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Solicitudes publicadas en los últimos 60 días / Last 60 days publications
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TREATMENT OF DISEASE

Publication No.:  WO2025264823A1 26/12/2025
Applicant: 
METASHAPE PHARMA AG [CH]
MEHRLING THOMAS [CH]
BANTIA SHANTA [CH]
METASHAPE PHARMA AG,
MEHRLING, Thomas,
BANTIA, Shanta

Absstract of: WO2025264823A1

This disclosure relates to the use of Purine Nucleoside Phosphorylase (PNP) inhibitors such as ulodesine and its salts, in the treatment and/or prevention of diseases associated with nicotinamide adenine dinucleotide (NAD+) depletion, including diseases of mitochondrial dysfunction (including neurodegeneration and peripheral neuropathies), the preservation of cognitive function and in muscular disorders such as sarcopenia; and in metabolic syndrome and associated conditions. In particular the disclosure provides the use of PNP inhibitors such as ulodesine in the treatment of neurodegenerative conditions such as Parkinson's disease and amyotrophic lateral sclerosis.

AGENTS AND/OR COMPOSITIONS USEFUL FOR MODULATING CIS-REGULATORY ELEMENTS IN SYNUCLEINOPATHIES AND METHODS FOR IDENTIFYING AGENTS AND COMPOSITIONS THEREOF

Publication No.:  WO2025264967A1 26/12/2025
Applicant: 
THE JOHNS HOPKINS UNIV [US]
THE JOHNS HOPKINS UNIVERSITY

Absstract of: WO2025264967A1

The present disclosure relates to methods of preventing, or delaying the progression of, death of neurons and/or microgliosis and/or astrogliosis that contributes to the death of neurons. The present disclosure also relates to methods of treating, preventing, or delaying the progression of, a synucleinopathy (e.g., Parkinson's disease). Also disclosed are related in vitro, ex vivo, and in vivo methods of identifying agents and/or compositions useful for preventing, or delaying the progression of, death of neurons and/or microgliosis and/or astrogliosis that contributes to the death of neurons and agents and/or compositions useful for treating, preventing, or delaying the progression of, a synucleinopathy. The agents and/or compositions of the present disclosure decrease the level and/or activity of a cis-regulatory element that propagates the misfolding and aggregation of proteins encoded by synucleinopathy-associated genes in neurons and/or glial cells.

COMPOSITION COMPRISING HAPLN1 AS ACTIVE INGREDIENT OR PREVENTING OR TREATING SENILE DEGENERATIVE BRAIN DISEASES

Publication No.:  WO2025263948A1 26/12/2025
Applicant: 
CHUNG ANG UNIV INDUSTRY ACADEMIC COOPERATION FOUNDATION [KR]
\uC911\uC559\uB300\uD559\uAD50 \uC0B0\uD559\uD611\uB825\uB2E8

Absstract of: WO2025263948A1

The present invention relates to a composition comprising HAPLN1 as an active ingredient for preventing or treating senile degenerative brain diseases. Specifically, recombinant human HAPLN1 protein (rhHAPLN1) lowers the protein level of p16 in cultured human astrocytes to inhibit cellular senescence caused by the accumulation of beta amyloid peptides, and further inhibits phosphorylation (p-p38 MAPK) of p38 MAPK protein, thereby also having the possibility of inhibiting inflammatory responses associated with the onset of Alzheimer's disease and Parkinson's disease. In addition, the recombinant human HAPLN1 protein (rhHAPLN1) exhibits significant memory and learning improvement effects in in vivo experiments performed using a mouse acute Alzheimer's disease model, and thus can be expected to exhibit preventive and therapeutic effects against Alzheimer's disease that may occur with aging and the like. In addition, the inhibitory effect of the rhHAPLN1 protein on cellular senescence and inflammatory responses of astrocytes can provide a very important clue for establishing prevention and treatment strategies not only for aging itself but also for brain functions, motor behaviors, memory, seizures, dementia, brain tumors, and the like.

