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120
results.
Updated on
13/07/2025 [07:36:00]
Absstract of: US2025223589A1
Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate diseases, including neurodegenerative diseases, such as Huntington's Disease (HD).
Absstract of: US2025222072A1
Aspects of the disclosure relate to compositions and methods useful for treating Parkinson's disease. In some embodiments, the disclosure provides a method for treating Parkinson's disease comprising administration of a viral vector comprising a GDNF nucleic acid sequence. In some embodiments, administration is locally to the subject putamen. In some embodiments, administration is systemically, e.g., via the viral vector comprising a modified viral capsid, such as for preferentially targeting cells in the CNS or PNS.
Absstract of: US2025223290A1
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.
Absstract of: EP4582144A2
The invention relates to novel monoclonal anti-alpha-synuclein antibodies. 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.
Absstract of: EP4582412A1
The present invention relates to: a method for preparing a chiral gamma lactam derivative or a pharmaceutically acceptable salt thereof by using a chiral organocatalytic compound; and a composition for preventing, alleviating or treating muscle diseases, mental diseases, or neurodegenerative diseases, comprising the derivative or the pharmaceutically acceptable salt thereof. The chiral gamma lactam derivative or the pharmaceutically acceptable salt thereof, of the present invention, has an effect of inhibiting MAO-B and MSTN, targets D1-mClu5, and can be used in the prevention, alleviation, or treatment of muscle diseases including sarcopenia, mental diseases including depression, neurodegenerative diseases including Parkinson's disease, and the like.
Absstract of: WO2025141029A1
Disclosed are compounds of formula (I), or a pharmaceutically acceptable salt thereof. Also disclosed are a medicament and a pharmaceutical composition comprising said compounds of formula (I), and said compounds (I) for use in the treatment of a neurodegenerative disease such as Parkinson's disease. Further disclosed are a solid form of a compound of Formula (I-a), characterized as crystalline Form A, as well as a pharmaceutical composition comprising said solid form, and said solid form for use in treating a neurodegenerative disease.
Absstract of: WO2025141063A1
The present invention relates to Phosphatidylinositol 3,5-bisphosphate (PI(3,5)P2) of formula (I), preferably formulated in LNP, preferably SANP, and optionally together with a miRNA or antagomir, for use in the treatment of neurodegenerative disorders, like AD, PD and ALS, characterized by impaired/blocked autophagy. It also relates to a LNP, preferably SANP, containing PI(3,5)P2 and optionally a miRNA or antagomir.
Absstract of: AU2023408627A1
The present invention is directed to the compound golexanolone for use in the treatment of Parkinson's Disease (PD) or for use in the treatment of L-dopa Induced Dyskinesia (LID) in Parkinson's Disease (PD) patients. