There are disclosed certain 3-azabicyclo[3.1.0]hexanes, and pharmaceutically acceptable salts thereof, together with compositions containing them and their use in therapy. The compounds are GLP-1 receptor modulators and are thereby particularly useful in the treatment or prophylaxis of cardiovascular disease and metabolic conditions, for example Type 2 diabetes.
There are disclosed certain 3-azabicyclo[3.1.0]hexanes, and pharmaceutically acceptable salts thereof, together with compositions containing them and their use in therapy. The compounds are GLP-1 receptor modulators and are thereby particularly useful in the treatment or prophylaxis of cardiovascular disease and metabolic conditions, for example Type 2 diabetes.
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
A61K 31/4184 - 1,3-Diazoles condensed with carbocyclic rings, e.g. benzimidazoles
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
Provided herein is a vaccine against RSV. The vaccine comprises an mRNA encoding a stabilised prefusion RSV F protein immunogen linked to a scaffold based on lumazine synthase.
The present disclosure generally relates to antibodies, antigen binding fragments thereof, polypeptides, and immunoconjugates that bind to CD123 antigen (the α chain of the interleukin-3 receptor) and deliver a payload to CD123 expressing cells. The payload is a topoisomerase inhibitor that efficiently kills CD123 expressing tumor cells while sparing hematopoietic stem cells and mature hematopoietic cells.
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 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
The present disclosure provides a non-naturally occurring CRISPR-Cas system comprising: a Cas9 effector protein capable of generating cohesive ends (stiCas9), and a guide polynucleotide that forms a complex with the stiCas9 and comprising a guide sequence, wherein the guide sequence hybridizes with a target sequence in a eukaryotic cell but does not hybridize to a sequence in a bacterial cell, and wherein the complex does not occur in nature. The present disclosure also provides a method of introducing a sequence of interest into a chromosome of a cell. Finally, the present disclosure provides for a method of modifying one or more nucleotides using seamless mutagenesis.
Provided are multivalent saccharide-containing compounds which are bonded to nucleic acids. These compounds comprise one or more nitrogen-modified phosphate groups which link the cargo moieties (e.g., therapeutic nucleic acids) and/or the ligands (e.g., GalNAc) to the core of the complex. The compounds are useful for delivering nucleic acids to cells or tissues, e.g. for use in therapeutic treatments. Also provided are pharmaceutical compositions comprising the aforementioned compounds and medical uses of the same, including their use in treating or preventing conditions such as liver diseases.
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 1/16 - Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
8.
MULTIVALENT CARGO-CARRYING COMPLEXES AND USES THEREOF
Provided are multivalent saccharide-containing compounds which are bonded to nucleic acids. These compounds contain a cysteine-derived core and are useful for delivering nucleic acids to cells or tissues, e.g. for use in therapeutic treatments. Also provided are pharmaceutical compositions comprising the aforementioned compounds and medical uses of the same, including their use in treating or preventing conditions such as liver diseases.
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 1/16 - Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
The invention relates to a method of treating or preventing a neurodegenerative disease in a patient in need thereof, the method comprising administering to the patient a therapeutically effective amount of an inhibitor of type I IFN signalling. Also provided are pharmaceutical compositions for use in such methods, and related kits and injection devices.
C07K 16/24 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
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 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans
A61P 9/04 - Inotropic agents, i.e. stimulants of cardiac contractionDrugs for heart failure
A61K 39/00 - Medicinal preparations containing antigens or antibodies
10.
MULTIVALENT CARGO-CARRYING COMPLEXES AND USES THEREOF
Provided are multivalent saccharide-containing compounds which are bonded to nucleic acids. These compounds contain a shikimic acid-derived core and are useful for delivering nucleic acids to cells or tissues, e.g. for use in therapeutic treatments. Also provided are pharmaceutical compositions comprising the aforementioned compounds and medical uses of the same, including their use in treating or preventing conditions such as liver diseases.
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 1/16 - Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
Provided herein is a vaccine against RSV. The vaccine comprises an mRNA encoding a stabilised prefusion RSV F protein immunogen linked to a scaffold based on lumazine synthase.
