The disclosure relates to a compound of formula (I)
The disclosure relates to a compound of formula (I)
wherein X1, X2, and X3 are independently selected from CR6 or N; R1 represents a 5 or 6-membered heteroaryl group, wherein said heteroaryl is optionally substituted; R2 represents a 4-, 5- or 6-membered heteroaryl in which 1 to 3 ring atoms are independently selected from nitrogen, oxygen or sulfur, a 5 or 6-membered heterocycle in which 1 to 3 ring atoms are independently selected from nitrogen and oxygen or a spiro(C7-C10)heterobicyclic; R3 represents H or a (C1-C4)alkyl group; R4 represents H or a (C1-C4)alkyl group; or alternatively R3 and R4 together form with the carbon atom to which they are attached a cyclopropyl or a cyclobutyl ring; R5 and R6 independently represents H, a (C1-C6)alkyl group or a halogen; and/or a pharmaceutically acceptable salt, solvate, or stereoisomer thereof.
The disclosure relates to a compound of formula (I)
wherein X1, X2, and X3 are independently selected from CR6 or N; R1 represents a 5 or 6-membered heteroaryl group, wherein said heteroaryl is optionally substituted; R2 represents a 4-, 5- or 6-membered heteroaryl in which 1 to 3 ring atoms are independently selected from nitrogen, oxygen or sulfur, a 5 or 6-membered heterocycle in which 1 to 3 ring atoms are independently selected from nitrogen and oxygen or a spiro(C7-C10)heterobicyclic; R3 represents H or a (C1-C4)alkyl group; R4 represents H or a (C1-C4)alkyl group; or alternatively R3 and R4 together form with the carbon atom to which they are attached a cyclopropyl or a cyclobutyl ring; R5 and R6 independently represents H, a (C1-C6)alkyl group or a halogen; and/or a pharmaceutically acceptable salt, solvate, or stereoisomer thereof.
It further relates to the pharmaceutical compositions containing said new compounds.
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 31/506 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
A61K 31/53 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with three nitrogens as the only ring hetero atoms, e.g. chlorazanil, melamine
C07D 417/14 - 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 three or more hetero rings
2.
CHIMERIC ANTIGEN RECEPTOR (CAR) COMPRISING AN ANTI-CD19 VHH AS TARGETING DOMAIN
The present disclosure relates generally to compositions and methods for in vivo gene therapy, and more specifically to delivering mRNA encoding anti-CD19 chimeric antigen receptors (CARs) to immune cells (e.g., T cells, such as CD8+ T cells) in vivo.
A61K 40/11 - T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cellsLymphokine-activated killer [LAK] cells
A61K 9/1272 - Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61P 35/02 - Antineoplastic agents specific for leukemia
A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection
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
C07K 17/06 - Peptides being immobilised on, or in, an organic carrier attached to the carrier via a bridging agent
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
A61K 31/553 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole having at least one nitrogen and at least one oxygen as ring hetero atoms, e.g. loxapine, staurosporine
A61K 31/4745 - QuinolinesIsoquinolines ortho- or peri-condensed with heterocyclic ring systems condensed with ring systems having nitrogen as a ring hetero atom, e.g. phenanthrolines
Provided are ionizable cationic lipids and lipid nanoparticles for the delivery of nucleic acids to cells (e.g., HSC), and methods of making and using such lipids and targeted lipid. nanoparticles.
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 47/68 - 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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
The present disclosure relates generally to compositions and methods for in vivo gene therapy, and more specifically to delivering mRNA encoding anti-CD19 chimeric antigen receptors (CARs) to immune cells (e.g., T cells, such as CD8+ T cells) in vivo.
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
A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
This disclosure relates to engineered adeno-associated virus (AAV) capsid proteins and related nucleic acids, recombinant AAVs, vectors, cells, and methods of using the same.
B01D 15/16 - Selective adsorption, e.g. chromatography characterised by constructional or operational features relating to the conditioning of the fluid carrier
B01D 15/36 - Selective adsorption, e.g. chromatography characterised by the separation mechanism involving ionic interaction, e.g. ion-exchange, ion-pair, ion-suppression or ion-exclusion
B01D 15/38 - Selective adsorption, e.g. chromatography characterised by the separation mechanism involving specific interaction not covered by one or more of groups , e.g. affinity, ligand exchange or chiral chromatography
B01D 15/42 - Selective adsorption, e.g. chromatography characterised by the development mode, e.g. by displacement or by elution
B01J 20/281 - Sorbents specially adapted for preparative, analytical or investigative chromatography
B01J 41/05 - Processes using organic exchangers in the strongly basic form
B01J 41/20 - Anion exchangers for chromatographic processes
B01J 47/014 - Ion-exchange processes in generalApparatus therefor in which the adsorbent properties of the ion-exchanger are involved, e.g. recovery of proteins or other high-molecular compounds
B01J 49/57 - Regeneration or reactivation of ion-exchangersApparatus therefor characterised by the regeneration reagents for anionic exchangers
C07K 14/005 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from viruses
C12N 7/00 - Viruses, e.g. bacteriophagesCompositions thereofPreparation or purification thereof
e.ge.g., rAAV vectors), viral particles, pharmaceutical compositions and kits for expressing a disorder-related polypeptide in an individual in need thereof.
The present disclosure provides a non-human primate model for geographic atrophy related to macular degeneration such as age-related macular degeneration, methods of generating the model, and methods of using the model to screen therapeutic compounds useful in treating macular degeneration, for example, by slowing progression of geographic atrophy.
The present application provides methods of treating late-onset Pompe disease (LOPD) using a pharmaceutical composition comprising an oligosaccharide-acid α-glucosidase (GAA) conjugate, such as avalglucosidase alfa.
A61K 38/47 - Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
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 47/54 - 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 compound
A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
A medicament delivery device is described. The device includes a body having proximal and distal ends defining a longitudinal direction; a needle for injecting medicament into a user and configured to be arranged in an injecting position in which the needle protrudes from the distal end of the body; a cap moveable along the longitudinal direction to be arrangeable in a capped position in which the cap conceals the distal end of the body, and an uncapped position in which at least a portion of the cap is spaced apart from the distal end; and a blocking element. When the cap is in the uncapped position, the blocking element is arrangeable in a first state in which movement of the cap from the uncapped position into the capped position is permitted, and a second state in which movement of the cap from the uncapped position into the capped position is prevented.
This specification relates to systems and methods for identifying locations of potential patients and/or healthcare providers from geofencing data using machine-learned models. According to a first aspect of this specification, there is described a computer implemented method of identifying potential disease diagnoses and/or healthcare providers. The method comprises: receiving geofencing data from one or more healthcare locations, the geofencing data relating to the disease comprising one or more impressions made at the healthcare location and/or one or more clicks made at the healthcare location; extracting, for each of a plurality of entities, a plurality of features from the geofencing data, wherein each entity is associated with a respective healthcare location in the one or more healthcare locations.
G16H 50/20 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for computer-aided diagnosis, e.g. based on medical expert systems
The present technology relates to a novel cleaning method for chromatography media and/or supporting equipment comprising treatment with a novel cleaning solution comprising sodium hydroxide and hexylene glycol.
