Resumen de: WO2026190230A1
The present invention relates to alpha-synuclein antibodies which are useful in diagnostic assays for detecting aggregates, oligomers and fibrillar forms of alpha- synuclein in patient samples. These antibodies enable diagnosis and efficient treatment of alpha-synucleinopathies such as Parkinson's Disease (PD) or Multiple System Atrophy (MSA) patients by assessing and monitoring the pathological alpha-synuclein levels in patients.
Resumen de: WO2026193123A1
The present disclosure provides combinations of anti-CD3 antibodies (e.g. Foralumab), anti-amyloid therapies, and anti-tau therapies for the treatment of Alzheimer's disease.
Resumen de: WO2026188369A1
The present invention belongs to the technical field of biopharmaceuticals and specifically relates to use of a pharmaceutical composition in the preparation of a formulation for treating Parkinson's syndrome. Research has found that NPAFP1, NPAFP4, and NPAFP12 proteins can ameliorate the pathological features of a PD model by increasing the fluidity of α-synuclein.
Resumen de: WO2026193437A1
The present disclosure provides methods and kits related to treatment of Parkinson's disease in a subject by reducing the expression of a CaV1.3 protein in a target cell by administering to the subject a therapeutically effective amount of an engineered genetic system.
Resumen de: WO2026189517A1
Disclosure herein Mirtazapine for use in treating bulbar-onset ALS (amyotrophic lateral sclerosis), and kit comprising Mirtazapine for treating bulbar-onset ALS. Also disclosed herein a method for treating bulbar-onset ALS comprising administration of Mirtazapine, optionally with an additional therapy for treating ALS.
Resumen de: WO2026193281A1
Provided herein are compositions and methods for reducing expression of RBM39 and for treating diseases and/or conditions characterized by increased expression of RBM39 (e.g., a cancer) in subjects in need thereof, by administering compositions comprising compounds described herein. Also provided herein are compositions and methods for reducing expression of MSH3 and for treating diseases and/or conditions characterized by increased expression of MSH3 (e.g., Huntington's Disease) in subjects in need thereof, by administering compositions comprising compounds described herein.
Resumen de: AU2025215085A1
Disclosed herein is a compound of Formulas (I) and (II) as an FTO inhibitor with novel structures. Also disclosed herein is a pharmaceutical composition comprising the same, and a method of inhibiting weight gain, promoting weight loss, reducing serum LDL, cholesterol, LDL-c, or triglycerides, or treating obesity or an obesity-related disease (esp. obesity-related diabetes, hyperglycemia, diabetic nephropathy, hyperlipemia, coronary heart disease, atherosclerosis, hypertension, cardiovascular or cerebrovascular disease) or Alzheimer's disease by inhibiting FTO by using the compound disclosed herein.
Resumen de: AU2025225323A1
The present invention relates to a solid composition, and its use in the treatment of Parkinson's disease, comprising at least two compartments, a first active pharmaceutical ingredient (API) and a second API, preferably for increasing uptake of the second API thereby optimizing its use in the treatment of Parkinson's disease, in particular improving its clinical activity and reducing its side effects associated with the treatment of Parkinson's disease.
Resumen de: AU2026226429A1
An effective amount of quercetin, vitamin B3, vitamin C, zafirlukast and optionally folic acid for use in treating amyotrophic lateral sclerosis in a subject. Also disclosed are said combination for use in reducing, slowing or abating the progression of amyotrophic lateral sclerosis or a symptom thereof. ep e p
Resumen de: US20260275351A1
Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a Superoxide Dismutase 1 (SOD1) gene. The SOD1 RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an SOD1 gene. Pharmaceutical compositions that include one or more SOD1 RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described SOD1 RNAi agents to central nervous system (CNS) tissue, in vivo, provides for inhibition of SOD1 gene expression and a reduction in SOD1 activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including amyotrophic lateral sclerosis (ALS).
Resumen de: US20260272976A1
Described herein are methods of monitoring and treating a subject with ALS based on biomarkers.
Resumen de: US20260275333A1
Disclosed herein are oligonucleotides with one or more spacers or without a spacer. In various embodiments, STMN2, KCNQ2, UNC13A, or SMN2 oligonucleotides with spacer(s) reduce STMN2 transcripts with cryptic exons or reduce mis-spliced KCNQ2, UNC13A, or SMN2 transcripts and increase full length STMN2, KCNQ2, UNC13A, SMN transcripts, thereby imparting therapeutic efficacy against neurological diseases such as amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), Alzheimer's disease (AD) or spinal muscular atrophy (SMA).
Resumen de: WO2025101652A1
Described herein are compounds that are MST1 inhibitors, methods of making such compounds, pharmaceutical compositions and medicaments comprising such compounds, and methods of using such compounds in the treatment of conditions, diseases, or disorders that would benefit from modulation of MST1 activity, such as diabetes and liver regeneration.
Resumen de: EP4806513A2
Die vorliegende Erfindung Erfindung betrifft Derivate der Malonsäure zur Verwendung als Arzneimittel zur Behandlung einer neurodegenerativen Erkrankung bei einem Patienten, dadurch gekennzeichnet, dassa) das Malonsäure-Derivat der nachstehenden Formel (I) entspricht:worin X und Y jeweils unabhängig aus -O-M+, -OR1 und -NR2R3 ausgewählt sind, worin M+ aus ein- oder mehrwertigen Metallkationen ausgewählt ist und R1, R2 und R3 jeweils unabhängig aus Wasserstoff und gesättigten oder ungesättigten, unverzweigten, verzweigten oder zyklischen Kohlenwasserstoff-Resten mit 1 bis 15 Kohlenstoffatomen ausgewählt sind, worin ein oder mehrere Kohlenstoffatome gegebenenfalls durch O oder N ersetzt sind, wobei R2 und R3 gegebenenfalls miteinander verbunden sind und zusammen mit dem Stickstoffatom einen heterozyklischen Kohlenwasserstoff-Rest bilden; mit der Maßgabe, dass R2 und R3 keine Phenylreste sind; undb) die Behandlung der neurodegenerativen Erkrankung mittels Förderung der Metallothionein-Biosynthese zur Wiederherstellung der Metallhomöostase des Patienten erfolgt;und mit der Maßgabe, dass das Malonsäure-Derivat der Formel (I) keines der folgenden ist: Malonsäure, Malonsäurediamid, Dinatriummalonat, Malonsäuredimethylester, Malonsäuremethylester-Kaliumsalz, Malonsäuremethylesteramid, N-(3-Aminopropyl)-malonsäureamid, Malons
Resumen de: KR20260136354A
본 발명은 바닐릭산 유도체 화합물에 관한 것으로서, 바닐릭산과 카테콜아민 간의 탈수축합반응에 의해 형성될 수 있다. 본 발명에 따른 바닐릭산 유도체 화합물은 알츠하이머병 및 신경염증 치료제제로 사용할 수 있다는 점에 특징이 있다. 또한 본 발명은 상기 화합물을 포함하는 알츠하이머 치료제 조성물 및 신경염증 치료제 조성물에 관한 것이다.
Resumen de: KR20260135747A
본 발명은 HCN1 채널의 CNBD 도메인에 특이적으로 결합하는 세계 최초의 소분자 개방제(ind_5F_3pyr, 5-플루오로-1H-인다졸-3-일-피리딘-3-일)에 관한 것이다. 알츠하이머병에서 HCN1 채널 발현이 감소하여 신경 흥분성 이상이 발생하며, 기존 HCN 약물은 모두 차단제로서 이를 해결할 수 없다. 본 발명의 화합물은 HCN1 CNBD에 cAMP보다 강하게 결합하고(ΔG = -8.08 vs -7.51 kcal/mol), 열린 상태를 안정화하며(apo ΔG = -8.32), 혈액뇌장벽을 투과하고(MW 213, TPSA 57), TRIP8b 대체를 통한 뇌 선택성을 달성한다. 119개 후보 중 가상 스크리닝과 분자동역학으로 동정되었다. JPEGpat00001.jpg5157
Resumen de: WO2026184543A1
Provided are a magnetogenetic system based on a clMagR gene and a use thereof in Parkinson's disease. The clMagR gene is injected into neurons by means of an adeno-associated virus (AAV) vector, and on the basis of exogenous iron and magnetic field stimulation technology, a biological function of cells or tissues is regulated. Compared with optogenetics, the system avoids optical fiber implantation and direct light source irradiation required for optogenetics by means of precise control of an external magnetic field, reduces the risk of invasive surgery, and can more widely regulate cell functions in deep neural regions in the body, especially in deep brain regions. Compared with traditional treatment methods, the present invention provides a non-invasive and regulatable gene therapy strategy, providing a new technical paradigm for fields such as stem cell therapy, nerve regeneration, drug delivery, and elucidation of the neural circuit mechanism of diseases.
