Described herein are molecular glue degrader compounds that bind to both a target protein and a RING E3 Ubiquitin Ligase, as well as related compositions and methods of use, e.g., for degradation of the target protein and/or the treatment of a disease, disorder, or condition.
A61K 31/551 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole having two nitrogens as ring hetero atoms, e.g. clozapine, dilazep
A61K 31/519 - Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
123455, y, R, M, W, L, V, T, Y, J, K and A are as described herein, therapeutic uses of said compounds, uses of said compounds as research chemicals, a pharmaceutical composition and combinations comprising said compounds, and methods for manufacturing the compounds of the invention.
C07D 487/12 - Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups in which the condensed system contains three hetero rings
A61K 31/519 - Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
A61P 35/02 - Antineoplastic agents specific for leukemia
3.
METHODS OF TREATING ESTROGEN RECEPTOR-MEDIATED DISORDERS
The present disclosure provides methods for treating an estrogen receptor mediated disease, disorder, or condition in a subject comprising administering to the subject a composition comprising Compound 1 or a pharmaceutically acceptable salt thereof in combination with ribociclib, or a pharmaceutically acceptable salt thereof.
A61K 31/395 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
A61K 31/435 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
A61K 31/397 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having four-membered rings, e.g. azetidine
A61K 31/4353 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
A61K 31/437 - 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
The present disclosure provides methods for treating an estrogen receptor mediated disease, disorder, or condition in a subject comprising administering to the subject a composition comprising Compound (1) or a pharmaceutically acceptable salt thereof in combination with alpelisib, or a pharmaceutically acceptable salt thereof.
A61K 31/395 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
A61K 31/435 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
A61K 31/397 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having four-membered rings, e.g. azetidine
A61K 31/4353 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
A61K 31/437 - 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
This invention relates to novel processes for synthesizing N-(3-(6-Amino-5-(2-(N- methylacrylamido)ethoxy)pyrimidin-4-yl)-5-fluoro-2-methylphenyl)-4-cyclopropyl-2-fluorobenzamide and to intermediates which are used in such processes.
C07C 231/02 - Preparation of carboxylic acid amides from carboxylic acids or from esters, anhydrides, or halides thereof by reaction with ammonia or amines
C07C 233/59 - Carboxylic acid amides having carbon atoms of carboxamide groups bound to carbon atoms of rings other than six-membered aromatic rings having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by halogen atoms or by nitro or nitroso groups
The invention provides methods of making immune effector cells (for example, T cells, NK cells) that express a chimeric antigen receptor (CAR), and compositions generated by such methods, and therapeutic uses thereof for treating autoimmune diseases or disorders.
A61K 39/00 - Medicinal preparations containing antigens or 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
A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection
The instant disclosure relates to compositions comprising SARS-CoV-2 binding molecules and methods of use thereof. Provided herein are dosing regimens for administration of SARS-CoV-2 binding molecules, including the DARPin® protein ensovibep.
The present invention relates to the field of pharmacy, particularly to a pharmaceutical composition comprising N (3-(2-(2-hydroxyethoxy)-6-morpholinopyridin-4- yl)-4-methylphenyl)-2-(trifluoromethyl)isonicotinamide, or a pharmaceutically acceptable salt thereof. The present invention also provides a process for preparing said pharmaceutical compositions for oral administration and methods of treatment with said pharmaceutical compositions.
The present invention relates to novel pyridazin-3-yl phenol compounds of formula (I): wherein R1, R2, R3, R4and R5 are defined herein, which inhibit NOD-like receptor protein 3 (NLRP3) inflammasome activity. The invention further relates to the processes for their preparation, pharmaceutical compositions and medicaments containing them, and their use in the treatment of diseases and disorders mediated by NLRP3.
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
The present disclosure relates to the field of pharmacy, particularly to a NLRP3 inhibitor for use in the treatment of an auto-inflammatory syndrome. The disclosure also relates to a NLRP3 inhibitor or a pharmaceutical combination comprising a NLRP3 inhibitor, or a pharmaceutically acceptable salt thereof, and at least one further therapeutic agent, for use in the treatment of an auto-inflammatory syndrome; to a method for the treatment of an auto-inflammatory syndrome that involves administering a NLRP3 inhibitor or the combination; and to the use of a NLRP3 inhibitor or the combination for the manufacture of a medicament for the treatment of an auto- inflammatory syndromes. In particular N'-((1,2,3,5,6,7-hexahydro- s-indacen-4-yl)carbamoyl)-2-(2-hydroxypropan-2-yl)thiazole-5- sulfonimidamide and enantiomers thereof are used for treating auto-inflammatory syndrome, in particular cryopyrin-associated periodic syndromes (CAPS), familial cold auto-inflammatory syndrome (FCAS), Muckle Wells syndrome (MWS), neonatal onset multisystem inflammatory disease / chronic, infantile, neurological, cutaneous and articular syndrome (NOMID/CINCA), or Familial Mediterranean Fever (FMF).
The present invention pertains to the use for treating organ injury, in particular acute organ injury, such as acute kidney injury (AKI) using CD39, human recombinant CD39 and variants thereof.
The present invention pertains to the use of miRNA technology for improving recombinant production of CD39 or variants thereof in CHO cells. The miRNA is used for knockdown of the endogenous protein CCL2 of the CHO cells which is difficult to separate from CD39 or variants thereof during purification.
The present invention relates to methods of identifying a subject for treatment with or treating a subject with a tricyclic AKR1C3 dependent KARS inhibitor of formula (I), or a pharmaceutically acceptable salt thereof. The methods may comprise determining in a subject sample a level of at least one of the following biomarkers: AKR1C3, NFE2L2, KEAP1, or CUL3, wherein an elevated level of the biomarker identifies the subject as being in need of treatment; or detecting in a subject sample a somatic mutation in at least one of the following genes: NFE2L2, KEAP1, or CUL3, wherein detecting the somatic mutation identifies the subject as being in need of treatment.
The present invention relates to solid phase pharmaceutical compositions of 6'-fluoro-N-(4-fluorobenzyl)-4'-oxo-3',4'-dihydro-1'H-spiro[piperidine-4,2'-quinoline]-1-carboxamide that is useful as a AKR1C3 dependent KARS inhibitor. The present invention also relates to processes for the preparation of said pharmaceutical compositions of said compound, methods of using said pharmaceutical compositions in the treatment of various diseases and disorders, and their use in diseases and disorders mediated by an AKR1C3 dependent KARS inhibitor.