B01D 15/20 - Selective adsorption, e.g. chromatography characterised by constructional or operational features relating to the conditioning of the sorbent material
B01D 15/32 - Bonded phase chromatography, e.g. with normal bonded phase, reversed phase or hydrophobic interaction
The present technology relates to a novel sanitization method for chromatography media and supporting equipment comprising treatment with a sanitization/sterilization solution comprising acetic acid and hexylene glycol.
Provided are lipid-based systems, compositions, and methods of making and using the same. The lipid-based systems contain lipid nanoparticles and specifically formulated liposomes to reduce or prevent lipid nanoparticle clearance in vivo in subjects towards LNPs or components thereof.
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
18.
AAV GENOMES ENCODING ENGINEERED HUMAN FRATAXIN TRANSGENE
Provided herein are expression cassettes for expressing a transgene in a cell, wherein the transgene encodes a disorder-related polypeptide. Also provided are methods to treat Friedreich's ataxia. Further provided herein are vectors (e.g., rAAV vectors), viral particles, pharmaceutical compositions and kits for expressing a disorder-related polypeptide in an individual in need thereof.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
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
The invention provides ionizable cationic lipids and lipid nanoparticles for the delivery of nucleic acids to target cells, such as immune cells and hematopoietic stem cells, and methods of making and using, such lipids and targeted lipid nanoparticles.
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 47/68 - 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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
C07D 317/30 - Radicals substituted by carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals
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 409/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms containing three or more hetero rings
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Pharmaceutical preparations for the prevention and treatment
of cardiovascular diseases and central nervous system
diseases; pharmaceutical preparations for treatment of
cancer, thrombosis and respiratory diseases; pharmaceutical
preparations for the treatment of rare diseases,
particularly lysosomal storage disorders and autosomal
dominant polycystic kidney disease; pharmaceutical
preparations for the prevention and treatment of infectious
diseases, namely, bacterial and viral infections.
21.
HOLD ASSISTANCE DEVICE FOR USE WITH A MEDICAMENT DELIVERY DEVICE
A hold assistance device for use with a medicament delivery device is described. The hold assistance device includes a body that includes: a receiving volume for receiving a medicament delivery device; a proximal portion comprising a proximal end of the body; a distal portion comprising a distal end of the body, the proximal and distal ends defining an axial direction; and a connecting portion arranged between the proximal and distal portions, wherein the connecting portion is configured to flex into the receiving volume in a direction generally normal to the axial direction, causing the proximal portion to move axially relative to the distal portion.
A61M 5/20 - Automatic syringes, e.g. with automatically actuated piston rod, with automatic needle injection, filling automatically
A61M 5/32 - NeedlesDetails of needles pertaining to their connection with syringe or hubAccessories for bringing the needle into, or holding the needle on, the bodyDevices for protection of needles
22.
HOLD ASSISTANCE DEVICE FOR USE WITH A MEDICAMENT DELIVERY DEVICE
A hold assistance device for use with a medicament delivery device is described. The hold assistance device includes: a housing configured to be fixedly coupled to a medicament delivery device, the housing comprising: a proximal end and a distal end defining an axial direction, and an inclined surface angled relative to the axial direction; a blocking element moveable along the inclined surface; and an actuation element moveable relative to the housing and arrangeable in an activated position in which the actuation element applies an activation force to the blocking element in a direction towards the distal end, such that movement of the actuation element into the activated position causes the blocking element to move against the inclined surface towards the distal end and to exert a clamping force generally normal to the axial direction.
A61M 5/20 - Automatic syringes, e.g. with automatically actuated piston rod, with automatic needle injection, filling automatically
A61M 5/32 - NeedlesDetails of needles pertaining to their connection with syringe or hubAccessories for bringing the needle into, or holding the needle on, the bodyDevices for protection of needles
23.
HOLD ASSISTANCE DEVICE FOR USE WITH A MEDICAMENT DELIVERY DEVICE
A hold assistance device for use with a medicament delivery device is described. The hold assistance device includes: a housing configured to be coupled to a medicament delivery device; a chamber for containing air, the chamber having a proximal end and a distal end; and a piston movable within the chamber between the proximal and distal ends of the chamber, wherein the piston is arrangeable in an activated position inside the chamber in which the piston is arranged proximate to the proximal end of the chamber such that the chamber is at least partially evacuated of air and movement of the piston towards the distal end of the chamber is resisted by a vacuum force.
A61M 5/20 - Automatic syringes, e.g. with automatically actuated piston rod, with automatic needle injection, filling automatically
A61M 5/315 - PistonsPiston-rodsGuiding, blocking or restricting the movement of the rodAppliances on the rod for facilitating dosing
A61M 5/32 - NeedlesDetails of needles pertaining to their connection with syringe or hubAccessories for bringing the needle into, or holding the needle on, the bodyDevices for protection of needles
24.
HOLD ASSISTANCE DEVICE FOR USE WITH A MEDICAMENT DELIVERY DEVICE
A hold assistance device for use with a medicament delivery device including a moveable needle cover. The hold assistance device comprises a housing coupled to a medicament delivery device, the housing comprising a proximal end and a distal end defining an axis between the proximal and distal ends; a holding element coupled to the housing and moveable relative to the housing in a direction substantially normal to the axis of the housing between a disengaged position and an engaged position; wherein the holding element is disposed outwardly from the axis in the disengaged position and is disposed inwardly towards the axis in the engaged position configured in the engaged position such that the holding element can engage with an aperture or recess in a needle cover of a medicament delivery device when the hold assistance device is coupled thereto, to resist movement of the needle cover relative to the housing.
A61M 5/32 - NeedlesDetails of needles pertaining to their connection with syringe or hubAccessories for bringing the needle into, or holding the needle on, the bodyDevices for protection of needles
A61M 5/20 - Automatic syringes, e.g. with automatically actuated piston rod, with automatic needle injection, filling automatically
A medicament delivery device for injecting a medicament is described. The medicament delivery device comprises a housing having a proximal end and a distal end, the housing being configured to receive a medicament container. A plunger is moveable in a longitudinal direction within the housing for dispensing medicament from the medicament container when the medicament container is received within the housing. A plunger drive magnet is operatively coupled to the housing, and the plunger drive magnet is configured to provide a magnetic field. A component is configured to magnetically interact with the magnetic field of the plunger drive magnet so as to cause a force to be exerted on the plunger for causing the plunger to move in the longitudinal direction.
A61M 5/32 - NeedlesDetails of needles pertaining to their connection with syringe or hubAccessories for bringing the needle into, or holding the needle on, the bodyDevices for protection of needles
A61M 5/20 - Automatic syringes, e.g. with automatically actuated piston rod, with automatic needle injection, filling automatically
A61M 5/315 - PistonsPiston-rodsGuiding, blocking or restricting the movement of the rodAppliances on the rod for facilitating dosing
A hold assistance device for use with a medicament delivery device is described. The hold assistance device includes: a first support configured to be fixedly coupled to a main body of a medicament delivery device, wherein the first support comprises a first attraction component; and a second support moveable relative to the first support between an initial position and a hold position and configured to be fixedly coupled to a needle cover of the medicament delivery device, wherein the second support includes a second attraction component, and wherein at least one of the first attraction component and the second attraction component is a magnet, and the other of the first attraction component and the second attraction component is a magnet or a ferromagnetic material; and wherein movement of the second support towards the hold position moves the second attraction component towards the first attraction component.