The present invention relates to pharmaceutical compositions suitable for oral administration, and more particularly to pharmaceutical compositions, including pharmaceutical tablet compositions, containing N-(2-{2-dimethylaminoethyl-methylamino}-4-methoxy-5-{[4-(1-methylindol-3-yl)pyrimidin-2-yl]amino}phenyl)prop-2-enamide (“AZD9291”) or a pharmaceutically acceptable salt thereof, wherein such compositions comprise a certain amount of microcrystalline cellulose and at least one other pharmaceutical diluent.
The specification relates to compounds of Formula (I):
The specification relates to compounds of Formula (I):
The specification relates to compounds of Formula (I):
and to pharmaceutically acceptable salts thereof, to processes and intermediates used for their preparation, to pharmaceutical compositions containing them and to their use in the treatment of diseases such as liver disease.
C07D 413/06 - 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 only aliphatic carbon atoms
C07D 409/06 - 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 carbon chain containing only aliphatic carbon atoms
C07D 413/04 - 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 directly linked by a ring-member-to-ring- member bond
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
The invention concerns pharmaceutical compositions containing a hydrogen sulphate salt of 6-(4-bromo-2-chloro-phenylamino)-7-fluoro-3-methyl-3H-benzoimidazole-5-carboxylic acid (2-hydroxy-ethoxy)-amide and solvates, crystalline forms and amorphous forms thereof, to the use of said compositions as a medicament, and to processes for the preparation of said compositions.
A61K 9/48 - Preparations in capsules, e.g. of gelatin, of chocolate
A61K 47/22 - Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
A61K 47/36 - PolysaccharidesDerivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
C07D 235/06 - BenzimidazolesHydrogenated benzimidazoles with only hydrogen atoms, hydrocarbon or substituted hydrocarbon radicals, directly attached in position 2
The present disclosure relates to binding proteins (e.g. antibodies) that specifically bind LILRB2. The present disclosure also provides methods of treatment, uses, pharmaceutical compositions and kits comprising the binding proteins (e.g. antibodies).
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
The specification relates to epidermal growth factor receptor (EGFR) tyrosine kinase inhibitors (TKIs) for use in the adjuvant treatment after tumour resection of patients with epidermal growth factor receptor-mutation-positive (EGFRm) non-small cell lung cancer (NSCLC).
The present invention relates to certain 2-(2,4,5-substituted-anilino)pyrimidine compounds and pharmaceutically acceptable salts thereof which may be useful in the treatment or prevention of a disease or medical condition mediated through certain mutated forms of epidermal growth factor receptor (for example the L858R activating mutant, the Exon19 deletion activating mutant and the T790M resistance mutant). Such compounds and salts thereof may be useful in the treatment or prevention of a number of different cancers. The invention also relates to pharmaceutical compositions comprising said compounds and salts thereof, especially useful polymorphic forms of these compounds and salts, intermediates useful in the manufacture of said compounds and to methods of treatment of diseases mediated by various different forms of EGFR using said compounds and salts thereof.
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
C07D 401/02 - 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
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 403/04 - 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 directly linked by a ring-member-to-ring- member bond
Provided herein are compositions and methods that permit the controlled interaction of polypeptides to which a target protein and binding protein are fused. The compositions and methods make use of a target protein that binds to the hepatitis C virus protease inhibitor grazoprevir to form a complex, and a Tn3 binding protein that specifically binds the complex. The target protein is or is derived from a viral protease, such as the HCV NS3/4A protease.
There are disclosed certain 2,5-diazabicyclo[4.2.0]octanes of formula (I), and pharmaceutically acceptable salts thereof, together with compositions containing them and their use in therapy. The compounds are GLP-1 receptor modulators and are thereby particularly useful in the treatment or prophylaxis of cardiovascular disease and metabolic conditions, for example Type 2 diabetes.
There are disclosed certain 2,5-diazabicyclo[4.2.0]octanes of formula (I), and pharmaceutically acceptable salts thereof, together with compositions containing them and their use in therapy. The compounds are GLP-1 receptor modulators and are thereby particularly useful in the treatment or prophylaxis of cardiovascular disease and metabolic conditions, for example Type 2 diabetes.
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
A61K 31/498 - Pyrazines or piperazines ortho- or peri-condensed with carbocyclic ring systems, e.g. quinoxaline, phenazine
The present disclosure provides compounds represented by Formula (A), or a pharmaceutically acceptable salt and/or stereoisomer thereof: (A), wherein Ra, Rb, R7, R8, R9, R10, R12, R12a, R12b, R12c, m, X3, W, L, and E are as defined in the specification. Compounds represented by Formula (A) are SMARCA2 protein degraders and are thus useful for treating cancer and other diseases.