A61K 31/438 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom the ring being spiro-condensed with carbocyclic or heterocyclic ring systems
15.
CRYSTALLINE FORMS OF AN AKR1C3 DEPENDENT KARS INHIBITOR
A61K 31/438 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom the ring being spiro-condensed with carbocyclic or heterocyclic ring systems
16.
TREATMENT OF AIHA WITH BAFF OR BAFF RECEPTOR INHIBITORY ANTIBODIES
The disclosure relates to a molecule that inhibits BAFF pathway, particularly an antibody or a binding fragment thereof, particularly an anti-BAFF-R antibody, e.g. ianalumab, for use in the treatment of autoimmune hemolytic anemia (AIHA), in particular, warm autoimmune hemolytic anemia (wAIHA).
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
Systems and processes for guiding a user through the initial use and general use of an injection device are described. In some embodiments, a user's biometric characteristics are considered prior to recommending proceeding with initiating an injection process. In some embodiments, an injection device with one or more sensors is used to help identify issues with the manner a user is performing an injection process.
A61M 5/32 - Syringes - Details - Details of needles pertaining to their connection with syringe or hub; Accessories for bringing the needle into, or holding the needle on, the body; Devices for protection of needles
G16H 20/17 - 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 via infusion or injection
G16H 40/63 - ICT specially adapted for the management or administration of healthcare resources or facilities; ICT specially adapted for the management or operation of medical equipment or devices for the operation of medical equipment or devices for local operation
G16H 40/67 - ICT specially adapted for the management or administration of healthcare resources or facilities; ICT specially adapted for the management or operation of medical equipment or devices for the operation of medical equipment or devices for remote operation
A61M 5/20 - Automatic syringes, e.g. with automatically actuated piston rod, with automatic needle injection, filling automatically
A61B 5/00 - Measuring for diagnostic purposes ; Identification of persons
G09B 23/28 - Models for scientific, medical, or mathematical purposes, e.g. full-sized device for demonstration purposes for medicine
An autoinjector kit, the autoinjector kit comprising an autoinjector device comprising: a longitudinal housing extending along a longitudinal axis and having an open proximal end to contact a trigger site, a distal end opposite to the proximal end and a hollow interior; a barrel body mounted inside the housing; a stopper slidably mounted within the barrel body and movable from a distal position to a proximal position by a plunger rod; a drive mechanism connected to the plunger rod, the drive mechanism having a primed state in which the plunger rod is held against a driving force in a retracted position between the distal end and the stopper in the distal position and a fired state in which the plunger rod extends through at least a portion of the barrel body and contacts the stopper in the proximal position; a trigger mechanism operable to release the plunger rod and initiate a transition of the drive mechanism from the primed state to the fired state to thereby cause the plunger rod to drive the stopper from the distal position to the proximal position; an indicator component which, when triggered, moves distally from an initial position to an indication position to indicate a state of the autoinjector device; wherein the autoinjector kit comprises an indicator reset and the housing of the autoinjector device comprises a reset access opening in the distal end of the longitudinal housing, the indicator reset comprising a base and a reset projection, the reset projection insertable through the reset access opening to contact the indicator component and force the indicator component into the initial position.
G09B 23/28 - Models for scientific, medical, or mathematical purposes, e.g. full-sized device for demonstration purposes for medicine
A61M 5/20 - Automatic syringes, e.g. with automatically actuated piston rod, with automatic needle injection, filling automatically
A61M 5/315 - Pistons; Piston-rods; Guiding, blocking or restricting the movement of the rod; Appliances on the rod for facilitating dosing
A61M 5/32 - Syringes - Details - Details of needles pertaining to their connection with syringe or hub; Accessories for bringing the needle into, or holding the needle on, the body; Devices for protection of needles
19.
S)-ENGINEERED OXYNITRILASE POLYPEPTIDES AND USES THEREOF
The present disclosure relates to a process for producing chiral β-nitro alcohol compounds. The invention relates in particular to an (S)-selective oxynitrilase, which enantioselectively can catalyze the Henry reaction, wherein an aldehyde or ketone compound is converted to the corresponding β-nitro alcohol compound in the presence of a nitroalkane compound and an oxynitrilase.
The disclosure relates to a subcutaneous dosage of Compound (I), or a pharmaceutically acceptable salt thereof, for the treatment of diseases or disorders mediated by negative allosteric modulation or inhibition of NR2B-containing NMDA receptor, including, but not limited to, depression disorder, including, major depressive disorder, treatment resistant depression, suicidality in major depressive disorder, major depressive disorder with suicidal ideation, major depressive disorder with suicidal ideation and suicidal intent, suicidality, and seasonal affective disorder. The disclosure also relates to the use of Compound (I), or a pharmaceutically acceptable salt thereof, in the treatment of treatment resistant depression in patients with major depressive disorder.
A61K 31/4439 - Non-condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
The present disclosure relates to antibody-drug conjugates (ADCs) comprising an antibody or an antigen-binding fragment thereof covalently linked to two pharmaceutically active drugs through a dual linker. Linker-drug conjugates comprising the dual linker and the pharmaceutically active drugs are also disclosed. Such linkers are a convenient way of delivering two (e.g. two different or two of the same) drugs connected to a single antibody. Such linkers may be particularly useful in improving the solubility of antibody drug conjugates (ADCs) which comprise one or more hydrophobic drug compounds.
A61K 47/68 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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 relates to methods for treating giant cell arteritis (GCA), using IL-17 antagonists, e.g., secukinumab. Also disclosed herein are uses of IL-17 antagonists, e.g., IL-17 antibodies, such as secukinumab, for treating GCA patients, as well as medicaments, dosing regimens, pharmaceutical formulations, dosage forms, and kits for use in the disclosed uses and methods.
A61P 9/00 - Drugs for disorders of the cardiovascular system
C07K 16/24 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
23.
METHODS OF SELECTIVELY TREATING TENDINOPATHY USING INTERLEUKIN-17 (IL-17) ANTAGONISTS
The present disclosure relates to methods for treating tendinopathy, e.g., rotator cuff tendinopathy, using IL-17 antagonists, e.g., secukinumab. Also disclosed herein are uses of IL-17 antagonists, e.g., IL-17 antibodies, such as secukinumab, for treating tendinopathy patients, as well as medicaments, dosing regimens, pharmaceutical formulations, dosage forms, and kits for use in the disclosed uses and methods.