A61M 5/32 - NeedlesDetails of needles pertaining to their connection with syringe or hubAccessories for bringing the needle into, or holding the needle on, the bodyDevices for protection of needles
A61M 5/20 - Automatic syringes, e.g. with automatically actuated piston rod, with automatic needle injection, filling automatically
A61M 5/24 - Ampoule syringes, i.e. syringes with needle for use in combination with replaceable ampoules or cartridges, e.g. automatic
27.
CAPTURE PROBE, FAMILIAL CHYLOMICRONEMIA SYNDROME DETECTION KIT CONTAINING THE SAME, AND USE THEREOF
The present application relates to the field of biomedical detection, and particularly to a capture probe, a familial chylomicronemia syndrome detection kit containing the same, and use thereof. The present application aims to detect FCS-related gene mutations stably and accurately, and to provide a more convenient and accurate FCS detection method for clinical practice. It is of great significance for the clinical diagnosis and treatment of FCS.
The disclosure provides methods of purifying adeno-associated virus (AAV) particles by introducing a flocculant step into a large scale a manufacturing process, thereby improving AAV particle purity and yield.
Compositions and methods for treating macular degeneration are disclosed. The methods utilize gene delivery to human eyes of soluble Flt-1 receptors, as well fusion proteins including a soluble Flt-1 receptor.
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
C07K 14/71 - ReceptorsCell surface antigensCell surface determinants for growth factorsReceptorsCell surface antigensCell surface determinants for growth regulators
H02J 7/02 - Circuit arrangements for charging or depolarising batteries or for supplying loads from batteries for charging batteries from AC mains by converters
The present disclosure provides methods and systems for modeling, simulating, and treating hemophilia. The methods and systems disclosed herein include a quantitative systems pharmacology (QSP) model to predict features of thrombin generation in people with hemophila A and people with hemophilia B in the context of antithrombin (AT) lowering.
G16B 5/00 - ICT specially adapted for modelling or simulations in systems biology, e.g. gene-regulatory networks, protein interaction networks or metabolic networks
The invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, and for the treatment of cancer.
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 31/454 - 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
A61K 31/46 - 8-Azabicyclo [3.2.1] octaneDerivatives thereof, e.g. atropine, cocaine
A61K 31/506 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
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 38/47 - Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
C07D 453/00 - Heterocyclic compounds containing quinuclidine or iso-quinuclidine ring systems, e.g. quinine alkaloids
C07D 453/02 - Heterocyclic compounds containing quinuclidine or iso-quinuclidine ring systems, e.g. quinine alkaloids containing not further condensed quinuclidine ring systems
C07D 455/02 - Heterocyclic compounds containing quinolizine ring systems, e.g. emetine alkaloids, protoberberineAlkylenedioxy derivatives of dibenzo [a, g] quinolizines, e.g. berberine containing not further condensed quinolizine ring systems
The present disclosure provides methods of producing in a single cell a mixed population of recombinant adeno-associated viruses for expressing two or more transgenes and methods of producing recombinant adeno-associated viruses containing self-complementary genomes. Also provided are methods of treating diseases using the rAAV compositions produced herein.
The disclosure relates to compound of formula (I)
The disclosure relates to compound of formula (I)
wherein R1 represents a phenyl or a monocyclic heteroaryl, which is optionally substituted by one, two or three R3; R2 represents an aryl or a heteroaryl, which is optionally substituted by one, two or three R6; each R3 is independently chosen from a halogen, a cyano, a (C1-C4)alkyl group or a (C1-C4)alkoxy group; R4 and R5 are independently chosen from a halogen, a (C1-C4)alkyl group, a (C1-C4)alkoxy group, or R4 and R5 form together a (C1-C4)alkylene bridge; m and s are independently 0 or 1; p, q, r and t are independently 0 or 1; each R6 is independently selected from halogen, cyano, a —NH2 group, —OH, a (C1-C4)alkyl group, —CF3, —C(O)NH2, a —C(O)NH—(C1-C4)alkyl group, —C(O)OH, a —C(O)O—(C1-C4)alkyl group, —SO2NH2, a (C1-C4)alkoxy group, a —O—(C1-C4)alkylene-(C3-C6)cycloalkyl group, a —O—(C3-C6)cycloalkyl group, a —O—(C3-C6)heterocycloalkyl group, a (C3-C6)heterocycloalkyl group, a heteroaryl group and oxo; or a pharmaceutically acceptable salt, solvate, or stereoisomer thereof.
The disclosure relates to compound of formula (I)
wherein R1 represents a phenyl or a monocyclic heteroaryl, which is optionally substituted by one, two or three R3; R2 represents an aryl or a heteroaryl, which is optionally substituted by one, two or three R6; each R3 is independently chosen from a halogen, a cyano, a (C1-C4)alkyl group or a (C1-C4)alkoxy group; R4 and R5 are independently chosen from a halogen, a (C1-C4)alkyl group, a (C1-C4)alkoxy group, or R4 and R5 form together a (C1-C4)alkylene bridge; m and s are independently 0 or 1; p, q, r and t are independently 0 or 1; each R6 is independently selected from halogen, cyano, a —NH2 group, —OH, a (C1-C4)alkyl group, —CF3, —C(O)NH2, a —C(O)NH—(C1-C4)alkyl group, —C(O)OH, a —C(O)O—(C1-C4)alkyl group, —SO2NH2, a (C1-C4)alkoxy group, a —O—(C1-C4)alkylene-(C3-C6)cycloalkyl group, a —O—(C3-C6)cycloalkyl group, a —O—(C3-C6)heterocycloalkyl group, a (C3-C6)heterocycloalkyl group, a heteroaryl group and oxo; or a pharmaceutically acceptable salt, solvate, or stereoisomer thereof.
It further relates to the pharmaceutical compositions containing said new compounds.
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 31/422 - Oxazoles not condensed and containing further heterocyclic rings
A61K 31/427 - Thiazoles not condensed and containing further heterocyclic rings
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 31/444 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring hetero atom, e.g. amrinone
A61K 31/497 - Non-condensed pyrazines containing further heterocyclic rings
A61K 31/506 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
C07D 261/02 - Heterocyclic compounds containing 1,2-oxazole or hydrogenated 1,2-oxazole rings not condensed with other rings
C07D 413/08 - 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 carbon chain containing alicyclic 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
C07D 417/14 - 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 three or more hetero rings
A fluid transfer device comprises a body comprising a first connecting portion comprising a first fluid channel to fluidically communicate with an interior of a diluent container, a second connecting portion comprising a second fluid channel to fluidically communicate with an interior of a medicament container, a third connecting portion comprising a third fluid channel to fluidically communicate with an interior of a pooling container, and a fourth fluid channel to fluidically communicate with a pump device. A movable part is engaged with and movable relative to the body to transfer the fluid transfer device into one of a first configuration, a second configuration and a third configuration. When in the first configuration, the second fluid channel is fluidically coupled to the third fluid channel, and when in the second configuration, the first fluid channel is fluidically connectable to the third fluid channel.
