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TYK2 DEGRADERS

Resumen de: WO2026156134A1

The present invention provides a compound of formula I or a pharmaceutically acceptable salt thereof T-L-B (I), useful for the modulation of TYK2 protein via degradation, their manufacture, pharmaceutical compositions containing them and their use as therapeutically active substances. The active compounds of the present invention are useful in the therapeutic and/or prophylactic treatment of autoimmune disorders like type 1 diabetes, ankylosing spondylitis, cutaneous lupus erythematosus, systemic lupus erythematosus, lupus nephritis, multiple sclerosis, systemic sclerosis, psoriasis, Crohn's disease, ulcerative colitis, proteasome-associated autoinflammatory syndromes (PRAAS), ISG15 deficiency, and inflammatory bowel disease; inflammatory disorders like rheumatoid arthritis, asthma, chronic obstructive pulmonary disease, psoriasis, Crohn's disease, ulcerative colitis, STING-associated vasculopathy with onset in infancy (SAVI). and inflammatory bowel disease; proliferative disorders like hematological cancer, polycythemia vera, myelofibrosis, essential thrombocythemia, and thrombocytosis; endocrine disorders like polycystic ovary' syndrome, Crouzon's syndrome, and type 1 diabetes; neurological disorders like Alzheimer's disease. Aicardi-Goiuteieres syndrome (AGS). Parkinson's disease, amyotrophic lateral sclerosis, Huntington's disease, cerebral ischemia, and neurodegenerative disease caused by traumatic injury, glutamate neurotoxicity and hypoxia: and/or disorders associated

TREATMENT FOR SOD1 ASSOCIATED DISEASE

Resumen de: EP4778582A2

The present invention relates to antisense oligonucleotides that are complimentary to SOD1, leading to decreased expression of SOD1. Reduced expression of SOD1 is beneficial in medical disorders such as Amyotrophic Lateral Sclerosis.

5,6-DISUBSTITUTED ALKYL 1H AND 2H-INDAZOLES AS DUAL BUTYRYLCHOLINESTERASE AND P38ALPHA MITOGEN-ACTIVATED PROTEIN KINASE INHIBITORS FOR USE IN THE TREATMENT OF NEURODEGENERATIVE AND INFLAMMATORY DISEASES

Resumen de: WO2025056518A1

The present invention relates to the field of medicinal chemistry and more specifically to 5,6- disubstituted 1H- and 2H- indazoles as pharmaceutically active compounds. The compounds of the present invention are dual inhibitors of butyrylcholinesterase and p38α mitogen-activated protein kinase, and thus are particularly useful for the treatment of various diseases which may be therapeutically modified by altering the activity of butyrylcholinesterase (BChE) and/or p38α MAP kinase such as dementias including Alzheimer's disease (AD) and other conditions involving chronic inflammation.

COMPOSITIONS AND METHODS FOR TREATMENT RELATED TO FALL AND FALL FREQUENCY IN NEURODEGENERATIVE DISEASES

Resumen de: EP4778518A2

0001 The present disclosure provides methods and compositions for treating fall-related symptoms in patients with neurodegenerative diseases such as Parkinson's disease or Parkinson-related diseases. In some embodiments, the disclosure utilizes nicotine or a salt thereof in combination of dopaminergic agent treatments for reducing fall-related symptoms such as reducing frequency of fall, reducing injuries related to fall, reducing severity of injuries related to fall, freezing of gait, improving posture stability, improving locomotion ability, improving balance and gait. In some embodiments, the methods predict fall frequency and tendency of recurrent falls in patients with Parkinson's disease, in particular, patients with typical Parkinson's disease.

NEUROPROTECTIVE DRUG SUBSTANCE-LOADED NANOMOTOR FORMULATIONS

Resumen de: US20260199232A1

A polymer layer containing a pharmaceutical drug substance-loaded amino group and a nanomotor containing a metal layer for orientation by a magnetic field is used to treat Alzheimer's Disease; Memantine HCl-loaded nanomotors are synthesized with a biocompatible poly-L-lysine polymer whose movement is directly controlled by the magnetic field and targets the brain by crossing the blood brain barrier by providing movement in the magnetic field.

