drugs for disorders of the metabolism
  • C07D 215/56 - Carbon atoms having three bonds to hetero atoms with at the most one bond to halogen attached in position 3 with oxygen atoms in position 4

77.

MODULATORS OF ALPHA-1 ANTITRYPSIN

      
Application Number 18463796
Status Pending
Filing Date 2023-09-08
First Publication Date 2024-05-16
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Grey, Jr., Ronald Lee
  • Allen, Emily Elizabeth
  • Boyd, Michael J.
  • Fimognari, Jr., Robert F.
  • Giroux, Simon
  • Lai-Chen, Michelle
  • Medek, Ales
  • Li, Mengqi
  • Poff, Christopher David
  • Richter, Daniel Tyler
  • Scott, Tony Z.
  • Sokolowsky, Kathleen Paige
  • Sperry, Jeffrey Braden
  • Tsay, Charlene
  • Wang, Xiaoxu
  • Zaky, Mariam
  • Zhang, Chenlong

Abstract

Novel compounds, compositions, and methods of using and preparing the same, which may be useful for treating alpha-1 antitrypsin deficiency (AATD).

IPC Classes  ?

78.

GENETICALLY ENGINEERED CELLS

      
Application Number US2023078272
Publication Number 2024/097697
Status In Force
Filing Date 2023-10-31
Publication Date 2024-05-10
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Cooke, Michael
  • Conway, Michael
  • Kissler, Stephan
  • Carey, Bryce
  • Raj, Suyash

Abstract

Disclosed herein are compositions and methods related to genetically engineered mammalian cells comprising adjusted expression of select genes. The genetically engineered mammalian cells described herein advantageously possess improved survivability.

IPC Classes  ?

  • C12N 5/074 - Adult stem cells
  • C12N 5/0735 - Embryonic stem cellsEmbryonic germ cells
  • A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection
  • C12N 5/077 - Mesenchymal cells, e.g. bone cells, cartilage cells, marrow stromal cells, fat cells or muscle cells
  • C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
  • C12N 15/79 - Vectors or expression systems specially adapted for eukaryotic hosts

79.

CELL HOUSING DEVICE

      
Application Number 18415877
Status Pending
Filing Date 2024-01-18
First Publication Date 2024-05-09
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Thanos, Christopher
  • Lavin, Danya M.
  • Bintz, Briannan E.
  • Bhatnagar, Divya
  • Mills, John
  • Billings, Megan

Abstract

The present disclosure provides a cell housing device and a method of manufacturing such a device that has an array of channels to increase the ratio of surface area to volume.

IPC Classes  ?

  • A61K 9/00 - Medicinal preparations characterised by special physical form
  • A61K 35/39 - PancreasIslets of Langerhans
  • A61K 47/32 - Macromolecular compounds obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. carbomers
  • A61K 47/34 - Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers

80.

MODULATORS OF CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR

      
Application Number 18248071
Status Pending
Filing Date 2021-10-06
First Publication Date 2024-05-09
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Mccartney, Jason
  • Abela, Alexander Russell
  • Abraham, Sunny
  • Anderson, Corey Don
  • Arumugam, Vijayalaksmi
  • Chau, Jaclyn
  • Clemens, Jeremy
  • Cleveland, Thomas
  • Coon, Timothy Richard
  • Dwight, Timothy A.
  • Fanning, Lev Tyler Dewey
  • Frieman, Bryan A.
  • Grootenhuis, Peter
  • Gulevich, Anton V.
  • Hadida Ruah, Sara Sabina
  • Ishihara, Yoshihiro
  • Khatuya, Haripada
  • Krenitsky, Paul
  • Melillo, Vito
  • Miller, Mark Thomas
  • Paraselli, Prasuna
  • Pierre, Fabrice
  • Silina, Alina
  • Tran, Joe A.
  • Uy, Johnny
  • Valdez, Lino
  • Vickers, Troy
  • Zhou, Jinglan

Abstract

This disclosure provides modulators of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) having core structure (I), pharmaceutical compositions containing at least one such modulator, methods of treatment of CFTR mediated diseases, including cystic fibrosis, using such modulators and pharmaceutical compositions, combination pharmaceutical compositions and combination therapies employing those modulators, and processes and intermediates for making such modulators. This disclosure provides modulators of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) having core structure (I), pharmaceutical compositions containing at least one such modulator, methods of treatment of CFTR mediated diseases, including cystic fibrosis, using such modulators and pharmaceutical compositions, combination pharmaceutical compositions and combination therapies employing those modulators, and processes and intermediates for making such modulators.