A medicament delivery device comprises a syringe body configured to receive a medicament. The syringe body comprises a distal end and a proximal end, a plunger located in and moveable relative to the syringe body, and a plunger rod assembly extending from the proximal end of the syringe body and configured to move the plunger longitudinally within the syringe body. The plunger rod assembly comprises a plunger barrel and a plunger rod. The plunger barrel contacts the plunger and is configured to cooperate with the plunger rod, which is moveable relative to the plunger barrel from a first position, in which the plunger rod is maximally extended from the plunger barrel, to a second position, in which the plunger rod is in a distal position relative to the first position, to set a specific dose of medicament to be delivered less than the amount of medicament in the syringe body.
Methods, systems, and apparatus, including computer programs encoded on a computer storage medium, for determining whether to select a patient, e.g., to receive a drug that targets a target gene. In one aspect, a method comprises: obtaining gene expression data for a collection of cells from a patient; processing a model input comprising the gene expression data using a machine learning model, in accordance with values of a set of machine learning model parameters, to generate a predicted gene essentiality score for the target gene; and determining whether to select the patient based at least in part on the predicted gene essentiality score for the target gene.
G16H 20/10 - ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance relating to drugs or medications, e.g. for ensuring correct administration to patients
38.
AN APPARATUS FOR FILLING A MEDICAMENT PREPARATION VIAL
An apparatus for filling a medicament preparation vial with a fixed volume of diluent from a storage container comprises a container, an inlet comprising an inlet spike, and an outlet comprising an outlet spike. An inlet valve located downstream of the inlet spike has a first state allowing fluid communication between the inlet and the chamber, and a second state in preventing fluid communication between the inlet and the chamber. An outlet valve located upstream of the outlet spike has a first state preventing fluid communication between the chamber and the outlet, and a second state allowing fluid communication between the chamber and the outlet. The outlet valve is in the first state when the inlet valve is in the first state, and is moveable from the first state to the second state when the inlet valve is in the first state.
A medical device for use in the preparation of a medicament comprising a tray movable from a first configuration to a second configuration and comprising a first tray portion and a second tray portion. The first tray portion comprises a first retainer to retain a first medicament vial and the second tray portion comprises a second retainer configured to retain a second medicament vial. The first tray portion is pivotable relative to the second tray portion to move the tray from the first configuration to the second configuration. The tray is configured such that the first medicament constituent and the second medicament constituent can be accessed by a user.
A method and medical device (200, 500, 600, 700, 800, 900) for mixing a medicament (106) contained within a medicament vial (210), the medical device comprising a track (220) configured to support the medicament vial, wherein the track comprises an inclined first track section (241) arranged such that, in use, the medicament vial can roll along the track to mix the medicament contained within the medicament vial.
The present disclosure relates to a visual aid for assisting a user in the preparation of a plurality of syringes, the visual aid comprising a plurality of images, wherein each image: is visually representative of a respective syringe of the plurality of syringes; and comprises a respective first visual feature that is representative of a respective first visual feature of the respective syringe and a respective second visual feature that is representative of a respective second visual feature of the respective syringe, the visual features arranged such that they indicate a respective volume of a medicament to be drawn into the respective syringe. The present disclosure also relates to a system comprising a visual aid and a plurality of syringes. The present disclosure also relates to a method of using a visual aid.
A61M 5/00 - Devices for bringing media into the body in a subcutaneous, intra-vascular or intramuscular wayAccessories therefor, e.g. filling or cleaning devices, arm rests
A vial adapter comprises an adapter body comprising a top wall and a sidewall projecting from the top wall in a longitudinal direction to form a receptacle with a distal side of the top wall. The receptacle is configured to receive at least a portion of a barrel head of an injection vial. A movable part comprises a base part and a spike fixed to the base part. The spike extends through the top wall in a distal direction into the receptacle. The movable part is movable relative to the adapter body from an undeployed position into a deployed position. An interlock is operable to lock the movable part in the undeployed position, and an actuating member extending into the receptacle is coupled to the interlock and operable to unlock the interlock when actuated.
The invention is related to methods of producing rod-derived cone viability factor (RdCVF). This invention also relates to the treatment of an ocular disease in a mammal using RdCVF. Also provided are expression vectors for high secreted expression of RdCVF of using nucleotide sequences encoding heterologous signal proteins and optionally markers for furin cleavage.
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
44.
VIAL ADAPTER AND INJECTION KIT FOR WITHDRAWING A LIQUID MEDICAMENT FROM AN INJECTION VIAL
A vial adapter comprises an adapter body comprising a top wall and a sidewall projecting from the top wall in a longitudinal direction to form a receptacle with a distal side of the top wall. The receptacle is configured to receive at least a portion of a barrel head of an injection vial. A movable part comprises a base part and a spike fixed to the base part. The spike extends through the top wall in a distal direction into the receptacle. An actuating member is mechanically coupled to the movable part, and is movable relative to the adapter body from a start position to an end position along a first direction to induce a distally directed displacement of the movable part relative to the adapter body. The mechanical coupling between the movable part and the actuating member is releasable when the actuating member is in the end position.
The invention provides ionizable cationic lipids and lipid nanoparticles for the delivery of nucleic acids to immune cells, and methods of making and using, such lipids and targeted lipid nanoparticles.
A61K 31/395 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
C07D 233/61 - Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings having two double bonds between ring members or between ring members and non-ring members with only hydrogen atoms or radicals containing only hydrogen and carbon atoms, attached to ring carbon atoms with hydrocarbon radicals, substituted by nitrogen atoms not forming part of a nitro radical, attached to ring nitrogen atoms
46.
VIAL ADAPTER AND INJECTION KIT FOR WITHDRAWING A LIQUID MEDICAMENT FROM AN INJECTION VIAL
A vial adapter for connecting to an injection vial comprises an adapter body comprising a top wall and a sidewall, the sidewall projecting from the top wall in a longitudinal direction to form a receptacle with a distal side of the top wall. The receptacle is configured to receive at least a portion of a barrel head of an injection vial. A movable part comprises a base part and a tipped cannula fixed to the base part. The tipped cannula extends through the top wall into the receptacle, wherein the movable part is movable relative to the adapter body in a longitudinal direction. The tipped cannula comprises an elongated fluid channel, a tipped distal end configured to penetrate a pierceable stopper of the injection vial, and a channel aperture located inside the receptacle and located proximally offset from the tipped distal end.
A medical device for mixing a medicament contained within a medicament vial, wherein the medical device comprises a track configured to support the medicament vial, and wherein the track comprises an inclined first track section arranged such that, in use, the medicament vial can roll along the track to mix the medicament contained within the medicament vial. A system comprising the medical device and a medicament vial containing the medicament. A method for using the medical device.
