Resumen de: WO2025250667A1
The present disclosure relates to RIPK2 inhibitors of the formulae (I) or (II) for the treatment of e.g. inflammatory diseases, autoimmune diseases, granulomatous disease, neurodegenerative diseases or cancer, and, more specifically, for the treatment of inflammatory bowel disease, such as Crohn's disease or ulcerative colitis, rheumatoid arthritis, inflammatory arthritis, peritonitis, ischemia reperfusion injury in kidney transplant, non-alcohol steatohepatitis, alcohol steatohepatitis, insulin-resistant type 2 diabetes, allergic rhinitis, asthma, atopic dermatitis, Sjogren's syndrome, spondyloarthritis, ankylosing spondylitis, pemphigus vulgaris, idiopathic plasmacytic lymphadenopathy, atherosclerosis, myocardial infarction, thrombosis, alpha-synucleinopathy, Parkinson's disease, dementia with Lewy body, multiple system atrophy, Alzheimer's disease, amyotrophic lateral sclerosis, and chronic obstructive pulmonary disease.
Resumen de: WO2025250650A1
The present disclosure relates to crystalline forms of N-(l-(tert-butyl)-lH-pyrazol-4-yl)-2-( 4-((6-((methylsulfonyl)quinolin-4-yl)oxy)-3-methylphenyl)acetamide of formula (II) and crystalline forms of salts thereof. The compound of formula (II) is a RIPK2 inhibitor for the treatment of e.g. inflammatory diseases, autoimmune diseases, granulomatous disease, neurodegenerative diseases or cancer, and more specifically for the treatment of inflammatory bowel disease, such as Crohn's disease or ulcerative colitis, rheumatoid arthritis, inflammatory arthritis, peritonitis, ischemia reperfusion injury in kidney transplant, non-alcohol steatohepatitis, alcohol steatohepatitis, insulin-resistant type 2 diabetes, allergic rhinitis, asthma, atopic dermatitis, Sjogren's syndrome, spondyloarthritis, ankylosing spondylitis, pemphigus vulgaris, idiopathic plasmacytic lymphadenopathy, atherosclerosis, myocardial infarction, thrombosis, alpha-synucleinopathy, Parkinson's disease, dementia with Lewy body, multiple system atrophy, Alzheimer's disease, amyotrophic lateral sclerosis, and chronic obstructive pulmonary disease
Resumen de: US2025367123A1
Provided herewith is a pharmaceutical composition comprising, separately or together, a pulsatile release component comprising levodopa and a DOPA decarboxylase inhibitor for the management of OFF-time episodes in patients with Parkinson's disease.
Resumen de: ZA201902226B
Aspects of the disclosure relate to compositions and methods useful for treating Huntington' s disease. In some embodiments, the disclosure provides interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and methods of treating Huntington's disease using the same.
Resumen de: MX2025002727A
The disclosure relates to a combination of active ingredients/adjuvants for the treatment of neurological disorders and diseases such as Alzheimer's disease and mild cognitive impairment (MCI) and memory and cognitive disorders and conditions. In particular, combinations of scyllo-inositol and treatments for Alzheimer's disease such as lecanemab are disclosed as useful.
Resumen de: MX2025013089A
The purpose of the present invention is to provide a drug that exhibits the effect of inhibiting aggregation of a causative protein of an HRE-related neurodegenerative disease such as ALS. According to the present invention, rifampicin or a related substance selected from the group consisting of rifampicin, a derivative thereof, and salts thereof, and/or resveratrol or a related substance selected from the group consisting of resveratrol and a derivative thereof is an active ingredient of a preventive or therapeutic agent for a neurodegenerative disease caused by TDP-43 accumulation, or an active ingredient of a preventive or therapeutic agent for ALS.
Resumen de: MX2025010647A
Methods for preventing, limiting or delaying clinical motor progression in a subject with Parkinson's disease with low GCase activity, such as a PD patient with a pathogenic variant in the glucocerebrosidase 1 (GBA1) gene (GBA-PD) is provided, said methods comprising administering a therapeutically effective amount of 5,7-dimethyl-N-((1R,4R)-4- (pentyloxy)cyclohexyl)pyrazolol1,5-apyrimidine-3-carboxamide (Compound A), or a pharmaceutically acceptable salt thereof, to said subject.
Resumen de: MX2025010867A
Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a Ataxin-2 (ATXN2) gene. The ATXN2 RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an ATXN2 gene. Pharmaceutical compositions that include one or more ATXN2 RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described ATXN2 RNAi agents to central nervous system (CNS) tissue, in vivo, provides for inhibition of ATXN2 gene expression and a reduction in ATXN2 activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including spinocerebellar ataxia type 2 (SCA2) or amyotrophic lateral sclerosis (ALS.)
Nº publicación: CA3255657A1 01/12/2025
Solicitante:
GENZYME CORP [US]
Genzyme Corporation
Resumen de: CA3255657A1
Provided herein are RNAi molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed region of the guide sequences; e.g., opposite the cleavage sequence. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.