A61K 39/395 - Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
A61P 19/02 - Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
A61P 19/04 - Drugs for skeletal disorders for non-specific disorders of the connective tissue
A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
A61P 29/00 - Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
A61P 37/00 - Drugs for immunological or allergic disorders
A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection
C07K 16/24 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
24.
PIPERIDINYL-METHYL-PURINE AMINE FUMARIC ACID SALTS, CRYSTALLINE FORMS, AND THEIR USE IN TREATING MEDICAL DISEASES AND CONDITIONS
The invention provides piperidinyl-methyl-purine amine fumaric acid salts, crystalline forms, pharmaceutical compositions, their use in inhibiting NSD2, and their use in the treatment of a disease or condition, such as cancer.
The invention provides piperidinyl-methyl-purine amine salts, crystalline forms, pharmaceutical compositions, their use in inhibiting NSD2, and their use in the treatment of a disease or condition, such as cancer.
e.ge.g., the antigen expressed on a tumor or other cancer cells. The disclosure further relates to methods and compositions for use in the treatment of cancers by administering the antibody-drug conjugates provided herein. Linker-drug conjugates comprising Bcl-xL inhibitor drug moiety and methods of making the same are also disclosed.
A61K 47/68 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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
27.
IMPROVED PROTEIN PRODUCTION USING miRNA TECHNOLOGY
The present invention pertains to the use of miRNA technology for improving recombinant production of polypeptides of interest in host cells. Expression cassettes are provided which produce a miRNA targeting and down-regulating a host cell protein which interferes with production of the polypeptide of interest.
Anti-Met antibody-drug conjugates that bind to human oncology targets are disclosed. The antibody-drug conjugates comprise a Bcl-xL inhibitor drug moiety and an anti-Met antibody or antigen-binding fragment thereof that binds the antigen target, e.g., the antigen expressed on a tumor or other cancer cells. The disclosure further relates to methods and compositions for use in the treatment of cancers by administering the antibody¬ drug conjugates provided herein. Linker-drug conjugates comprising Bcl-xL inhibitor drug moiety and methods of making same are also disclosed.
A61K 47/68 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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
29.
ANTIBODY-DRUG CONJUGATES OF ANTINEOPLASTIC COMPOUNDS AND METHODS OF USE THEREOF
Antibody-drug conjugates that bind to human oncology targets are disclosed. The antibody-drug conjugates comprise an antibody or an antigen-binding fragment thereof covalently linked to two antineoplastic payloads through a dual linker, wherein at least one antineoplastic payload is a BH3 mimetic. The disclosure further relates to methods and compositions for use in the treatment of cancers by administering the antibody-drug conjugates provided herein. Linker-drug conjugates comprising at least one BH3 mimetic and methods of making the same are also disclosed.
A61K 47/68 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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 adeno-associated virus (AAV) VP2 fusion polypeptides comprising an AAV VP2 capsid polypeptide and a polypeptide ligand. The disclosure further relates to rAAV virions comprising such AAV VP2 fusion polypeptides and libraries of nucleic acids encoding such AAV VP2 fusion polypeptides, pharmaceutical compositions comprising such rAAV virions, and related methods and uses.
C12Q 1/6897 - Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids involving reporter genes operably linked to promoters
C12Q 1/70 - Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving virus or bacteriophage
31.
PYRAZOLOPYRIMIDINE DERIVATIVES AND USES THEREOF AS TET2 INHIBITORS
The present invention relates to novel pyrazolopyrimidine compounds that are TET2 inhibitors, processes for their preparation, pharmaceutical compositions, and medicaments containing them, and their use in diseases and disorders mediated by a TET2 inhibitor.
Handheld dispensing system devices are disclosed. Aspects of the dispensing devices include a first pathway for directing the liquid from an ampoule to a chamber having an aperture through which the liquid from the ampoule can be dispensed. The device includes an actuator to oscillate a membrane to dispense the liquid through the membrane. The device further includes a second pathway in fluid communication with the interior of the ampoule and the atmosphere to equalize the pressure in the ampoule as liquid is dispensed. The second pathway can be sufficiently designed to prevent liquid from escaping through the second pathway.
B05B 1/08 - Nozzles, spray heads or other outlets, with or without auxiliary devices such as valves, heating means designed to produce a jet, spray, or other discharge of particular shape or nature, e.g. in single drops of pulsating nature, e.g. delivering liquid in successive separate quantities
A61M 11/00 - Sprayers or atomisers specially adapted for therapeutic purposes
A61F 9/00 - Methods or devices for treatment of the eyes; Devices for putting in contact-lenses; Devices to correct squinting; Apparatus to guide the blind; Protective devices for the eyes, carried on the body or in the hand
A61J 1/14 - Containers specially adapted for medical or pharmaceutical purposes - Details; Accessories therefor
33.
MULTISPECIFIC ANTIBODIES TARGETING IL-13 AND IL-18
Described herein are multispecific antibodies targeting IL-13 and IL-18. The multispecific antibodies can be antagonistic and/or therapeutic antibodies targeting IL-13 and IL-18. Also described herein are methods of making said multispecific antibodies, methods of inhibiting IL- 13 and IL-18 simultaneously with said multispecific antibodies, and methods of treating an IL- 13/IL-18 mediated disorder, such as atopic dermatitis, by administering a multispecific antibody described herein.
C07K 16/24 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
C07D 403/12 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group containing two hetero rings linked by a chain containing hetero atoms as chain links
A61K 31/4439 - Non-condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
35.
DOSAGE REGIMENS FOR ANTI-CD19 AGENTS AND USES THEREOF
The present disclosure relates to dosage regimes of anti-CD19 agents, in particular an anti-CD19 x anti-CD3 x anti-CD2 trispecific agent administered intravenously (i.v.) and subcutaneously (s.c.), and their use for treating diseases and disorders associated with expression of CD19 such as B cell malignancies, in particular relapsed and/or refractory B-cell malignancies.