The present disclosure relates to a visual aid for assisting a user in the preparation of a plurality of syringes, the visual aid comprising a plurality of images, wherein each image: is visually representative of a respective syringe of the plurality of syringes; and comprises a respective first visual feature that is representative of a respective first visual feature of the respective syringe and a respective second visual feature that is representative of a respective second visual feature of the respective syringe, the visual features arranged such that they indicate a respective volume of a medicament to be drawn into the respective syringe. The present disclosure also relates to a system comprising a visual aid and a plurality of syringes. The present disclosure also relates to a method of using a visual aid.
A vial adapter for connecting to an injection vial comprises an adapter body comprising a top wall and a sidewall, the sidewall projecting from the top wall in a longitudinal direction to form a receptacle with a distal side of the top wall. The receptacle is configured to receive at least a portion of a barrel head of an injection vial. A movable part comprises a base part and a tipped cannula fixed to the base part. The tipped cannula extends through the top wall into the receptacle, wherein the movable part is movable relative to the adapter body in a longitudinal direction. The tipped cannula comprises an elongated fluid channel, a tipped distal end configured to penetrate a pierceable stopper of the injection vial, and a channel aperture located inside the receptacle and located proximally offset from the tipped distal end.
A vial adapter comprises an adapter body comprising a top wall and a sidewall projecting from the top wall in a longitudinal direction to form a receptacle with a distal side of the top wall. The receptacle is configured to receive at least a portion of a barrel head of an injection vial. A movable part comprises a base part and a spike fixed to the base part. The spike extends through the top wall in a distal direction into the receptacle. The movable part is movable relative to the adapter body from an undeployed position into a deployed position. An interlock is operable to lock the movable part in the undeployed position, and an actuating member extending into the receptacle is coupled to the interlock and operable to unlock the interlock when actuated.
The present disclosure relates generally to compositions and methods for gene therapy, and more specifically to delivering mRNA-based therapeutics to immune cells in vivo.
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 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 47/68 - 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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
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
52.
INDOLYL-PYRIDINONES AND RELATED COMPOUNDS AND THEIR USE IN THERAPY
The invention provides indolyl-pyridinones and related compounds, pharmaceutical compositions, their use for inhibiting signal transducer and activator of transcription 6 (STAT6), and their use in the treatment of a disease or condition, such as an inflammatory disorder, allergic condition, or proliferative disorder.
In one aspect the present disclosure relates to a mobile electronic device (10) for illuminating veins (9) of a human or animal body, the mobile electronic device (10) comprising: - a housing (11), - a tunable light source (20; 26) inside or attached to the housing (11) and configured to emit illuminating light (21) at a correlated color temperature, - a processor (40) inside the housing (11) and coupled to the tunable light source (20; 26) to adjust the correlated color temperature of the tunable light source (20; 26).
RECOMBINANT AAV VECTORS EXPRESSING OSTEOPROTECTIVE GENES, INCLUDING HAS2 AND LUBRICIN, USEFUL IN THE TREATMENT OF OSTEOARTHRITIS AND RELATED JOINT CONDITIONS IN MAMMALS
The present disclosure relates to recombinant viral vectors, to pharmaceutical compositions comprising such recombinant vectors, and to methods for prevention and treatment of osteoarthritis in mammals. In particular, this disclosure provides adeno-associated virus (AAV) vectors capable of expressing, in a host, osteoprotective/chondroprotective bioactive proteins, including hyaluronan synthase 2 (HAS2) and lubricin (PRG4). Methods of production of these AAV are provided, as are methods of treatment of osteoarthritis in mammalian joints, by the long-term gene expression of osteoprotective/chondroprotective proteins, including HAS2 and PRG4, in both synovial and chondrocyte cells.
A medicament delivery device for injecting medicament is described. The device has a body having a proximal end and a distal end, a needle for injecting medicament, an actuation member which is movable relative to the body from a first position to a second position for dispensing medicament from the needle, a lock ring which is rotatable relative to the body from a pre-use position, in which movement of the actuation member from the first position to the second position is prevented, to a use position in which movement of the actuation member from the first position to the second position is permitted and a cap which is removably attached to the medicament delivery device. The cap has a portion which is configured to prevent rotation of the lock ring from the pre-use position to the use position when the cap is attached to the medicament delivery device.
Provided are lipid nanoparticles, compositions, and methods of making and using the same. The lipid nanoparticles are formulated to specifically target CNS endothelial cells. The lipid nanoparticles may contain a CNS endothelial cell targeting group coupled to a PEG- lipid. The lipid nanoparticles may carry a cargo, e g., a mRNA. The lipid nanoparticles are used for transfection of CNS endothelial cells.
A61K 47/68 - 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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
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
A61P 25/00 - Drugs for disorders of the nervous system
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
57.
METHODS FOR ENHANCED REMOVAL OF IMPURITIES DURING PROTEIN A CHROMATOGRAPHY
Provided herein are methods relating to the purification of a polypeptide comprising an Fc region (e.g., an antibody) via protein A chromatography; methods relating to the use of a wash solution comprising a benzoate salt and/or benzyl alcohol during protein A chromatography; and methods of adjusting a harvest using sodium benzoate prior to protein A chromatography.
C07K 1/22 - Affinity chromatography or related techniques based upon selective absorption processes
B01D 15/38 - Selective adsorption, e.g. chromatography characterised by the separation mechanism involving specific interaction not covered by one or more of groups , e.g. affinity, ligand exchange or chiral chromatography
B01D 15/42 - Selective adsorption, e.g. chromatography characterised by the development mode, e.g. by displacement or by elution
C07K 1/34 - ExtractionSeparationPurification by filtration, ultrafiltration or reverse osmosis
C07K 1/36 - ExtractionSeparationPurification by a combination of two or more processes of different types
C07K 16/00 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies
The hemitartrate salt of a compound represented by the following structural formula:
The hemitartrate salt of a compound represented by the following structural formula:
The hemitartrate salt of a compound represented by the following structural formula:
(Formula I Hemitartrate), which may be used in pharmaceutical applications, are disclosed. Particular single crystalline forms of the Formula (I) Hemitartrate are characterized by a variety of properties and physical measurements. As well, methods of producing crystalline Formula (I) Hemitartrate, and using it to inhibit glucosylceramide synthase or lowering glycosphingolipid concentrations in subjects to treat a number of diseases, are also discussed. Pharmaceutical compositions are also described.
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 31/357 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having two or more oxygen atoms in the same ring, e.g. crown ethers, guanadrel
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
Methods for inserting a targeting moiety (such as an antigen-binding protein moiety, a fragment antigen-binding moiety, or the like) into a lipid nanoparticle and compositions resulting from such methods are provided. The methods generally utilize a reaction that forms targeted nanoparticles by combining a targeting moiety and a lipid nanoparticle. A quenching operation is then provided by cooling the reaction to a temperature that stops the insertion of the targeting moiety. The reaction substantially preserves the integrity of a nucleic acid cargo (such as an mRNA encoding a VHH binding molecule).