A61K 47/55 - 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 the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug, i.e. a dimer, oligomer or polymer of pharmacologically or therapeutically active compounds
31.
3-[3-AMINO-6-(2-HYDROXYPHENYL)PYRIDAZIN-4-YL]-3,8-DIAZABICYCLO[3.2.1]OCTANE DERIVATIVES AS SMARCA2 DEGRADING PROTACS FOR THE TREATMENT OF CANCER
The present disclosure provides compounds represented by Formula (A), or a pharmaceutically acceptable salt and/or stereoisomer thereof: Formula (A), wherein E is: Formula (I), Formula (II), or Formula (III); wherein Ra, Rb, Rc, R7, R8, R9, R10, X3, W, L, and E are as defined in the specification. Compounds represented by Formula (A) are SMARCA2 protein degraders and are thus useful for treating cancer and other diseases.
The specification relates to peptide dendrons comprising one or more residues derived from a modified lysine of formula (I), pharmaceutical delivery systems comprising these peptide dendrons, pharmaceutical compositions containing them, and to their use in therapy.
The specification relates to peptide dendrons comprising one or more residues derived from a modified lysine of formula (I), pharmaceutical delivery systems comprising these peptide dendrons, pharmaceutical compositions containing them, and to their use in therapy.
A61K 47/64 - Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
A61K 47/18 - AminesAmidesUreasQuaternary ammonium compoundsAmino acidsOligopeptides having up to five amino acids
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
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
Provided herein is are methods of treating Chronic Obstructive Pulmonary Disease (COPD) in a patient, comprising administering to the patient an effective amount of benralizumab or an antigen-binding fragment thereof.
C07K 16/24 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
A61P 11/00 - Drugs for disorders of the respiratory system
Pyrrolopyrimidine carboxamides and pharmaceutically acceptable salts thereof; pharmaceutical compositions comprising such compounds and salts; use of such compounds and salts to treat or prevent cancers, including PKMYT1-dependent cancers; kits comprising such compounds and salts; and methods for manufacturing such compounds and salts.
The present disclosure provides methods of inserting a polynucleotide of interest into the genome of a eukaryotic cell, wherein said methods comprise improving the efficiency of CRISPR/Cas-mediated polynucleotide insertion by addition of an inhibitor of the microhomology-mediated end-joining (MMEJ) pathway to the eukaryotic cell. The present disclosure further provides compositions for inserting a polynucleotide of interest into the genome of a eukaryotic cell, and kits for inserting a gene of interest into the genome of a eukaryotic cell.
This disclosure relates to methods for treating a chronic inflammatory disease or disorder in a subject having type 2 diabetes mellitus or being predisposed to type 2 diabetes mellitus. This disclosure also relates to methods for preventing development of diabetes mellitus due to glucocorticoid-induced hyperglycemia or inhibiting progression of preexisting type 2 diabetes mellitus in a subject.
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
A61P 3/10 - Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
A61P 29/00 - Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agentsNon-steroidal antiinflammatory drugs [NSAID]
41.
METHODS, COMPOSITIONS, AND COMBINATIONS FOR THE TREATMENT OF OVARIAN CANCER
The disclosure relates to methods, compositions, and combinations for the treatment of ovarian cancer. Specifically, the disclosure relates to methods of treating ovarian cancer in a subject in need thereof, comprising administering to the subject one or more chemotherapy agents, an anti- VEGF antibody or an antigen-binding fragment, an anti-PD-L1 antibody or an antigen-binding fragment thereof, and optionally a PARP inhibitor. The disclosure also relates to combinations for use in the treatment of ovarian cancer comprising one or more chemotherapy agents, an anti- VEGF antibody or an antigen-binding fragment, an anti-PD-L1 antibody or an antigen-binding fragment thereof, and optionally a PARP inhibitor.
C07K 16/22 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against growth factors
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
The disclosure relates to methods and compositions for the treatment of type I IFN mediated disease. Specifically, the disclosure relates to a subcutaneous dose of a type I IFN receptor inhibitor.
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 39/00 - Medicinal preparations containing antigens or antibodies
A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection
A61P 43/00 - Drugs for specific purposes, not provided for in groups
43.