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 present invention provides a KRAS G12C inhibitor for use in a method of treatment of a cancer or solid tumor such as NSCLC which harbors a KRAS G12C mutation and a PD-L1 expression <1% regardless of STK11 mutation status. The present invention also provides a KRAS G12C inhibitor for use in a method of treatment of a cancer or solid tumor such as NSCLC which harbors a KRAS G12C mutation and a PD-L1 expression <1% regardless of STK11 mutation status or a cancer or solid tumor such as NSCLC which harbors a KRAS G12C mutation and a PD-L1 expression ≥1% and a STK11 co-mutation.
Provided herein are compounds of formula (I) and pharmaceutical compositions thereof useful for treating diseases or disorders mediated by the complement factor B.
C07D 209/18 - 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 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 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 403/06 - 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 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
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 31/41 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which is nitrogen, e.g. tetrazole
A61K 31/55 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
The present disclosure features compounds (e.g., oxaziridine-based compounds), as well as related compositions and methods of use thereof, e.g., for selectively labeling a methionine residue in a target peptide or protein.
C07D 273/01 - Heterocyclic compounds containing rings having nitrogen and oxygen atoms as the only ring hetero atoms, not provided for by groups having one nitrogen atom
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 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
Described herein are methods of using an anti-natriuretic peptide receptor 1 (NPR1) antibody or antigen binding fragment thereof for the treatment or prevention of a disease or disorder in a human, in particular for a disorder or a disease associated with natriuretic peptide receptor activity (including, but not limited to heart failure, e.g., HFrEF, HFmrEF, or HFpEF; or hypertension, e.g., resistant hypertension).
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
Membrane actuated hydrodynamic dispensing system devices are disclosed. Aspects of the dispensing devices include an ampoule assembly and an actuator. The ampoule assembly contains liquid to be dispensed and includes the actuated membrane, one or more flexible apertures, and a stationary member to seal the aperture. The actuator is configured to vibrate the membrane to hydrodynamic excite the fluid contained in the ampoule assembly. Once the fluid is hydrodynamically excited sufficiently, the liquid is dispensed out of the aperture.
A61F 9/00 - Methods or devices for treatment of the eyes; Devices for putting in contact-lenses; Devices to correct squinting; Apparatus to guide the blind; Protective devices for the eyes, carried on the body or in the hand
F16F 9/32 - Springs, vibration-dampers, shock-absorbers, or similarly-constructed movement-dampers using a fluid or the equivalent as damping medium - Details
The present invention relates to pyrazine amide derivative compounds, such as those according to formula (I) and compositions including said compounds, The invention also provides such pyrazine amide derivative compounds for use in the treatment of plasmodium related diseases such as malaria.
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 407/14 - 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 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
The invention relates to methods for treating neovascular age-related macular degeneration (nAMD) in a patient, the methods comprising (a) administering to the patient as a loading phase of two individual doses of a VEGF antagonist at 6-week interval (q6w regimen), (b) assessing the patient for disease activity after the second dose of the loading phase, and (c) optionally if presence of disease activity is identified after the second dose of the VEGF antagonist, the method further comprises administering to the patient as part of the loading phase a third dose of the VEGF antagonist 6 weeks after the second dose.
The present disclosure relates to improved processes to recover acetonitrile from aqueous and organic waste streams, particularly those generated during oligonucleotide manufacturing processes.
B01J 3/00 - Processes of utilising sub-atmospheric or super-atmospheric pressure to effect chemical or physical change of matter; Apparatus therefor
C07H 21/00 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
The invention provides a RAF inhibitor and a MEK inhibitor for combined use in the treatment of a solid tumor, and is based on identifying certain RAS mutations, a method for identifying a patient suffering from a solid tumor for amenability to combined treatment with a RAF inhibitor and a MEK inhibitor, a corresponding method of treatment and related invention aspects or embodiments as described in detail below. The RAS mutations are in codon Q61 or a G13R mutation.
C12Q 1/6886 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material for cancer
45.
USE OF IPTACOPAN FOR THE TREATMENT OF LUPUS NEPHRITIS
Described herein are methods of treating lupus nephritis with the Factor B inhibitor iptacopan or a pharmaceutically acceptable salt thereof, e.g. iptacopan hydrochloride.
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 45/00 - Medicinal preparations containing active ingredients not provided for in groups
A61P 37/00 - Drugs for immunological or allergic disorders
46.
REMIBRUTINIB FOR USE IN THE TREATMENT OF HIDRADENITIS SUPPURATIVA
The present disclosure relates to methods for treating and/or preventing Hidradenitis Suppurativa in a subject having such disease or condition comprising administering a therapeutically effective dose of LOU064. Also disclosed are medicaments, dosing regimens, pharmaceutical compositions, combinations, dosage forms, and kits for use in the disclosed uses and methods.
The invention relates to a pharmaceutical formulation comprising the Active Pharmaceutical Ingredient (3S,4S)-8-(6-amino-5-((2-amino-3-chloropyridin-4-yl)thio)pyrazin-2-yl)-3-methyl-2- oxa-8-azaspiro[4.5]decan-4-amine, or a pharmaceutically acceptable salt thereof, and at least one pharmaceutically acceptable excipient, where, in particular, the pharmaceutical formulation is made by a process comprising wet granulation, direct compression or especially roller compaction, and related invention aspects disclosed herein.
The disclosure concerns a pharmaceutical composition comprising a 225Ac radiolabeled complex formed by a 225Ac radionuclide, and a target binding moiety linked to a chelating agent; and a bismuth sequestering agent, typically capable of sequestering Bi3+. The disclosure also concerns a method for preparing said pharmaceutical composition.
A61K 51/12 - Preparations containing radioactive substances for use in therapy or testing in vivo characterised by a special physical form, e.g. emulsion, microcapsules, liposomes
2-((4-((S)-2-(4-CHLORO-2-FLUOROPHENYL)-2-METHYLBENZO[D][1,3]DIOXOL-4-YL)PIPERIDIN-1-YL)METHYL)-1-(((S)-OXETAN-2-YL)METHYL)-1H-IMIDAZOLE DERIVATIVES AS ACTIVATORS OF THE GLP1 RECEPTOR FOR THE TREATMENT OF OBESITY
The present invention relates to compounds of formula (I): as activators of glucagon-like peptide 1 (GLP1) receptor for the treatment of obesity, type 2 diabetes mellitus, insulin resistance, hyperinsulinemia, glucose intolerance, hyperglycemia, one or more diabetic complications, diabetic nephropathy, dyslipidemia, non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), hypertension, atherosclerosis, peripheral arterial disease, stroke, cardiomyopathy, atrial fibrillation, heart failure, coronary heart disease and neuropathy.