APP-TARGETING RNA INTERFERENCE METHOD, NUCLEIC ACID, AND USE THEREOF

Publication No.:  WO2025261396A1 26/12/2025
Applicant: 
EXORNA BIOSCIENCE NANJING CO LTD [CN]
EXORNA BIOSCIENCE SHANGHAI CO LTD [CN]
\u827E\u7801\u751F\u7269\u79D1\u6280\uFF08\u5357\u4EAC\uFF09\u6709\u9650\u516C\u53F8,
\u827E\u7801\u7533\u534E\u751F\u7269\u79D1\u6280\uFF08\u4E0A\u6D77\uFF09\u6709\u9650\u516C\u53F8

Absstract of: WO2025261396A1

Provided is a nucleic acid molecule used for regulating the level or amount of APP mRNA. Specifically, provided is delivery of primary microRNA for formation of pre- and microRNA following in-vivo processing and for use in the in-vivo inhibition of APP mRNA expression. Provided is a delivery system for the nucleic acid molecule, the delivery system comprising a carrier, an exosome, and a cell, and a pharmaceutical composition containing same. Provided is the use of the nucleic acid molecule and the delivery system in amyloidosis treatment and drug preparation, in particular a method and a drug for Alzheimer's disease.

CRYSTALLINE FORMS OF 3-(5-(4-((3,3-DIMETHYL-4-((1-(6-(5-(1-METHYLCYCLOPROPOXY)-1HINDAZOL-3-YL)PYRIMIDIN-4-YL)PIPERIDIN-4-YL)METHYL)PIPERAZIN-1-YL)METHYL)PIPERIDIN-1-YL)-4-FLUORO-1-OXOISOINDOLIN-2-YL)PIPERIDINE-2,6-DIONE

Publication No.:  WO2025265035A1 26/12/2025
Applicant: 
ARVINAS OPERATIONS INC [US]
ARVINAS OPERATIONS, INC

Absstract of: WO2025265035A1

Provided are crystalline forms of 3-(5-(4-((3,3-dimethyl-4-((1-(6-(5-(1- methylcyclopropoxy)-1H-indazol-3-yl)pyrimidin-4-yl)piperidin-4-yl)methyl)piperazin-1- yl)methyl)piperidin-1-yl)-4-fluoro-1-oxoisoindolin-2-yl)piperidine-2, 6-dione, pharmaceutical compositions comprising the crystalline form, and their use in treating conditions associated with LRRK2, such as Parkinson's disease, dementia, Progressive Supranuclear Palsy, Huntington's disease, or neuroinflammation.

ANTI-SOD1 NANOBODIES

Publication No.:  US2025388697A1 25/12/2025
Applicant: 
UNIV OF MASSACHUSETTS [US]
University of Massachusetts
WO_2023250016_A2

Absstract of: US2025388697A1

Composition and methods of diagnosing, monitoring, and treating subjects with a motor neuron pathology, such as motor neuron disorders (including but not limited to amyotrophic lateral sclerosis (ALS)) and neuropathies.

(AZA)SPIROHEPTANE DERIVATIVES FOR THE TREATMENT OF NEURODEGENERATIVE DISORDERS

Publication No.:  US2025388592A1 25/12/2025
Applicant: 
REMYND N V [BE]
REMYND N.V
JP_2025527459_PA

Absstract of: US2025388592A1

The application relates to compounds of formula (B1A), (B1B), (B1C) or a salt, a solvate, a hydrate, a polymorph, a tautomer, a racemate or a stereoisomer thereof, to pharmaceutical compositions comprising such a compound, and to the compounds for use as a medicine, in particular for use in the treatment of neurodegenerative disorders such as Alzheimer's disease.

COMPOSITIONS AND METHODS FOR THE TREATMENT OF PARKINSON S AND GAUCHER DISEASE

Nº publicación: EP4667461A2 24/12/2025

Applicant:

CHILDRENS HOSPITAL MED CT [US]
Children's Hospital Medical Center

EP_4667461_A2

Absstract of: EP4667461A2

Compounds and pharmaceutical compositions are useful for the treatment of Gaucher disease and Parkinson's disease. Methods of treating Gaucher disease or Parkinson's disease include administration of one or more compounds or pharmaceutically acceptable salts or prodrugs thereof to a subject diagnosed with, or at risk of developing Gaucher disease or Parkinson's disease.

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