HALO-SUBSTITUTED PIPERIDINES AS OREXIN RECEPTOR MODULATORS
The present application relates to certain halo-substituted piperidine compounds, pharmaceutical compositions containing them, and methods of using them, including methods for treating substance addiction, panic disorder, anxiety, post-traumatic stress disorder, pain, depression, seasonal affective disorder, an eating disorder, or hypertension.
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
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 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
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
The present specification relates to pharmaceutical compositions comprising Compound [I] ((S)-4- amino-N-(1-(4-chlorophenyl)-3-hydroxypropyl)-1-(7H-pyrrolo[2,3-d]pyrimidin-4-yl)piperidine-4- carboxamide), microcrystalline cellulose (MCC) and dicalcium phosphate anhydrous (DCPA), for example tablets with immediate release properties.
The disclosure relates to methods and compositions for the treatment of SLE. Specifically, the disclosure relates to methods comprising administering to a subject a type I IFN receptor inhibitor.
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
46.
Devices and a System for Detection and Analysis of Inhaler Use
Devices, methods and a system for detection and analysis of inhaler use, in particular breath detection modules and inhaler device counters. An electronic inhaler counter device (100) comprises a rocker arm (102) comprising a proximal end (110) providing a pivot (104) and a distal end (112) providing a head (114), a return spring (108) coupled to the rocker arm pivot, and a count switch (106), wherein, in response to a first selected degree of linear actuation motion, the rocker arm is arranged to perform a first rocker movement and engage the count switch with the rocker head; and in response to further linear actuation motion, the spring is engaged to enable the rocker arm to perform a second rocker movement, such to facilitate over travel. An inhaler breath detection module coupled to an airpath of the inhaler comprises a sensing means configured to provide signals indicative of a change in parameter in the inhaler airpath as a function of time caused by an inhalation breath, and a controller configured to determine the presence of breath, based on a change in parameter in the inhaler airpath as a function of time.
G16H 20/13 - 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 delivered from dispensers
The specification relates to compounds of Formula (I):
The specification relates to compounds of Formula (I):
The specification relates to compounds of Formula (I):
and to pharmaceutically acceptable salts thereof, to processes and intermediates used for their preparation, to pharmaceutical compositions containing them and to their use in the treatment of cell proliferative disorders.
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
A61K 31/497 - Non-condensed pyrazines containing further heterocyclic rings
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
48.
2,4,6-TRISUBSTITUTED 1,3,5-TRIAZINES AS MODULATORS OF CX3CR1
There are disclosed certain 2,4,6-trisubstituted 1,3,5-triazine compounds of Formula (I),
There are disclosed certain 2,4,6-trisubstituted 1,3,5-triazine compounds of Formula (I),
There are disclosed certain 2,4,6-trisubstituted 1,3,5-triazine compounds of Formula (I),
and pharmaceutically acceptable salts thereof, together with compositions containing them and their use in therapy. The compounds are modulators of CX3CR1 and are thereby particularly useful in the treatment or prophylaxis of cardiovascular disorders such as non-ischemic dilated cardiomyopathy and heart failure.
C07D 251/18 - Heterocyclic compounds containing 1,3,5-triazine rings not condensed with other rings having three double bonds between ring members or between ring members and non-ring members with hydrogen or carbon atoms directly attached to at least one ring carbon atom to only one ring carbon atom with nitrogen atoms directly attached to the two other ring carbon atoms, e.g. guanamines
C07D 401/06 - 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 carbon chain containing only aliphatic carbon atoms
C07D 403/06 - 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 carbon chain containing only aliphatic carbon atoms
C07D 405/10 - 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 carbon chain containing aromatic rings
C07D 413/06 - 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 only aliphatic carbon atoms
The disclosure relates to methods, combinations, and uses for treating cervical cancer using an anti-PD-L1 binding protein or an anti-PD-1 binding protein and chemoradiation therapy.
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
Provided are Cas9 effector proteins having enhanced stability. Embodiments of the Cas9 effector proteins have a first nuclear localization signal attached to the N-terminus and a second nuclear localization signal attached to the C-terminus. Also provided are Cas9 systems comprising Cas9 effector proteins having enhanced stability and a guide polynucleotide that forms a complex with the Cas9 effector protein. Further provided are methods for providing site-specific modification of a target sequence in a eukaryotic cell using the Cas9 effector proteins.