A61K 47/68 - 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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
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
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Pharmaceutical preparations for the treatment and prevention of cardiovascular diseases and diseases of the central nervous system; Pharmaceutical preparations for the treatment of cancer, thrombosis, and respiratory diseases; Pharmaceutical preparations for the treatment of rare diseases, namely, lysosomal storage disorders and autosomal dominant polycystic kidney disease; Pharmaceutical preparations for the treatment and prevention of infectious diseases, namely, viral and bacterial infections
The present disclosure provides methods of producing recombinant adeno-associated virus (rAAV) wherein rAAV production occurs under a controlled partial pressure of carbon dioxide. Also are methods of producing rAAV under air sparge conditions for modulating the partial pressure of carbon dioxide.
Provided herein are methods of selecting an optimal fixed orientation of a dissolved oxygen (DO) probe in a perfusion bioreactor and culturing a mammalian cell in a perfusion bioreactor having the DO probe positioned at the selected fixed orientation.
Methods for inserting a targeting moiety (such as an antigen-binding protein moiety, a fragment antigen-binding moiety, or the like) into a lipid nanoparticle and compositions resulting from such methods are provided. The methods generally utilize a reaction that forms targeted nanoparticles by combining a targeting moiety and a lipid nanoparticle. A quenching operation is then provided by cooling the reaction to a temperature that stops the insertion of the targeting moiety. The reaction substantially preserves the integrity of a nucleic acid cargo (such as an mRNA encoding a VHH binding molecule).
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 47/18 - AminesAmidesUreasQuaternary ammonium compoundsAmino acidsOligopeptides having up to five amino acids
A61K 47/22 - Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
A61K 47/68 - 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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
65.
PROCESS FOR PREPARING (S)-5-BENZYL-N-(5-METHYL-4-OXO-2,3,4,5- TETRAHYDROPYRIDO[3,2-B][L,4]OXAZEPIN-3-YL)-4H-L,2,4-TRIAZOLE-3- CARBOXAMIDE
Provided herein are methods for preparing (S)-5-benzyl-N-(5-methyl-4-oxo-2,3,4,5-tetrahydropyrido[3,2-b][1,4]oxazepin-3-yl)-4H-1,2,4-triazole-3-carboxamide and intermediates useful therein.
A61K 31/553 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole having at least one nitrogen and at least one oxygen as ring hetero atoms, e.g. loxapine, staurosporine
The present invention relates to humanized monoclonal antibodies comprising the CDRs of murine antibody BMA031, which bind to the αβTCR.CD3 complex and possess improved biological properties.
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
In one aspect the present disclosure relates to an adapter for connecting to at least one of a diluent vial (80) and an injection vial (90), wherein the diluent vial (80) or injection vial (90) comprises a barrel (81, 91) and a barrel head (82, 92) with a pierceable seal (87, 97), the adapter (10) comprising: an adapter body (11) comprising a vial mount (12) at a distal end (13) and a needle interface (14) at a proximal end (15), wherein the vial mount (12) is configured to detachably connect to the barrel head (82, 92) and wherein the needle interface (14) is configured to receive an injection needle (60), a needle passage (24) extending through the adapter body (11) from the needle interface (14) to the vial mount (12) and configured to guide the injection needle (60) longitudinally through the adapter body (11).
Provided herein are humanized rodent models comprising a human SERPINA1 transgene, optionally wherein the rodents develop Alpha- 1 antitrypsin deficiency (A1ATD). Further provided herein are methods related to the use of the humanized rodent models for screening therapeutic efficacy for A1ATD, for example, for screening the efficacy of any treatment selected from the group consisting of liver transplantation, respiratory agents, purified alpha- 1 antitrypsin (A1AT) protein, and gene therapy.
The invention provides ionizable cationic lipids and lipid nanoparticles for the delivery of nucleic acids to target cells, such as immune cells and hematopoietic stem cells, and methods of making and using, such lipids and targeted lipid nanoparticles.
C07D 317/28 - Radicals substituted by nitrogen atoms
A61K 9/127 - Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
A61K 47/50 - 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
A61K 47/54 - 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 compound
C07D 317/30 - Radicals substituted by carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals
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
The present disclosure provides methods of producing in a single cell a mixed population of recombinant adeno-associated viruses for expressing two or more transgenes and methods of producing recombinant adeno-associated viruses containing self-complementary genomes. Also provided are methods of treating diseases using the rAAV compositions produced herein.
The present disclosure provides a method of reducing the levels of preexisting neutralizing antibodies against adeno-associated viruses (AAV) prior to an AAV-mediated gene therapy to treat an ocular disease.
A61K 38/16 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
The present disclosure is related to improved compositions and methods for treating T-cell-mediated diseases and disorders (e.g., autoimmune disorders, graft-versus-host-disease, and graft rejection). Provided are anti-αβTCR binding polypeptides, including antibodies, which comprise at least one amino acid substitution or modification that increases the stability of the binding polypeptide by reducing fragmentation of the light chain variable region. The methods provided herein generally involve administering to a subject in need thereof an effective amount of a stabilized, humanized binding polypeptide that is specific to the alpha beta T-cell receptor (αβTCR).
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
A medicament delivery device has a needle for injecting medicament, a body and a needle cover. The needle cover is axially movable relative to a locked position in which the needle cover covers the distal end of the needle. The device has an inner housing with an aperture. A collar is located at least partially within the inner housing and is configured to rotate relative to the inner housing. The collar has one or more arms. A rotational member rotates the collar from a first position to a second position. When the needle cover is in the locked position and the collar is in the second position then the arm extends radially through the aperture and blocks proximal movement of the needle cover away from the locked position.
A61M 5/20 - Automatic syringes, e.g. with automatically actuated piston rod, with automatic needle injection, filling automatically
A61M 5/32 - NeedlesDetails of needles pertaining to their connection with syringe or hubAccessories for bringing the needle into, or holding the needle on, the bodyDevices for protection of needles
The present disclosure relates to a medicament delivery device comprising a housing, a needle, a needle cover, a needle cover biasing member, a drive mechanism, and a needle cover extension mechanism. The needle cover is axially movable between an extended position and a retracted position. The needle cover biasing member is configured to bias the needle cover axially in the distal direction towards the extended position. The needle cover extension mechanism comprises a second biasing member and a release element. The second biasing member is configured to bias the needle cover into its extended position post-use of the device. The release element has a first state configured to prevent the second biasing member from biasing the needle cover into the extended position and a second state configured to allow the second biasing member to bias the needle cover into the extended position.
A61M 5/20 - Automatic syringes, e.g. with automatically actuated piston rod, with automatic needle injection, filling automatically
A61M 5/32 - NeedlesDetails of needles pertaining to their connection with syringe or hubAccessories for bringing the needle into, or holding the needle on, the bodyDevices for protection of needles
A61M 5/32 - NeedlesDetails of needles pertaining to their connection with syringe or hubAccessories for bringing the needle into, or holding the needle on, the bodyDevices for protection of needles
A61M 5/20 - Automatic syringes, e.g. with automatically actuated piston rod, with automatic needle injection, filling automatically
A61M 5/315 - PistonsPiston-rodsGuiding, blocking or restricting the movement of the rodAppliances on the rod for facilitating dosing
A medicament delivery device comprises: a needle for injecting a medicament; a body; a needle cover axially movable relative to the body between an extended position and a retracted position; a needle cover biasing member configured to bias the needle cover distally; a medicament delivery mechanism; and a latch movable by the needle cover between an engaged configuration, in which the latch prevents delivery of the medicament, and a disengaged configuration, in which the latch allows delivery of the medicament, wherein the needle cover is configured such that: proximal movement of the needle cover from the extended position towards the retracted position moves the latch from the engaged configuration to the disengaged configuration; and distal movement of the needle cover from the retracted position towards the extended position moves the latch from the disengaged configuration to the engaged configuration.