AGENTS, USES AND METHODS FOR THE TREATMENT OF SYNUCLEINOPATHY

Resumen de: US20260201025A1

The invention relates to combinational treatment using a monoclonal anti-alpha-synuclein antibody and an additional medicament. The antibodies can be used for treating a synucleinopathy such as Parkinson's disease (including idiopathic and inherited forms of Parkinson's disease), Diffuse Lewy Body Disease (DLBD), Lewy body variant of Alzheimer's disease (LBV), Combined Alzheimer's and Parkinson disease, pure autonomic failure and multiple system atrophy together with another medicament of the invention.

DEUTERATED HUPERZINE A COMPOUND OR PHARMACEUTICALLY ACCEPTABLE SALT THEREOF, AND PREPARATION METHOD THEREFOR AND USE THEREOF

Resumen de: WO2026148695A1

The present invention relates to the technical field of medicines, and specifically relates to a deuterated huperzine A compound or a pharmaceutically acceptable salt thereof, and a preparation method therefor and a use thereof. The structure of the deuterated huperzine A compound is as follows, wherein R1-R9 are each independently H or D, and are not both H. The present invention has the advantages of high chemical stability, long metabolic half-life, low toxicity, significant synergistic effect with bremelanotide in the treatment of Alzheimer's disease, and ability to reduce the toxicity of bremelanotide.

AZAINDAZOLE DERIVATIVES USEFUL AS INHIBITORS OF NOD-LIKE RECEPTOR PROTEIN 3

Resumen de: AU2024400795A1

Novel compounds of Formula (I), and the pharmaceutically acceptable salts thereof, are inhibitors of NLRP3 and may be useful in the treatment, prevention, management, amelioration, control and suppression of diseases mediated by NLPR3. The compounds of the present invention may be useful in the treatment, prevention or management of diseases, disorders and conditions mediated by NLRP3 such as, but not limited to, obesity, gout, pseudogout, CAPS, NASH, MASH, fibrosis, heart failure, idiopathic pericarditis, atopic dermatitis, inflammatory bowel disease, Alzheimer's Disease, Parkinson's Disease, dementia with Lewy bodies (DLB), and traumatic brain injury.

AGONISTS OF TREM2 ACTIVITY

Resumen de: AU2024403764A1

The present disclosure is directed to compounds of Formula (I) and their use as TREM2 agonists for treatment and prevention of a neurodegenerative disorder associated with a loss of function of human TREM2. The disclosed TREM2 agonists may be useful for the treatment of Alzheimer's Disease and associated neurological conditions.

AGONISTS OF TREM2 ACTIVITY

Resumen de: AU2024406047A1

The present disclosure is directed to compounds of Formula I and their use as TREM2 agonists for treatment and prevention of a neurodegenerative disorder associated with a loss of function of human TREM2. The disclosed TREM2 agonists may be useful for the treatment of Alzheimer's Disease and associated neurological conditions.

METHOD OF PROLONGING THE SURVIVAL OF A SUBJECT WITH ALS

Resumen de: AU2025210321A1

This invention provides a method of prolonging the survival of subjects afflicted with ALS by administering a composition comprising pridopidine or pharmaceutically acceptable salt thereof.

COMPOSITION FOR PREVENTING OR TREATING ALZHEIMER'S DISEASE

Resumen de: WO2026151017A1

The compound represented by chemical formula IA according to the present invention is useful for the prevention, alleviation, and treatment of degenerative brain diseases.