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 31/4439 - Non-condensed pyridines; Hydrogenated 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 1/00 - Drugs for disorders of the alimentary tract or the digestive system
A61P 1/16 - Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
A61P 9/00 - Drugs for disorders of the cardiovascular system
A61P 9/04 - Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
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
The disclosure provides compounds of Formulae (I) or (II), or a pharmaceutically acceptable salt thereof, (I), or (II) as described herein, along with pharmaceutically acceptable salts, pharmaceutical compositions containing such compounds, and methods to use these compounds, salts and compositions for treating viral infections, particularly infections caused by herpesviruses.
C07D 241/44 - Benzopyrazines with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to carbon atoms of the hetero ring
A61K 31/4375 - 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 six-membered ring having nitrogen as a ring hetero atom, e.g. quinolizines, naphthyridines, berberine, vincamine
A61K 31/497 - Non-condensed pyrazines containing further heterocyclic rings
A61K 31/498 - Pyrazines or piperazines ortho- or peri-condensed with carbocyclic ring systems, e.g. quinoxaline, phenazine
A61K 31/4985 - Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems
A61K 31/5025 - Pyridazines; Hydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
A61K 31/519 - Pyrimidines; Hydrogenated 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 31/5386 - 1,4-Oxazines, e.g. morpholine spiro-condensed or forming part of bridged ring systems
A61K 31/539 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and at least one oxygen as the ring hetero atoms, e.g. 1,2-oxazines having two or more oxygen atoms in the same ring, e.g dioxazines
A61K 31/541 - Non-condensed thiazines containing further heterocyclic rings
A61P 31/22 - Antivirals for DNA viruses for herpes viruses
51.
PROCESS FOR THE SYNTHESIS OF PYRAZOLYL DERIVATIVES USEFUL AS ANTI-CANCER AGENTS
The invention provides a novel process, novel process steps and novel intermediates useful in the synthesis of pharmaceutically active compounds, especially KRAS G12C inhibitors.The present invention provides a direct enantioselective chemical manufacturing method of making Compound A, or a pharmaceutically acceptable hydrate or solvent thereof: (I).The invention provides a process for preparing Intermediate B6*comprising reacting Intermediate B4*with Intermediate B5*in an atroposelective coupling reaction, using a chiral catalyst.
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
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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 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
Disclosed are pharmaceutical formulations containing 7-cyclopentyl-N,N-dimethyl-2-{[5-(piperazin-1-yl)pyridin-2-yl]amino}-7H-pyrrolo[2,3-d]pyrimidine-6-carboxamide succinate, methods of using said formulations in therapy and processes for preparing the same.
Provided herein are compounds of formula (I) and pharmaceutical compositions thereof useful for treating diseases or disorders mediated by the complement factor B. (I).
A61P 37/00 - Drugs for immunological or allergic disorders
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 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 405/14 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing three or more hetero rings
C07D 491/107 - Spiro-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring
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
54.
TLR7/8-ANTAGONIST FOR TREATING SJÖGREN'S SYNDROME OR MIXED CONNECTIVE TISSUE DISEASE
The present disclosure relates to methods for treating Sjögren's Syndrome or mixed connective tissue disease using a compound of Formula (I) or a pharmaceutically acceptable salt thereof. Also disclosed herein is a compound of Formula (I) or a pharmaceutically acceptable salt thereof, for treating Sjögren's Syndrome or mixed connective tissue disease patients, as well as medicaments, dosing regimens, pharmaceutical formulations, dosage forms, and kits for use in the disclosed uses and methods.
vv1.2 activators for the treatment of schizophrenia, bipolar disorder, major depressive disorder, substance use disorder, ADHD, Phelan-McDermid Syndrome, autism spectrum disorder, multiple sclerosis, frontotemporal dementia, Alzheimer's disease, Brugada Syndrome, Short QT syndrome, or early repolarization syndrome.
C07D 491/048 - Ortho-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring the oxygen-containing ring being five-membered
A61K 31/4355 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having oxygen as a ring hetero atom
A61P 25/00 - Drugs for disorders of the nervous system
A61P 25/18 - Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
A61P 9/00 - Drugs for disorders of the cardiovascular system
A61P 9/02 - Non-specific cardiovascular stimulants, e.g. drugs for syncope, antihypotensives
56.
TREATMENT OF ALLERGIC REACTIONS USING ANTI-IGE ANTIBODIES
The present disclosure relates to methods for modifying the course of a disease or disorder involving IgE, in particular an IgE mediated allergic reactions to one or more allergens, in subjects having such disease or condition.
The present disclosure relates to methods of treating and/or preventing IgE driven allergic reaction to one or more allergens, preferably food allergens, in subjects having such disease or condition, comprising administering a therapeuticallly effective dose of LOU064.
The invention relates to a compound of formula (I) or a pharmaceutically acceptable salt thereof, wherein A, R1 and R3 are as described herein, as well as compositions and methods of using such compounds.
A61K 31/4375 - 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 six-membered ring having nitrogen as a ring hetero atom, e.g. quinolizines, naphthyridines, berberine, vincamine
59.
MODULATORS OF ADENO-ASSOCIATED VIRUS TRANSDUCTION AND USES THEREOF
The disclosure provides compositions and methods for modulating transduction efficiency of AAV particles. Specifically, the disclosure provides AAV transduction modulators that modulate genes or gene products associated with AAV transduction efficiency in gene therapies.
Provided herein are methods for assaying the chondrogenesis-inducing activity of a therapeutic by measuring the expression and/or secretion levels of chondrogenesis biomarkers.
The present invention relates to methods fortreating small cell lung cancer (SCLC), in particular small cell lung cancer (SCLC) in a subject in need thereof wherein a therapeutically efficient amount of a radiopharmaceutical compound comprising a SSTR binding moiety, in particular [177Lu]Lu-DOTATE is administered to said subject in combination with one or more chemotherapeutic agents, such as carboplatin and etoposide, and, optionally an immune- oncology (I/O) agent, such as tislelizumab.
The disclosure relates to a compound of Formula (I) or a pharmaceutically acceptable salt thereof wherein A and B are as described herein, as well as compositions and methods of using such compounds.