A61M 5/20 - Automatic syringes, e.g. with automatically actuated piston rod, with automatic needle injection, filling automatically
A61M 5/32 - NeedlesDetails of needles pertaining to their connection with syringe or hubAccessories for bringing the needle into, or holding the needle on, the bodyDevices for protection of needles
A61M 5/315 - PistonsPiston-rodsGuiding, blocking or restricting the movement of the rodAppliances on the rod for facilitating dosing
A medicament delivery device comprises: a body; a needle cover axially movable between an extended position and a retracted position; a medicament delivery mechanism comprising a plunger and a drive member configured to move the plunger to dispense a medicament; an actuation member configured to be actuated relative to the body; and a ratchet mechanism coupled to the medicament delivery mechanism and sequentially movable between a first configuration, second configuration and third configuration, wherein the actuation member and ratchet mechanism are arranged such that: a first actuation of the actuation member moves the ratchet mechanism from the first configuration to the second configuration to cause a first dose of the medicament to be dispensed, and a second actuation of the actuation member, subsequent to the first actuation, moves the ratchet mechanism from the second configuration to the third configuration to cause a second dose of the medicament to be dispensed.
A61M 5/20 - Automatic syringes, e.g. with automatically actuated piston rod, with automatic needle injection, filling automatically
A61M 5/315 - PistonsPiston-rodsGuiding, blocking or restricting the movement of the rodAppliances on the rod for facilitating dosing
A61M 5/32 - NeedlesDetails of needles pertaining to their connection with syringe or hubAccessories for bringing the needle into, or holding the needle on, the bodyDevices for protection of needles
A medicament delivery device has a needle, a body and a needle cover which is axially movable from an initial position to a holding position to a locked position in which the needle cover covers the distal end of the needle. The device has a biasing member configured to bias the needle cover distally, and a guide member configured to rotate relative to the needle cover. One of the guide member and the needle cover has a pin and the other of the guide member and the needle cover has a guide path for guiding the pin. The guide path has a first ramp, a second ramp and a locking abutment surface. When the needle cover is in the locked position then the locking abutment surface is configured to engage the pin to prevent the needle cover from moving proximally away from the locked position.
A61M 5/32 - NeedlesDetails of needles pertaining to their connection with syringe or hubAccessories for bringing the needle into, or holding the needle on, the bodyDevices for protection of needles
A61M 5/20 - Automatic syringes, e.g. with automatically actuated piston rod, with automatic needle injection, filling automatically
A medicament delivery device has a needle for injecting medicament into a user, a body having a proximal end and a distal end and a needle cover. The needle cover is axially movable relative to the body between an initial position, in which the needle cover covers the distal end of the needle, and a holding position. In the holding position the needle protrudes from the distal end of the needle cover. The needle cover has a flexible arm. The device further has a needle cover guide comprising a track. The flexible arm engages the track. The track has a radially-extending ramp configured to flex the flexible arm when the needle cover moves from the initial position to the holding position. The flexible arm is flexed when the needle cover is in the holding position.
A61M 5/32 - NeedlesDetails of needles pertaining to their connection with syringe or hubAccessories for bringing the needle into, or holding the needle on, the bodyDevices for protection of needles
The present disclosure relates to a medicament delivery device. The medicament delivery device comprises a housing, a needle, and a needle cover. The needle cover is axially movable between an extended position and a retracted position. The medicament delivery device comprises a needle cover biasing member configured to bias the needle cover axially in the distal direction towards the extended position. The needle cover biasing member is configured to transition from a non-buckled state to a buckled state during proximal movement of the needle cover from the extended position to the retracted position. The present disclosure also relates to a method of preparing a medicament delivery device, and a method of locking a medicament delivery device.
A61M 5/32 - NeedlesDetails of needles pertaining to their connection with syringe or hubAccessories for bringing the needle into, or holding the needle on, the bodyDevices for protection of needles
A medicament delivery device comprises a housing, a needle, a needle cover, a needle cover biasing member, a skin sensor, a skin sensor biasing member, and a retaining mechanism. The needle cover is axially movable between an extended position and a retracted position. The needle cover biasing member is configured to bias the needle cover towards the extended position. The skin sensor extends from the distal end of the needle cover and is configured to contact an injection site. The skin sensor biasing member is configured to bias the skin sensor from a proximal position to a distal position away from the needle cover. The retaining mechanism is configured to temporarily retain the needle cover in the retracted position and transfer at least a part of the force of the needle cover biasing member to the housing to prevent the needle cover biasing member from moving the needle cover distally.
A61M 5/32 - NeedlesDetails of needles pertaining to their connection with syringe or hubAccessories for bringing the needle into, or holding the needle on, the bodyDevices for protection of needles
There is provided a medicament delivery device comprising: a main body arranged to receive a medicament cartridge; a needle for delivery of medicament from the medicament cartridge; a needle cover moveable between an initial position, in which the needle cover covers the needle, and an activated position for dispensing medicament from the medicament delivery device, wherein in the activated position the needle protrudes from a distal end of the needle cover; an outer frame, moveable relative to the main body between an extended position in which the outer frame extends past a distal end of the needle, and a retracted position in which the outer frame is moved towards the main body; an attachment element comprising an adhesive region for attaching the medicament delivery device to a surface when the needle cover is in the activated position, the attachment element comprising a protective barrier covering the adhesive region; wherein the protective barrier is coupled to the outer frame and is removed from the attachment element to expose the adhesive region as the outer frame is moved towards the retracted position. A method of operating a medicament delivery device is also provided.
A61M 5/32 - NeedlesDetails of needles pertaining to their connection with syringe or hubAccessories for bringing the needle into, or holding the needle on, the bodyDevices for protection of needles
The present disclosure relates to a medicament delivery device. The medicament delivery device comprises a housing, a needle, and a needle cover. The needle cover is axially movable between an extended position and a retracted position. The medicament delivery device comprises a needle cover biasing member configured to bias the needle cover axially in the distal direction towards the extended position. The needle cover biasing member is configured to transition from a non-buckled state to a buckled state during proximal movement of the needle cover from the extended position to the retracted position. The present disclosure also relates to a method of preparing a medicament delivery device, and a method of locking a medicament delivery device.
A61M 5/32 - NeedlesDetails of needles pertaining to their connection with syringe or hubAccessories for bringing the needle into, or holding the needle on, the bodyDevices for protection of needles
A61M 5/20 - Automatic syringes, e.g. with automatically actuated piston rod, with automatic needle injection, filling automatically
A medicament delivery device has a needle for injecting medicament into a user, a body having a proximal end and a distal end and a needle cover. The needle cover is axially movable relative to the body between an initial position, in which the needle cover covers the distal end of the needle, and a holding position. In the holding position the needle protrudes from the distal end of the needle cover. The needle cover has a flexible arm. The device further has a needle cover guide comprising a track. The flexible arm engages the track. The track has a radially-extending ramp configured to flex the flexible arm when the needle cover moves from the initial position to the holding position. The flexible arm is flexed when the needle cover is in the holding position.
A61M 5/20 - Automatic syringes, e.g. with automatically actuated piston rod, with automatic needle injection, filling automatically
A61M 5/32 - NeedlesDetails of needles pertaining to their connection with syringe or hubAccessories for bringing the needle into, or holding the needle on, the bodyDevices for protection of needles
85.
ADENO-ASSOCIATED VIRAL VECTORS FOR TREATING MYOCILIN (MYOC) GLAUCOMA
Provided herein are methods for treating myocilin (MYOC) glaucoma using adeno-associated viral (AAV) vectors encoding R-spondin 3 (RSPO3). In one aspect, viral particles are administered to the eye of a human subject. Viral particles encoding RSPO3 are contemplated. In some aspects, variant AAV2 particles encoding RSPO3 that transduce the trabecular meshwork are provided.
A61K 31/711 - Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
Provided herein are methods to characterize preparations of recombinant viral particles using analytical ultracentrifugation. Recombinant viral particles include recombinant adeno- associated viral particles, recombinant adenoviral particles, recombinant lentiviral particles and recombinant herpes simplex virus particles. Variant species of recombinant viral particles including empty capsids and recombinant viral particles with variant genomes (e.g., truncated genomes, aggregates, recombinants) can be identified and quantitated. The methods can be used to characterize preparations of recombinant viral particles regardless of the sequence of the recombinant viral genome or the serotype of the recombinant viral capsid.
A preparation assembly for preparing a liquid medicament, the preparation assembly comprising: - a flexible bag, wherein the flexible bag comprises: - a first medicament chamber portion comprising a first medicament chamber wall confining a first medicament chamber cavity configured to hold a first medicament, - a second medicament chamber portion comprising a second medicament chamber wall confining a second medicament chamber cavity configured to hold a second medicament, - a diluent chamber portion comprising a diluent chamber wall confining a diluent chamber cavity configured to hold a diluent, the preparation assembly further comprising: - a first fluid path between the diluent chamber cavity and the first medicament chamber cavity, - a second fluid path between the diluent chamber cavity and the second medicament chamber cavity, - a first valve device located in the first fluid path, - a second valve device located in the second fluid path.
A preparation assembly for preparing a liquid medicament, the preparation assembly comprises: - a first preparation container, a second preparation container and a first connecting portion mutually mechanically connecting the first preparation container and the second preparation container, wherein the preparation assembly is transferable from a storage configuration into a set-up configuration.
Provided herein are novel methods for delivering recombinant adeno-associated viral (rAAV) particles to the central nervous system of a mammal (e.g., a human). In aspects, the methods involve administering rAAV particles containing a heterologous nucleic acid to the striatum and causing expression of the heterologous nucleic acid in at least the cerebral cortex and the striatum of the mammal.
The disclosure relates, at least in part, to methods and compositions for improving rAAV production. The disclosure provides methods of contacting a cell culture with one or more enhancer elements to improve rAAV yield.
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
A61K 47/68 - 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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
A medicament delivery device comprising: a proximal end and a distal end defining a longitudinal axis; a body; a button arranged at the proximal end and configured to actuate the medicament delivery device; and a lock ring configured to rotate relative to the body about the longitudinal axis between a first position in which the button is not permitted to move along the longitudinal axis relative to the body, and a second position in which the button is permitted to move along the longitudinal axis relative to the body; wherein movement of the lock ring from the first position towards the second position causes the button to move along the longitudinal axis relative to the body.
A61M 5/20 - Automatic syringes, e.g. with automatically actuated piston rod, with automatic needle injection, filling automatically
A61M 5/32 - NeedlesDetails of needles pertaining to their connection with syringe or hubAccessories for bringing the needle into, or holding the needle on, the bodyDevices for protection of needles
Provided herein are methods for isolating intracellular products (e.g., viral particles or nucleic acid molecules) from a suspension of cells. Also provided herein are also methods for mechanically lysing cells. In some embodiments, provided herein are methods for mechanically lysing cells for isolating intracellular products (e.g., viral particles or nucleic molecules) from a suspension of cells. Also provided herein are apparatuses and cartridges for use in mechanically lysing cells.
The present invention relates to a method for targeted generation of degradants from a biological product exposed to a low pH stress, wherein said method comprises a) selecting a set of different low pH stress conditions, wherein each stress condition is determined by a combination of a low pH and at least two additional factors, b) exposing said biological product to each low pH stress condition of said set, c) analyzing the degradants obtained in each low pH stress condition, d) processing the data obtained from said analysis, to obtain a predictive model of the degradants generated in function of the low pH and of the at least two additional factors and e), optionally, determining from said predictive model, a combination of values or ranges of values of low pH and of the at least two additional factors to be used to generate desired degradants from said biological product exposed to a low pH stress.
The specification describes systems, methods, and apparatus for using machine-learning techniques to assess the severity of gastrointestinal diseases such as ulcerative colitis. According to a first aspect of this specification, there is described a computer implemented method for assessing the severity of a gastrointestinal disease, the method comprising: receiving a video of an endoscopy performed on a portion of a gastrointestinal tract of a subject, the video comprising a plurality of frames; determining, based on the plurality of frames, a local z-axis motion of a camera used to generate the endoscopy video; based on the local z-axis motion, processing, using one or more severity classification models, the plurality of frames to generate a plurality of severity scores, each severity score indicative of a severity of the gastrointestinal disease at a different location within the gastrointestinal tract; and generating, based on the plurality of severity scores, an overall severity score.
The present disclosure provides using a double-stranded oligonucleotide compound as a novel therapy to improve the quality of life and joint function of patients with hemophilia A and hemophilia B.
The present disclosure provides methods of producing recombinant adeno-associated virus (rAAV) wherein rAAV production occurs under perfusion conditions. Also contemplated are methods of producing rAAV under media exchange conditions.
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 31/4439 - 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 31/444 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring hetero atom, e.g. amrinone
A61K 31/496 - Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
A61K 31/497 - Non-condensed pyrazines containing further heterocyclic rings
A61K 31/504 - PyridazinesHydrogenated pyridazines forming part of bridged ring systems
A61K 31/5377 - 1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
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
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
100.
Macrocyclic compounds, compositions, and methods of using thereof
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 31/4439 - 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 31/444 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring hetero atom, e.g. amrinone
A61K 31/496 - Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
A61K 31/497 - Non-condensed pyrazines containing further heterocyclic rings
A61K 31/504 - PyridazinesHydrogenated pyridazines forming part of bridged ring systems
A61K 31/5377 - 1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
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
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