IPC Classes  ?

81.

TALK ABOUT PAIN

      
Serial Number 98517101
Status Registered
Filing Date 2024-04-24
Registration Date 2025-01-21
Owner Vertex Pharmaceuticals Incorporated ()
NICE Classes  ? 44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services

Goods & Services

Providing health information to patients and caregivers; providing health care information; providing patient education support services relating to medications, cell therapy and genetic therapy, in the nature of providing information about healthcare; providing health information in the field of acute pain management

82.

TALK ABOUT PAIN

      
Serial Number 98517104
Status Registered
Filing Date 2024-04-24
Registration Date 2025-01-21
Owner Vertex Pharmaceuticals Incorporated ()
NICE Classes  ? 44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services

Goods & Services

Providing health information to patients and caregivers; providing health care information; providing patient education support services relating to medications, cell therapy and genetic therapy, in the nature of providing information about healthcare; providing health information in the field of acute pain management

83.

IZLETYDE

      
Application Number 1785449
Status Registered
Filing Date 2023-12-19
Registration Date 2023-12-19
Owner Vertex Pharmaceuticals Incorporated (USA)
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceuticals and medical treatments in the field of diabetes; cell therapy products for medical or veterinary use.

84.

ISLAKEY

      
Application Number 1785450
Status Registered
Filing Date 2023-12-19
Registration Date 2023-12-19
Owner Vertex Pharmaceuticals Incorporated (USA)
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceuticals and medical treatments in the field of diabetes; cell therapy products for medical or veterinary use.

85.

FACTOR B PROTEASES

      
Application Number 18219384
Status Pending
Filing Date 2023-07-07
First Publication Date 2024-04-11
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Popkov, Mikhail
  • Soros, Vanessa
  • Le Moan, Natacha
  • Blouse, Grant E.
  • Myles, Timothy

Abstract

Provided herein are engineered proteases of the S1A family that are specific for, and capable of, cleaving Factor B. Also provided herein are methods of making and using such engineered proteases. The engineered proteases provided herein may be useful for treating a disease or condition associated with dysregulation of the complement system by reducing complement activation through cleavage and inactivation of Factor B.

IPC Classes  ?

86.

RE-AGGREGATION OF STEM CELL-DERIVED PANCREATIC BETA CELLS

      
Application Number 18391831
Status Pending
Filing Date 2023-12-21
First Publication Date 2024-04-11
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Poh, Yeh-Chuin
  • Harb, George
  • Pagliuca, Felicia J.

Abstract

The present application discloses cell clusters resembling the function and characteristics of endogenous pancreatic islets, and methods for making and using such cell clusters.

IPC Classes  ?

  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues
  • C12N 5/00 - Undifferentiated human, animal or plant cells, e.g. cell linesTissuesCultivation or maintenance thereofCulture media therefor

87.

Differentiation of pancreatic endocrine cells

      
Application Number 18391799
Grant Number 12006516
Status In Force
Filing Date 2023-12-21
First Publication Date 2024-04-11
Grant Date 2024-06-11
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Harb, George
  • Xie, Chunhi

Abstract

Disclosed herein are compositions and methods related to differentiation of stem cells into pancreatic endocrine cells. In some aspects, the methods provided herein relate to generation of pancreatic β cell, α cell, δ cells, and EC cells in vitro. In some aspects, the disclosure provides pharmaceutical compositions including the cells generated according to the methods disclosed herein, as well as methods of treatment making use thereof.

IPC Classes  ?

  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues
  • A61K 35/39 - PancreasIslets of Langerhans

88.

ISLATRUVE

      
Application Number 1781914
Status Registered
Filing Date 2023-12-19
Registration Date 2023-12-19
Owner Vertex Pharmaceuticals Incorporated (USA)
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceuticals and medical treatments in the field of diabetes; cell therapy products for medical or veterinary use.

89.

ISLETRUVE

      
Application Number 1782100
Status Registered
Filing Date 2023-12-19
Registration Date 2023-12-19
Owner Vertex Pharmaceuticals Incorporated (USA)
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceuticals and medical treatment products in the field of diabetes; cell therapy products for medical or veterinary use.

90.

JOURNAVX

      
Application Number 1782411
Status Registered
Filing Date 2024-01-26
Registration Date 2024-01-26
Owner Vertex Pharmaceuticals Incorporated (USA)
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceuticals and medicines for the treatment of cystic fibrosis, pain, sickle cell disease, beta thalassemia, muscular dystrophy, diabetes, respiratory diseases, liver disease, kidney disease, alpha-1 antitrypsin deficiency and APOL1-mediated kidney disease.

91.

REKAYA

      
Application Number 1782412
Status Registered
Filing Date 2024-01-26
Registration Date 2024-01-26
Owner Vertex Pharmaceuticals Incorporated (USA)
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceuticals and medicines for the treatment of cystic fibrosis, pain, sickle cell disease, beta thalassemia, muscular dystrophy, diabetes, respiratory diseases, liver disease, kidney disease, alpha-1 antitrypsin deficiency and APOL1-mediated kidney disease.

92.

ISLATYD

      
Application Number 1782101
Status Registered
Filing Date 2023-12-19
Registration Date 2023-12-19
Owner Vertex Pharmaceuticals Incorporated (USA)
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceuticals and medical treatment products in the field of diabetes; cell therapy products for medical or veterinary use.

93.

VERTEX CONNEXION

      
Application Number 231752100
Status Pending
Filing Date 2024-03-25
Owner Vertex Pharmaceuticals Incorporated (USA)
NICE Classes  ?
  • 41 - Education, entertainment, sporting and cultural services
  • 42 - Scientific, technological and industrial services, research and design
  • 44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services

Goods & Services

(1) Educational services, namely, providing classes, seminars, and workshops in the fields of medications, cell therapy and genetic therapy (2) Providing medical and scientific research information in the field of cell therapy; providing pharmaceutical research and development in the field of cell therapy; Providing medical and scientific research information in the field of genetic therapy; providing pharmaceutical research and development in the field of genetic therapy; Providing online non-downloadable software featuring a portal for order management and maintaining chain of identity and chain of custody of cell therapy and genetic therapy products and medicinal applications (3) Providing health information to patients, caregivers and health care professionals; providing health care information and patient education support services relating to medications, cell therapy and genetic therapy

94.

MODULATORS OF ATP-BINDING CASSETTE TRANSPORTERS

      
Application Number 18108053
Status Pending
Filing Date 2023-02-10
First Publication Date 2024-03-21
Owner Vertex Pharmaceuticals Incorporated (USA)
Inventor
  • Hadida Ruah, Sara S.
  • Grootenhuis, Peter D.J.
  • Van Goor, Fredrick
  • Zhou, Jinglan
  • Bear, Brian
  • Miller, Mark T.
  • Mccartney, Jason
  • Numa, Mehdi Michel Jamel
  • Yang, Xiaoqing

Abstract

Compounds of the present invention and pharmaceutically acceptable compositions thereof, are useful as modulators of ATP-Binding Cassette (“ABC”) transporters or fragments thereof, including Cystic Fibrosis Transmembrane Conductance Regulator (“CFTR”). The present invention also relates to methods of treating ABC transporter mediated diseases using compounds of the present invention.

IPC Classes  ?

  • C07D 405/12 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings linked by a chain containing hetero atoms as chain links
  • C07D 471/04 - Ortho-condensed systems
  • A61K 31/445 - Non-condensed piperidines, e.g. piperocaine
  • A61K 31/496 - Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
  • A61K 31/5377 - 1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
  • C07D 405/14 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing three or more hetero rings
  • C07D 403/12 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group containing two hetero rings linked by a chain containing hetero atoms as chain links
  • G01N 33/50 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing
  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids
  • C07B 59/00 - Introduction of isotopes of elements into organic compounds
  • C07D 487/04 - Ortho-condensed systems

95.

ENHANCED DIFFERENTIATION OF PANCREATIC ISLET CELLS

      
Application Number US2023074279
Publication Number 2024/059776
Status In Force
Filing Date 2023-09-15
Publication Date 2024-03-21
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Xie, Chunhui
  • Harb, George
  • Ryu, Elizabeth
  • Teceno, Nicholas
  • Farrar, Christopher
  • Carey, Bryce W.

Abstract

Disclosed herein are compositions and methods related to differentiation of stem cells into pancreatic islet cells. In some aspects, the methods provided herein relate to generation of pancreatic β cell, α cell, δ cells, and EC cells in vitro. In some aspects, the disclosure provides pharmaceutical compositions including the cells generated according to the methods disclosed herein, as well as methods of treatment making use thereof.

IPC Classes  ?

96.

MODULATORS OF ALPHA-1 ANTITRYPSIN

      
Application Number US2023032282
Publication Number 2024/054624
Status In Force
Filing Date 2023-09-08
Publication Date 2024-03-14
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Allen, Emily Elizabeth
  • Boyd, Michael J.
  • Fimognari, Jr., Robert F.
  • Giroux, Simon
  • Grey, Jr., Ronald Lee
  • Lai-Chen, Michelle
  • Medek, Ales
  • Li, Mengqi
  • Poff, Christopher David
  • Richter, Daniel Tyler
  • Scott, Tony Z.
  • Sokolowsky, Kathleen Paige
  • Sperry, Jeffrey Braden
  • Tsay, Charlene
  • Wang, Xiaoxu
  • Zaky, Mariam
  • Zhang, Chenlong

Abstract

Novel compounds, compositions, and methods of using and preparing the same, which may be useful for treating alpha-1 antitrypsin deficiency (AATD). (Formula (I))

IPC Classes  ?

  • C07D 405/04 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings directly linked by a ring-member-to-ring- member bond
  • C07D 487/04 - Ortho-condensed systems
  • A61P 11/00 - Drugs for disorders of the respiratory system
  • A61K 31/4162 - 1,2-Diazoles condensed with heterocyclic ring systems
  • A61P 1/16 - Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics

97.

MATERIALS AND METHODS FOR TREATMENT OF PAIN RELATED DISORDERS

      
Application Number 18466575
Status Pending
Filing Date 2023-09-13
First Publication Date 2024-02-29
Owner Vertex Phamaceuticals Incorporated (USA)
Inventor
  • Lundberg, Ante Sven
  • Kulkarni, Samarth
  • Klein, Lawrence
  • Padmanabhan, Hari Kumar

Abstract

The present application provides materials and methods for treating a patient with one or more conditions associated with SCN10A whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of SCN10A gene in a cell by genome editing.

IPC Classes  ?

  • A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
  • A61K 9/00 - Medicinal preparations characterised by special physical form
  • A61K 9/127 - Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
  • A61K 9/51 - Nanocapsules
  • A61K 35/30 - NervesBrainEyesCorneal cellsCerebrospinal fluidNeuronal stem cellsNeuronal precursor cellsGlial cellsOligodendrocytesSchwann cellsAstrogliaAstrocytesChoroid plexusSpinal cord tissue
  • A61P 43/00 - Drugs for specific purposes, not provided for in groups
  • C07K 14/705 - ReceptorsCell surface antigensCell surface determinants
  • C12N 5/0775 - Mesenchymal stem cellsAdipose-tissue derived stem cells
  • C12N 5/0793 - Neurons
  • C12N 9/22 - Ribonucleases
  • C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
  • C12N 15/90 - Stable introduction of foreign DNA into chromosome

98.

ALYFTREK

      
Application Number 1775604
Status Registered
Filing Date 2024-01-17
Registration Date 2024-01-17
Owner Vertex Pharmaceuticals Incorporated (USA)
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

Pharmaceutical preparations for the treatment of cystic fibrosis.

99.

COMPOSITIONS FOR THE TREATMENT OF CFTR-MEDIATED DISEASES

      
Application Number US2023071721
Publication Number 2024/031081
Status In Force
Filing Date 2023-08-04
Publication Date 2024-02-08
Owner VERTEX PHARMACEUTICALS INCORPORATED (USA)
Inventor
  • Dinehart, Kirk
  • Tips, Lauren, Bailey
  • Metzler, Catherine
  • Mccarty, Katie

Abstract

RNN-[2,4-Bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinoline-3-carboxamide. including formulations of the solid dispersions into powders, granules and mini-tablets, methods for manufacturing and processing the powders, granules and mini-tablets, and methods for treating cystic fibrosis employing the pharmaceutical compositions.

IPC Classes  ?

  • A61K 9/16 - AgglomeratesGranulatesMicrobeadlets
  • A61K 9/14 - Particulate form, e.g. powders
  • A61K 9/20 - Pills, lozenges or tablets
  • A61K 31/404 - Indoles, e.g. pindolol
  • A61K 31/4439 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
  • A61K 31/47 - QuinolinesIsoquinolines
  • A61P 11/00 - Drugs for disorders of the respiratory system

100.

REKAYA

      
Application Number 231836400
Status Pending
Filing Date 2024-01-26
Owner Vertex Pharmaceuticals Incorporated (USA)
NICE Classes  ? 05 - Pharmaceutical, veterinary and sanitary products

Goods & Services

(1) Pharmaceuticals and medicines for the treatment of cystic fibrosis, pain, sickle cell disease, beta thalassemia, muscular dystrophy, diabetes, respiratory diseases, liver disease, kidney disease, alpha-1 antitrypsin deficiency and APOL1-mediated kidney disease.
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