CRYSTALLINE FORMS OF 2-(TERT-BUTOXY)-4-(3-METHYL-3-(5-(METHYLSULFONYL)ISOINDOLIN-2-YL)BUTYL)PHENOL FUMARATE SALT

Resumen de: US20260200848A1

The present disclosure describes crystalline forms of 2-(tert-butoxy)-4-(3-methyl-3-(5-(methylsulfonyl)isoindolin-2-yl)butyl)phenol fumarate salts and pharmaceutical compositions of same. Also described are methods of using the crystalline forms treating Alzheimer's Disease, Dementia with Lewy Bodies and Dry age-related macular degeneration in a subject in need thereof, comprising administering the crystalline form to the subject. Methods of making the solid forms are also described.

THERAPEUTIC METHODS AND USES FOR EPERTINIB

Resumen de: WO2026148417A1

Disclosed herein are methods of using epertinib in the treatment of neurological conditions such as ALS.

GLS1 DIAGNOSTIC METHODS

Resumen de: WO2026151983A1

Aspects of the disclosure relate to compositions and methods for modulating levels, transcription, splicing, and/or translation of one or more RNA transcripts (e.g., mRNA transcripts) in a cell or subject. The disclosure is based, in part, on methods of identifying a subject of having or being at risk of developing a disease or disorder associated with dysregulated glutamine levels, such as a subject having a mutation that affects glutaminase (GLS1), which is an enzyme responsible for producing glutamate from glutamine. In some embodiments, compositions and methods of the disclosure are useful for treating a psychiatric disorder (e.g., depression (DEP), major depressive disorder (MDD), bipolar disorder (e.g., BPD1, BPD2), mania (MAN), psychosis (PSY), schizophrenia (SCZ), schizoaffective disorder (SZA), or post-traumatic stress disorder (PTSD)) and/or a disease associated with dementia, such as Alzheimer's disease.

GENE THERAPY FOR LEVODOPA-INDUCED DYSKINESIA IN PARKINSONS DISEASE

Resumen de: US20260201390A1

The present disclosure provides compositions and methods for the treatment of levodopa-induced dyskinesia (LID) and Parkinson's disease (PD) therewith. In particular, the present disclosure provides methods for treating LID in PD by inhibiting (e.g., expression of) Muscarinic M1 acetylcholine receptor (M1Rs), for example, in indirect pathway spiny projection neurons (iSPNs) to attenuate LID without compromising of the treatment with levodopa (e.g., boosting the efficacy of levodopa treatment).

SMALL MOLECULES FOR THE RESCUE OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) AND FRONTOTEMPORAL DEGENERATION (FTD) BY TARGETING C9ORF72 HEXANUCLEOTIDE G-QUADRUPEXES

Resumen de: US20260199329A1

0000 The subject invention pertains to a composition comprising a series of compounds selectively targeting G-quadruplexes (G4s) formed by the GGGGCC (G4C2) hexanucleotide repeats (HREs) of C9orf72, (G4C2). The composition comprises compounds G4008, G4009, G4010, G4012, and G4013, or a pharmaceutically acceptable salt, solvate, or hydrate thereof and at least one pharmaceutically acceptable carrier and/or excipient. The subject invention further pertains to a method for treating amyotrophic lateral sclerosis (ASL) and/or frontotemporal dementia (FTD), comprising administering to the subject an effective amount of the composition. The compounds can ameliorate the pathological signatures of ALS model caused by C9orf72 (G4C2)sequence.

Amyloid precursor protein (APP) RNAi agent compositions and methods of use thereof

Resumen de: AU2026204572A1

The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting the APP gene, as well as methods of inhibiting expression of an APP gene and methods of treating subjects having an APP-associated disease or disorder, such as cerebral amyloid angiopathy (CAA) and early onset familial Alzheimer disease (EOFAD or eFAD), using such dsRNAi agents and compositions. un u n