C07D 403/14 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group containing three or more hetero rings
C07D 413/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing three or more hetero rings
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
A61K 31/501 - Pyridazines; Hydrogenated pyridazines not condensed and containing further heterocyclic rings
A61K 31/4439 - Non-condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
Provided herein are methods for improving delivery of a pharmaceutical composition to the central nervous system of a subject in need thereof, the method comprising administering to the subject an agent that enhances glymphatic influx in combination with the pharmaceutical composition.
A61K 31/4535 - Non-condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a heterocyclic ring having sulfur as a ring hetero atom, e.g. pizotifen
A61K 31/7004 - Monosaccharides having only carbon, hydrogen and oxygen atoms
A61K 33/14 - Alkali metal chlorides; Alkaline earth metal chlorides
A61K 39/395 - Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
A61K 9/00 - Medicinal preparations characterised by special physical form
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61P 25/00 - Drugs for disorders of the nervous system
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
The present invention describes engineered immunoglobulin IgG Fc regions by transferring structural elements, several CH2 inter-chain disulfide bonds, from IgA to IgG immunoglobulin. The disclosed Fc variants created thereof exhibit marked reductions or complete abrogation of interaction of the engineered Fc with FcγR and C1q while retaining natural ability to interact with FcRn at acidic pH. Silenced Fc molecules disclosed are in comparable expression and purification yields and improved or maintained thermostability compared to wild-type Fc, thereby limiting the propensity for aggregation. In addition, the disclosed Fc variant silencing mutations are capable of reducing or compensating destabilizing effects of other half-life extending or chain pairing facilitating Fc mutations.
C07K 16/00 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies
C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans
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 present disclosure is directed to a method of treating glioblastoma in a subject in need thereof comprising administering to said subject an efficient amount of a radiopharmaceutical compound. The present disclosure is also directed to methods of treating glioblastoma in a subject in need thereof comprising administering to said subject an efficient amount of a radiopharmaceutical compound in combination with a step of irradiating the subject with an efficient dose of ionizing radiations, and optionally, with a therapeutically efficient amount of an alkylating agent, preferably temozolomide.
PHARMACEUTICAL COMBINATIONS COMPRISING AN MDM2 INHIBITOR, A BCL2 INHIBITOR AND A HYPOMETHYLATING AGENT AND USES THEREOF FOR THE TREATMENT OF HAEMATOLOGICAL MALIGNANCIES
The invention relates to a pharmaceutical combination comprising an MDM2 inhibitor, a BCL2 inhibitor and a hypomethylating agent. The present invention also relates to methods of treating haematological malignancies involving said combination.
A61K 31/506 - Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
A61K 31/635 - Compounds containing para-N-benzene- sulfonyl-N-groups, e.g. sulfanilamide, p-nitrobenzenesulfonohydrazide having a heterocyclic ring, e.g. sulfadiazine
A61K 31/706 - Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
HHH-spiro[imidazo[1,2- ]pyridine-2,5'-isoquinoline], or a pharmaceutically acceptable salt thereof, or a free form thereof are described. Further, processes for preparing said pharmaceutical 5 compositions for oral administration and uses of said pharmaceutical compositions in the manufacture of a medicament are described.
The present invention is directed to stabilized protein-containing formulations, stabilized or inhibited against protein aggregation, comprising an amphiphilic surfactant.
A61K 9/00 - Medicinal preparations characterised by special physical form
A61K 47/08 - Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen
A61K 47/14 - Esters of carboxylic acids, e.g. fatty acid monoglycerides, medium-chain triglycerides, parabens or PEG fatty acid esters
C07D 311/20 - Benzo [b] pyrans, not hydrogenated in the carbocyclic ring with oxygen or sulfur atoms directly attached in position 2 hydrogenated in the hetero ring
C08G 65/00 - Macromolecular compounds obtained by reactions forming an ether link in the main chain of the macromolecule
69.
A COMPUTER IMPLEMENTED METHOD FOR ASSESSING AND DETERMINING A COMPLEXITY LEVEL OF A CLINICAL TRIAL STUDY
The present invention relates to a computer-implemented method for assessing and determining a complexity level of at least a portion, or pillar, of a clinical trial study. The method may be implemented by a computing device including at least a processor; a memory and an input and/or output elements. It comprises the steps of selecting and/or defining a clinical trial protocol and, based on the selection and/or definition thereof, activating and/or generating data entries for a set of corresponding parameters correlated to a set-up of the portion of the clinical trial study. The parameters refer to objectives; and/or design; and/or methods; and/or patient assessment procedures and/or patient data collection schedules of the clinical trial study. The method further comprises the steps of automatically assigning a score value to each parameter in the set of parameters; applying statistical rules to the score value of each parameter to obtain a scaled score value; and calculating a complexity level for the portion of clinical trial study, based on the scaled score values of each parameter in the set of parameters.
G16H 10/20 - ICT specially adapted for the handling or processing of patient-related medical or healthcare data for electronic clinical trials or questionnaires
G16H 40/20 - ICT specially adapted for the management or administration of healthcare resources or facilities; ICT specially adapted for the management or operation of medical equipment or devices for the management or administration of healthcare resources or facilities, e.g. managing hospital staff or surgery rooms
G16H 40/63 - ICT specially adapted for the management or administration of healthcare resources or facilities; ICT specially adapted for the management or operation of medical equipment or devices for the operation of medical equipment or devices for local operation
The present invention relates to novel transcription factors for modulating the expression of gene of interest, by fusing to a DNA binding domain that targets the gene of interest.
The present invention relates to methods, treatment regimens, uses, kits and therapies for prevention of graft rejection in solid organ transplantation, particularly solid organ xenotransplantation, by administering an anti-CD40 antibody or a combination of an anti-CD40 antibody and an anti-C5 antibody.
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
A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection
A61K 39/00 - Medicinal preparations containing antigens or antibodies
72.
USE OF FACTOR B INHIBITORS FOR THE TREATMENT OF AGE-RELATED MACULAR DEGENERATION
Described herein are methods of treating early or intermediate age-related macular degeneration with the Factor B inhibitor iptacopan or a pharmaceutically acceptable salt thereof, e.g. iptacopan hydrochloride.