The specification generally relates to compounds of Formula (I), and pharmaceutically acceptable salts thereof, where R1, R2A, R2B, R2C, R2D, W, X, Y, and Z have the meanings defined herein. Such compounds are useful in inhibiting NLRP3 inflammasome activity and may be useful as therapeutic agents. The specification also relates to the use of such compounds to treat or prevent diseases and conditions in which the NLRP3 inflammasome is implicated. The specification further relates to compositions comprising such compounds.
The specification generally relates to compounds of Formula (I), and pharmaceutically acceptable salts thereof, where R1, R2A, R2B, R2C, R2D, W, X, Y, and Z have the meanings defined herein. Such compounds are useful in inhibiting NLRP3 inflammasome activity and may be useful as therapeutic agents. The specification also relates to the use of such compounds to treat or prevent diseases and conditions in which the NLRP3 inflammasome is implicated. The specification further relates to compositions comprising such compounds.
Disclosed are methods for treating extensive-stage small-cell lung cancer (ES-SCLC) with an antibody that inhibits PD1/PD-L1 activity in combination with etoposide and a platinum-based therapeutic agent.
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 9/00 - Medicinal preparations characterised by special physical form
A61K 31/555 - Heterocyclic compounds containing heavy metals, e.g. hemin, hematin, melarsoprol
A61K 31/7048 - Compounds having saccharide radicals and heterocyclic rings having oxygen as a ring hetero atom, e.g. leucoglucosan, hesperidin, erythromycin, nystatin
Pseudomonas aeruginosaPseudomonas aeruginosa PcrV protein and Psl exopolysaccharide and that comprises a modified Fc region. Such bispecific antibodies can, for example, have increased half-life and decreased aggregation in the manufacturing process without a decreased potency against Pseudomonas, as compared to bispecific antibodies without the modified Fc region.
There are disclosed certain substituted pyrazine-2-carboxamides of Formula (I), and pharmaceutically acceptable salts thereof, together with compositions containing them and their use in therapy. The compounds are inhibitors of hematopoietic progenitor kinase 1 (HPK1) and are thereby particularly useful in the treatment or prophylaxis of cancer.
A61K 31/5377 - 1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
A61K 31/497 - Non-condensed pyrazines containing further heterocyclic rings
A61K 31/541 - Non-condensed thiazines containing further heterocyclic rings
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 403/04 - 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 directly linked by a ring-member-to-ring- member bond
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 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
This specification relates to compounds according to Formula (I), which are of PROTACs of IRAK4, to pharmaceutical compositions containing them, and to their use in therapy for the treatment of IRAK4-mediated and/or IRAK-4 directed diseases and disorders.
This specification relates to compounds according to Formula (I), which are of PROTACs of IRAK4, to pharmaceutical compositions containing them, and to their use in therapy for the treatment of IRAK4-mediated and/or IRAK-4 directed diseases and disorders.
A61K 31/5025 - PyridazinesHydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
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 31/496 - Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
A61K 31/513 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim having oxo groups directly attached to the heterocyclic ring, e.g. cytosine
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
The specification relates to therapeutic combinations of capivasertib, a CDK4/6 inhibitor (such as palbociclib, ribociclib or abemaciclib) and fulvestrant that are useful for treating breast cancer, and to methods of treating breast cancer patients with combinations of capivasertib, a CDK4/6 inhibitor (such as palbociclib, ribociclib or abemaciclib) and fulvestrant.
A61K 31/506 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
A61K 31/519 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
A61K 31/565 - Compounds containing cyclopenta[a]hydrophenanthrene ring systemsDerivatives thereof, e.g. steroids not substituted in position 17 beta by a carbon atom, e.g. oestrane, oestradiol
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
A method of treating cancer in a subject in need thereof, comprising administering to the subject a first amount of a selective PARP1 inhibitor or a pharmaceutically acceptable salt thereof, and a second amount of a selective estrogen degrader or a pharmaceutically acceptable salt thereof, wherein the first amount and the second amount together comprise a therapeutically effective amount.
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
A61K 9/00 - Medicinal preparations characterised by special physical form
A61K 31/496 - Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
A61K 31/519 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
A61K 31/565 - Compounds containing cyclopenta[a]hydrophenanthrene ring systemsDerivatives thereof, e.g. steroids not substituted in position 17 beta by a carbon atom, e.g. oestrane, oestradiol
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
The application relates to methods of treating chronic kidney disease in a patient in need thereof involving administering an inhibitor of ARGHEF6 activity such as an antisense oligonucleotide. Use of ARHGEF6 inhibitors in medicine and in the manufacture of medicines are also disclosed.