Resumen de: US20260263373A1
Nanoparticle compositions and methods inhibiting amyloid beta (Aβ) fibrilization in a subject in need thereof using nanoparticles to target fibril β-amyloid (fAβ)-specific scavenger receptors. Also provided is a method of using nanoparticle compositions to treat Alzheimer's disease (AD) and similar amyloidopathies.
Resumen de: AU2025234423A1
Provided are compositions for delivery of zinc finger fusion proteins that inhibit expression of tau in the nervous system using the blood-brain barrier penetrant AAV capsid proteins comprising SEQ ID. NO: 1235, and methods of using the compositions to treat neurodegenerative diseases such as Alzheimer's disease, frontotemporal dementia, and other tauopathies.
Resumen de: AU2025232421A1
Described herein, in part, are methods useful for preventing and/or treating a disease or condition relating to aberrant function or activity of a T-type calcium channel, such as Parkinson's disease, psychiatric disorders (e.g., mood disorder (e.g., major depressive disorder)), pain, tremor (e.g., essential tremor), seizures (e.g., absence seizures), epilepsy, or an epilepsy syndrome (e.g., juvenile myoclonic epilepsy). The present invention further comprises methods for modulating the function of a T-type calcium channel and methods of administering a titrated dosage of a T-type calcium channel antagonist.
Resumen de: AU2025231257A1
The present invention relates to peptides for treatment of cognitive diseases, in particular Alzeheimer's Disease, mild cognitive impairment due to Alzheimer's disease, and mild dementia due to Alzheimer's disease.
Resumen de: WO2026183787A1
Provided is a compound or a pharmaceutically acceptable salt, hydrate or solvate thereof for use in the treatment of amyotrophic lateral sclerosis represented by the following Formula (I). Also provided is a pharmaceutical composition comprising the above compound or a pharmaceutically acceptable salt, hydrate or solvate thereof for use in the treatment of amyotrophic lateral sclerosis.
Resumen de: WO2026185106A1
The present invention relates to the use of a pharmaceutical combination comprising molecules that rebilance the pathway of the epigenetic modulators LiN28, EZH2 and Let-7 and increases the expression of GFAP protein in particular in the treatment of all the diseases of the central nervous system (CNS) wherein LiN28, EZH2 and Let-7 dysregulation influence the expression of GFAP gene, selected from Huntington's disease, Amyotrophic Lateral Sclerosis (ALS), Schizophrenia, Bipolar Disorder, Fetal alcohol spectrum disorder and Autism. The invention further relates to the use of a pharmaceutical composition comprising said combination and at least one pharmaceutical acceptable excipient in the treatment and/or prevention of neurodegenerative diseases and neurodevelopment diseases.
Resumen de: WO2026188235A1
The present disclosure relates to methods of preventing and/or treating neuroinflammation in a subject in need thereof. The present disclosure also relates to methods and systems for modeling aging related neurodegenerative diseases (e.g., Amyotrophic lateral sclerosis (ALS)) in vitro. The methods comprise administering one or more Angiotensin II Receptor I Blocker (ARB) to the subject. The present disclosure also relates to methods and systems for modeling aging related neurodegenerative diseases (e.g., ALS) in vitro.
Nº publicación: WO2026188223A1 10/09/2026
Solicitante:
UNIV OF MIAMI [US]
UNIVERSITY OF MIAMI
Resumen de: WO2026188223A1
The compositions and methods described herein include methods and agents that inhibit inflammasome signaling in a mammal such as antibodies directed against inflammasome components used alone or in combination with extracellular vesicle uptake inhibitor(s) or other agents. Also described herein are compositions and methods of use thereof for detecting and treating early-stage Alzheimer's disease as well as other inflammatory neurologic conditions such as Huntington's Disease.