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
The invention relates to a pharmaceutical combination comprising a TEAD inhibitor in combination with a first and optionally a second therapeutically active agent. The present invention also relates to methods of treating cancer involving administering to a subject in need thereof the TEAD inhibitor in combination with the first and optionally the second therapeutically active agent.
A61K 31/343 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide condensed with a carbocyclic ring, e.g. coumaran, bufuralol, befunolol, clobenfurol, amiodarone
A61K 31/351 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom not condensed with another ring
A61K 31/4025 - 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 31/443 - Non-condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with oxygen as a ring hetero atom
A61K 31/4439 - Non-condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
The present invention generally relates to crystalline polymorphic forms of the biaryl YAP/TAZ-TEAD protein-protein interaction inhibitors 4-((2S,4S)-5-Chloro-6-fluoro-2-phenyl-2-((S)-pyrrolidin-2-yl)-2,3-dihydrobenzofuran-4-yl)-5-fluoro-6-(2-hydroxyethoxy)-N-methylnicotinamide and 2-((2S,3S,4S)-5-Chloro-6-fluoro-3-methyl-2-((methylamino)methyl)-2-phenyl-2,3-dihydrobenzofuran-4-yl)-3-fluoro-4-methoxybenzamide and salts thereof, as well as methods of using the forms in the treatment of cancer.
C07D 307/81 - Radicals substituted by nitrogen atoms not forming part of a nitro radical
C07D 405/04 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings directly linked by a ring-member-to-ring- member bond
A61K 31/343 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide condensed with a carbocyclic ring, e.g. coumaran, bufuralol, befunolol, clobenfurol, amiodarone
A61K 31/443 - Non-condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with oxygen as a ring hetero atom
The invention relates to a TEAD inhibitor or a pharmaceutically acceptable salt thereof for use in the treatment of cancer, wherein the TEAD inhibitor is administered on each of the first 3 days of a 7 day treatment cycle, and wherein the treatment comprises at least two treatment cycles.
A61K 31/343 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide condensed with a carbocyclic ring, e.g. coumaran, bufuralol, befunolol, clobenfurol, amiodarone
The present disclosure relates to a conjugate comprising a YAP/TAZ-TEAD Protein Protein Interaction Inhibitor (PPII) linked to a Ligase Binder via a linker, or a pharmaceutically acceptable salt thereof, as well as methods of using such conjugates.
A61K 47/54 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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 47/55 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting 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
A61K 31/505 - Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
A61K 47/00 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
The invention concerns ofatumumab for use in the treatment or prevention of pediatric multiple sclerosis (MS). According to the invention, ofatumumab is administered during a loading dose regimen at weeks 0, 1, 2 of the dosage regimen; and ofatumumab is administered during a maintenance dose regimen starting at week eight of the dosage regimen and continuing thereafter every six weeks.
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
A61P 37/00 - Drugs for immunological or allergic disorders
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
79.
METHODS OF MAKING CHIMERIC ANTIGEN RECEPTOR–EXPRESSING CELLS
The invention provides methods of making immune effector cells (for example, T cells, NK cells) that express a chimeric antigen receptor (CAR), and compositions generated by such methods.
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
Provided herein are compounds and compositions for treating, managing or preventing coronaviral related diseases. In particular, provided herein are compounds which are inhibitors of SARS-CoV-2 main protease (Mpro), pharmaceutical compositions comprising such compounds, method for synthesizing such compounds and methods of using such compounds and compositions for the treatment, management or prevention of coronaviral related diseases.
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 401/14 - 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 31/4725 - Non-condensed isoquinolines, e.g. papaverine containing further heterocyclic rings
The disclosure provides compounds of Formula (I): (I) as further described herein, as well as pharmaceutical compositions comprising such compounds, and methods to use the compounds and pharmaceutical compositions for treatment of certain viral disorders, including influenza.
C07D 401/10 - 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 aromatic 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 405/10 - 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 405/12 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings linked by a chain containing hetero atoms as chain links
C07D 409/12 - Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms containing two hetero rings linked by a chain containing hetero atoms as chain links
C07D 413/10 - 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 aromatic rings
C07D 413/12 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings linked by a chain containing hetero atoms as chain links
C07D 413/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing three or more hetero rings
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 present disclosure relates to the field of pharmacy, particularly to an NLRP3 inhibitor for use in the treatment of osteoarthritis. The disclosure also relates to an NLRP3 inhibitor or a pharmaceutical combination comprising an NLRP3 inhibitor, and at least one further therapeutic agent, for use in the treatment of osteoarthritis; to a method for the treatment of osteoarthritis that involves administering an NLRP3 inhibitor or the combination; and to the use of an NLRP3 inhibitor or the combination for the manufacture of a medicament for the treatment of osteoarthritis.
The present invention provides a compound of formula (I) or a pharmaceutically acceptable salt thereof; a method for manufacturing the compounds of the invention, and its therapeutic uses. The present invention further provides a combination of pharmacologically active agents and a pharmaceutical composition.
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 403/12 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group containing two hetero rings linked by a chain containing hetero atoms as chain links
C07D 413/12 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings linked by a chain containing hetero atoms as chain links
C07D 413/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing three or more hetero rings
C07D 417/12 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group containing two hetero rings linked by a chain containing hetero atoms as chain links
C07D 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 457/04 - Heterocyclic compounds containing indolo [4, 3-f, g] quinoline ring systems, e.g. derivatives of ergoline, of the formula: , e.g. lysergic acid with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached in position 8
The invention relates to bivalent bispecific monoclonal antibodies (bbmAb) or variants thereof for use in the treatment or for use in alleviating the symptoms of hidradenitis suppurativa in a subject.
C07K 16/24 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
The present invention provides a compound of formula (I), or a pharmaceutically acceptable salt thereof, and the therapeutic uses of said compound. The present invention further provides a pharmaceutical composition comprising said compound.
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 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
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 491/048 - Ortho-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring the oxygen-containing ring being five-membered
C07D 491/056 - Ortho-condensed systems with two or more oxygen atoms as ring hetero atoms in the oxygen-containing ring
C07D 491/107 - Spiro-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring
The present invention relates to a pharmaceutical combination comprising a KRAS G12C inhibitor and one or more therapeutic agents which is selected from an agent targeting the MARK pathway or an agent targeting parallel pathways; and pharmaceutical compositions comprising the same. The invention also relates to KRAS G12C inhibitors alone or said combinations for use in methods of treating a cancer or a tumor, in particular wherein the cancer or tumor is KRAS G12C mutant.