The specification relates to conjugates comprising a linker of Formula (IA):
The specification relates to conjugates comprising a linker of Formula (IA):
The specification relates to conjugates comprising a linker of Formula (IA):
and pharmaceutically acceptable salts thereof. The specification also relates the use of the conjugates for the treatment of diseases such as cancer, and intermediates useful for the synthesis of the conjugates.
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
The specification relates to conjugates comprising a linker of Formula (ICA):
and pharmaceutically acceptable salts thereof. The specification also relates the use of the conjugates for the treatment of diseases such as cancer, and intermediates useful for the synthesis of the 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
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
The specification generally relates to compounds of Formula (I):
The specification generally relates to compounds of Formula (I):
The specification generally relates to compounds of Formula (I):
or a stereoisomer or pharmaceutically salt thereof, wherein G, Ga, Gb, X, Y, R1, R2, Q1, Q2, and Q3 have any of the meanings defined herein, together with compositions containing them and their use in therapy. The compounds are inhibitors of the polymerase, DNA polymerase theta (Polθ or POLQ), and are thereby particularly useful in the treatment of cancer.
The present disclosure relates to the endothelin receptor antagonist (ERA) zibotentan in combination with the sodium-dependent glucose cotransporter 2 (SGLT-2) inhibitor dapagliflozin for use in the treatment of certain endothelin related diseases.
A61K 31/497 - Non-condensed pyrazines containing further heterocyclic rings
A61K 31/7034 - Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin
A61P 13/12 - Drugs for disorders of the urinary system of the kidneys
The disclosure relates to methods and compositions for the treatment of type I IFN mediated disease. Specifically, the disclosure relates to a subcutaneous dose of a type I IFN receptor inhibitor.
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 39/00 - Medicinal preparations containing antigens or antibodies
A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection
A61P 43/00 - Drugs for specific purposes, not provided for in groups
The present disclosure relates to antibody molecules that bind epidermal growth factor receptor (EGFR) and/or c-Met and conjugates containing these antibody molecules. The antibody molecules and conjugates find application in the treatment of cancer, for example.
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 39/00 - Medicinal preparations containing antigens or antibodies
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
A61P 1/18 - Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
The specification relates to conjugates comprising a linker of Formula (ICA) and pharmaceutically acceptable salts thereof. The specification also relates the use of the conjugates for the treatment of diseases such as cancer, and intermediates useful for the synthesis of the conjugates.
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
This disclosure relates to compositions and methods for treating disease using chimeric antigen receptor cells and/or antigen binding domains targeting BCMA.
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
The specification relates to conjugates comprising a linker of Formula (IA): and pharmaceutically acceptable salts thereof. The specification also relates the use of the conjugates for the treatment of diseases such as cancer, and intermediates useful for the synthesis of the conjugates.
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
The present provides a method of treating cancer in a subject, the method comprising administering to the subject: (a) a KRAS inhibitor, and (b) an Ataxia Telangiectasia and Rad-3-related (ATR) inhibitor. Also disclosed are compositions and kits comprising (a) a KRAS inhibitor, and (b) an ATR inhibitor.
A61K 31/519 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
A61K 31/5377 - 1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
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 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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 39/00 - Medicinal preparations containing antigens or antibodies
There are disclosed certain 1-[2-(aminomethyl)benzyl]-2-thioxo-1,2,3,5-tetrahydro-4H-pyrrolo[3,2-d]pyrimidin-4-one compounds of formula (I),
There are disclosed certain 1-[2-(aminomethyl)benzyl]-2-thioxo-1,2,3,5-tetrahydro-4H-pyrrolo[3,2-d]pyrimidin-4-one compounds of formula (I),
There are disclosed certain 1-[2-(aminomethyl)benzyl]-2-thioxo-1,2,3,5-tetrahydro-4H-pyrrolo[3,2-d]pyrimidin-4-one compounds of formula (I),
and pharmaceutically acceptable salts thereof, together with compositions containing them and their use in therapy. The compounds are inhibitors of the enzyme MPO and are thereby particularly useful in the treatment or prophylaxis of cardiovascular disorders such as heart failure and coronary artery disease related conditions.