Novel Molecules for Therapy and Diagnosis

Resumen de: US20260201026A1

0000 The present invention relates to novel molecules that can be employed for the prevention, alleviation, treatment and/or diagnosis of diseases, disorders and abnormalities associated with alpha-synuclein (α-synuclein, A-synuclein, aSynuclein, A-syn, α-syn, aSyn, a-syn) aggregates, including, but not limited to, Lewy bodies and/or Lewy neurites, such as Parkinson's disease, Multiple System Atrophy, Lewy Body dementia (LBD; dementia with Lewy bodies (DLB) (“pure” Lewy body dementia), Parkinson's disease dementia (PDD)) or Diffuse Lewy Body Disease. The invention relates to alpha-synuclein binding molecules, in particular to alpha-synuclein antibodies or an antigen-binding fragment or a derivative thereof and uses thereof. The present molecules can also be used for determining a predisposition to such a disorder, disease or abnormality, monitoring residual disorder, disease or abnormality, or predicting the responsiveness of a patient who is suffering from such a disorder, disease or abnormality to treatment with a certain medicament.

METHODS FOR TREATING NEURODEGENERATIVE DISORDERS WITH TRAMIPROSATE

Resumen de: US20260199270A1

Provided herein are methods for treating Alzheimer's disease using a combination of tramiprosate, a tramiprosate prodrug or an active tramiprosate metabolite with at least one amyloid plaque clearing agent.

BIS-SULFONAMIDE SHIP1 ACTIVATORS

Resumen de: US20260199365A1

Provided are compounds and pharmaceutically acceptable salt, solvate and/or derivative thereof. Further, provided are methods of treating a disease, disorder or condition mediated or treatable by activation of SHIP1 comprising administering a SHIP1 activator compound or a pharmaceutically acceptable salt, solvate or derivative thereof. The compound or a pharmaceutically acceptable salt, solvate or derivative thereof may be used in the treatment of SHIP1 mediated disease, disorder or conditions including inflammatory bowel disease (IBD), Crohn′ disease, COPD, ulcerative colitis, arthritis, and Alzheimer's Disease.

RECOMBINANT AAV VECTORS FOR TREATING NEURODEGENERATIVE DISORDERS

Resumen de: US20260199527A1

0000 Provided is a recombinant adeno-associated viral (rAAV) vector comprising one or two of (a) to (c): (a) a nucleotide sequence encoding aromatic L-amino acid decarboxylase (AADC), (b) a nucleotide sequence encoding glucocerebrosidase (GBA1); and (c) a nucleotide sequence encoding a neurotrophic factor (NTF), such as cerebral dopamine neurotrophic factor (CDNF) or glial cell derived neurotrophic factor (GDNF), for treating neurodegenerative disorders, particularly Parkinson's disease (PD), Multiple system atrophy (MSA), Gaucher's disease (GD), and other proteinopathies. Also provided herein are viral particles comprising the rAAV vector, a pharmaceutical composition comprising the viral particles, and uses thereof.

SIRNA INHIBITING EXPRESSION OF AMYLOID PRECURSOR PROTEIN (APP) GENE, DRUG, AND USE

Resumen de: US20260201383A1

The present invention provides siRNA, peptide oligonucleotide drugs, and their applications for suppressing the expression of the amyloid precursor protein (APP) gene in human cells. The siRNA exhibits potent activity in inhibiting APP expression. Through appropriate modifications, its ability to silence the target is enhanced while reducing off-target activity. The described siRNA and its conjugates hold promise for clinical application in the prevention and treatment of diseases associated with the APP target, including cerebral amyloid angiopathy (CAA), early-onset familial Alzheimer's disease (EOFAD), or Alzheimer's disease (AD).

Modulators of chromosome 9 open reading frame 72 gene expression and uses thereof

Resumen de: AU2026204876A1

The present disclosure provides compositions and methods for modulating transcription of mutant C9orf72 gene alleles in patients in need thereof, including patients having a C9orf72-related disease such as amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD). un u n

PYRIDINE DERIVATIVES WHICH ACT AS INHIBITORS OF MPTP

Nº publicación: EP4774033A1 15/07/2026

Solicitante:

NRG THERAPEUTICS LTD [GB]
NRG Therapeutics LTD

Resumen de: WO2025052129A1

The invention relates to compounds of formula (I): and related aspects. The compounds are inhibitors of mPTP.