A61K 31/416 - 1,2-Diazoles condensed with carbocyclic ring systems, e.g. indazole
A61K 31/436 - 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 six-membered ring having oxygen as a ring hetero atom, e.g. rapamycin
A61K 31/4439 - Non-condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
A61K 31/497 - Non-condensed pyrazines containing further heterocyclic rings
A61K 31/506 - Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
A61K 31/517 - Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with carbocyclic ring systems, e.g. quinazoline, perimidine
A61K 31/519 - Pyrimidines; Hydrogenated 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 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
The present invention provides anti-lgE antibodies formulated as stable aqueous pharmaceutical compositions, suitable for injection. An aqueous pharmaceutical composition of the invention includes a sugar (trehalose), a buffering agent (histidine), and a surfactant (polysorbate 20). The aqueous pharmaceutical compositions are useful for delivery of a high concentration of the antibody (at least 50 mg/ml) active ingredient to a patient without high levels of antibody aggregation and without a high level of sub-visible particulate matter.
A61K 31/454 - Non-condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. pimozide, domperidone
A61P 7/00 - Drugs for disorders of the blood or the extracellular fluid
The present invention relates to a pharmaceutical combination comprising dabrafenib, trametinib and a SHP2 inhibitor; pharmaceutical compositions comprising the same; and methods of using such combinations and compositions in the treatment or prevention of conditions in which MAPK pathway inhibition is beneficial, for example, in the treatment of cancers.
The application relates to compounds of formula (I), pharmaceutical compositions comprising them and their use in reducing Widely Interspaced Zinc Finger Motifs (WIZ) expression levels, or inducing fetal hemoglobin (HbF) expression, and in the treatment of inherited blood disorders (e.g., hemoglobinopathies, e.g., beta- hemoglobinopathies), such as sickle cell disease and beta- thalassemia.
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 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 31/454 - 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 31/4545 - 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 31/55 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
92.
CD19 AND CD22 CHIMERIC ANTIGEN RECEPTORS AND USES THEREOF
The present disclosure provides methods for treating diseases associated with expression of CD 19 and/or CD22, e.g., by administering a recombinant T cell or natural killer (NK) cell comprising a CD22 CAR and a CD 19 CAR as described herein at a dosage based on cells/kg body weight and the age of the patient.
Provided herein are methods and dosage regimens for the treatment of osteoarthritis (e.g., knee osteoarthritis). These methods and dosage regimens include intra-articular injections of Compound 1.
Provided herein are methods and dosage regimens for the treatment of osteoarthritis (e.g., knee osteoarthritis). These methods and dosage regimens include intra-articular injections of Compound 1 alone, or in combination with an anti-inflammatory antibody (e.g., an anti-IL-10 antibody).
12345262727, y, R, M, W, L, V, T, Y, J, K and A are as described herein, therapeutic uses of said compounds, uses of said compounds as research chemicals, a pharmaceutical composition and combinations comprising said compounds, and methods for manufacturing the compounds of the invention.
Provided herein are compounds and pharmaceutical compositions useful for treating atopic dermatitis, contact dermatitis, chronic hand dermatitis, psoriasis, vitiligo, and alepecia areata, comprising administering to a subject in need thereof a therapeutically effective amount of a compound of Formula (I) or a compound of Formula (I'), or pharmaceutical composition described herein. (I) (I')
A61K 31/438 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom the ring being spiro-condensed with carbocyclic or heterocyclic ring systems
A61K 31/4439 - Non-condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
A61K 31/497 - Non-condensed pyrazines containing further heterocyclic rings
A61K 31/506 - Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
A61K 31/5377 - 1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
The present disclosure relates to pharmaceutical combinations comprising inhibitors of hypoxia-inducible factor-2α (HIF2α). The combination therapies can be used to treat or prevent cancerous conditions and disorders.
A61K 31/4743 - Quinolines; Isoquinolines ortho- or peri-condensed with heterocyclic ring systems condensed with ring systems having sulfur as a ring hetero atom
FORMULATIONS OF 3-((3-(4-(2-(ISOBUTYLSULFONYL)PHENOXY)-3-(TRIFLUOROMETHYL)PHENYL)-1,2,4-OXADIAZOL-5-YL)METHYL)-5,5-DIMETHYL-1-(2-MORPHOLINOETHYL)IMIDAZOLIDINE-2,4-DIONE
Formulations of 3-((3-(4-(2-(isobutylsulfonyl)phenoxy)-3-(trifluoromethyl)phenyl)-1,2,4-oxadiazol-5-yl)methyl)-5,5-dimethyl-1-(2-morpholinoethyl)imidazolidine-2,4-dione and pharmaceutically acceptable salts thereof, processes for their production, and uses thereof, including in the treatment of ocular diseases and disorders such as dry eye disease and Meibomian gland dysfunction (MGD).
A61K 47/32 - Macromolecular compounds obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. carbomers
A61K 47/44 - Oils, fats or waxes according to two or more groups of ; Natural or modified natural oils, fats or waxes, e.g. castor oil, polyethoxylated castor oil, montan wax, lignite, shellac, rosin, beeswax or lanolin
99.
COMPOSITIONS AND METHODS FOR ENHANCING VISUAL FUNCTION
The present disclosure provides compositions and methods of restoring or enhancing visual function in an individual by administering to the individual a pharmaceutical composition comprising a recombinant adeno-associated viral (rAAV) vector having a polynucleotide sequence that encodes a medium wavelength cone opsin (MW-opsin). The MW-opsin is expressed in a retinal cell in the individual, thereby restoring or enhancing visual function.
The present disclosure relates to dosing regimens and combinations comprising N-[4-(Chlorodifluoromethoxy)phenyl]-6-[(3R)-3-hydroxypyrrolidin-1-yl]-5-(1H-pyrazol-5-yl)pyridine-3-carboxamide or a pharmaceutically acceptable salt thereof, and their use for the treatment of breakpoint cluster region-abelson protein (BCR-ABL) mediated diseases or disorders.
A61K 31/4439 - Non-condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
A61K 31/496 - Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene
A61K 31/5025 - Pyridazines; Hydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
A61K 31/506 - Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
A61P 35/02 - Antineoplastic agents specific for leukemia