The present disclosure relates to certain (2S)—N-[(1S)-1-cyano-2-phenylethyl]-1,4-oxazepane-2-carboxamide compounds (including pharmaceutically acceptable salts thereof),
The present disclosure relates to certain (2S)—N-[(1S)-1-cyano-2-phenylethyl]-1,4-oxazepane-2-carboxamide compounds (including pharmaceutically acceptable salts thereof),
The present disclosure relates to certain (2S)—N-[(1S)-1-cyano-2-phenylethyl]-1,4-oxazepane-2-carboxamide compounds (including pharmaceutically acceptable salts thereof),
that inhibit dipeptidyl peptidase 1 (DPP1) activity, to their utility in treating and/or preventing clinical conditions including respiratory diseases, such as asthma and chronic obstructive pulmonary disease (COPD), to their use in therapy, to pharmaceutical compositions containing them and to processes for preparing such compounds.
C07D 267/10 - Seven-membered rings having the hetero atoms in positions 1 and 4 not condensed with other rings
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 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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
This specification relates to compounds according to Formula (I), which are of PROTACs of IRAK4, to pharmaceutical compositions containing them, and to their use in therapy for the treatment of IRAK4-mediated and/or IRAK-4 directed diseases and disorders (IA)
A61P 37/00 - Drugs for immunological or allergic disorders
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 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
A61K 31/513 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim having oxo groups directly attached to the heterocyclic ring, e.g. cytosine
A61K 31/5025 - PyridazinesHydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
A61K 31/496 - Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
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
78.
FLUIDIC MIXER UNIT DEVICE FOR NANOPARTICLE PRODUCTION
A device 100 for producing nanoparticles includes a microplate 102 which includes a plurality of fluidic mixing units 104 arranged in an array. Each fluidic mixing unit is configured to produce nanoparticles. The device enables high throughput lipid nanoparticle testing and development, and is configured to generate large numbers of novel or unique nanoparticle formulations.
B01F 25/10 - Mixing by creating a vortex flow, e.g. by tangential introduction of flow components
B01F 25/433 - Mixing tubes wherein the shape of the tube influences the mixing, e.g. mixing tubes with varying cross-section or provided with inwardly extending profiles
The present disclosure relates to methods of identifying subjects likely to respond to treatment with a myeloperoxidase (MPO) inhibitor based on their neutrophil levels and/or the concentration of at least one protein biomarker. The present disclosure also relates to methods of selecting subjects for treatment with an MPO inhibitor, methods of predicting whether a subject will respond to treatment with an MPO inhibitor and methods of treating subjects likely to respond to treatment with an MPO inhibitor. The present disclosure also relates to a method of predicting an improved clinical response in a subject having an MPO-related disorder following treatment with an MPO inhibitor.
A method of treatment or prophylaxis of diseases or conditions in which inhibition of PARP1 is beneficial, comprising administering to the subject AZD5305 in a daily dose of 10 to 140 mg, wherein AZD5305 may be in the form of a pharmaceutically acceptable salt.
The disclosure relates to methods and compositions for the treatment of Systemic Lupus Erythematosus (SLE). Specifically, the disclosure relates to methods comprising administering to a subject a type I IFN receptor inhibitor.
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 39/00 - Medicinal preparations containing antigens or antibodies
A61P 7/00 - Drugs for disorders of the blood or the extracellular fluid
C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids
A method of treatment or prophylaxis of diseases or conditions in which inhibition of PARP1 is beneficial, comprising administering to the subject AZD5305 in a daily dose of 10 to 140 mg, wherein AZD5305 may be in the form of a pharmaceutically acceptable salt.
The present disclosure provides methods of inserting a polynucleotide of interest into the genome of a eukaryotic cell, wherein said methods comprise improving the efficiency of CRISPR/Cas-mediated polynucleotide insertion by addition of an inhibitor of the microhomology- mediated end-joining (MMEJ) pathway to the eukaryotic cell. The present disclosure further provides compositions for inserting a polynucleotide of interest into the genome of a eukaryotic cell, and kits for inserting a gene of interest into the genome of a eukaryotic cell.
The present application relates to novel compounds of formula (I)
The present application relates to novel compounds of formula (I)
The present application relates to novel compounds of formula (I)
to their utility in treating and/or preventing clinical conditions including cardiovascular diseases (CVD), to methods for their therapeutic use, to pharmaceutical compositions containing them and to processes for preparing such compounds.
A61K 31/4155 - 1,2-Diazoles not condensed and containing further heterocyclic rings
A61K 31/4985 - Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems
A61P 9/00 - Drugs for disorders of the cardiovascular system
A61P 9/10 - Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
A61P 11/00 - Drugs for disorders of the respiratory system
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 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
The disclosure relates to methods, cells, and compositions for preparing cell populations and compositions for adoptive cell therapy. In particular, provided herein are methods for expansion and proliferation of primary immune cells including T cell populations.
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
Methods for treating high proteinuria chronic kidney disease and/or at least one disease, disorder, or condition associated therewith in patients by the use of a fixed-dose combination of zibotentan and dapagliflozin are disclosed.
A61K 31/497 - Non-condensed pyrazines containing further heterocyclic rings
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
The present specification relates to a crystalline form of (3S)-2-[(5-amino-6-fluoro-1H- pyrrolo[3,2-b]pyridin-2-yl)methyl]-1'-but-2-ynyl-6-fluoro-spiro[isoindoline-3,3'-pyrrolidine]-1,2'- dione, and to compositions and uses thereof.
A compound of Formula (I):or a pharmaceutically acceptable salt thereof, wherein: X1is CH or N; p is 0, 1 or 2; where: each R11-31-31-31-31-3alkoxy may be independently optionally substituted by one or more F; RNis selected from H and Me; n is 0, 1 or 2; m is 0 or 1; Q1is CH or N; when n & m are both other than 0, Q2is CH or N; when n & m are both 0, Q2is CH; when n is 0 or 1, Q3is CH; when n is 2 and Q2is N, Q3is CH; when n is 2 and Q2is CH, Q3is CH or O; R2aand R2bare substituents on the same or different C atoms other than at Q1or Q21-31-3alkyl, or R2a& R2b2rr- group where r is 1, 2 or 3; Q4is a single bond or -NR4C(=O); R4is H or Me; 0, 1 or 2 of Y1, Y2, Y3, Y4& Y5is/are N, and are otherwise C; each R3is a substituent on any C atom at Y1, Y2, Y3, Y4& Y51-31-31-31-31-3 alkoxy may be independently optionally substituted by one or more F; q is 0, 1 or 2; wherein Linker is attached at any available C atom at Y4& Y5; Linker is a saturated or a partially or fully unsaturated framework comprising C and H atoms and at least one heteroatom, wherein said framework has end points of attachment 'a' and 'b' and a length of from 5 to 26 atoms between 'a' and 'b'; wherein said framework may include one or more straight and/or branched chains and/or rings and is optionally substituted on any available C atom(s) by one or more F; and W is an E3 ubiquitin ligase cereblon binder unit.
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
The present disclosure relates to pharmaceutical formulations comprising heterodimeric Relaxin fusion polypeptides, in particular heterodimeric Relaxin 2 fusion polypeptides and uses thereof. Thus, the disclosure provides pharmaceutical formulations comprising Relaxin fusion polypeptides and kits comprising the same and uses of the same including methods of treatment. The pharmaceutical formulations of the disclosure may be useful, in particular, in the treatment of cardiovascular diseases, for example for the treatment of heart failure.
The present application relates to chemical compounds of Formula (1), and pharmaceutically acceptable salts thereof, that inhibit IRAK4 and consequently have potential utility in medicine.
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
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 31/501 - PyridazinesHydrogenated pyridazines not condensed and containing further heterocyclic rings
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 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 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
96.
CONTROL OF MULTI-GENE EXPRESSION USING SYNTHETIC PROMOTERS
The invention relates to expression vectors comprising mammalian synthetic promoters that can mediate expression of multiple genes at predictable relative stoichiometries.
A61K 47/64 - Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
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
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
The disclosure relates to methods, cells, and compositions for preparing cell populations and compositions for adoptive cell therapy. In particular, provided herein are methods for expansion and proliferation of primary immune cells including T cell populations.
This disclosure relates to compositions and methods for treating disease using chimeric antigen receptor cells and/or antigen binding domains targeting BCMA.
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
The invention provides a method for treating or preventing an a-synucleinopathy in a subject in need thereof, the method comprising administering to the subject a fixed dose of 50-5,000 mg of an anti-α-synuclein antibody, or antigen-binding fragment thereof. Also provided are corresponding compositions and kits.
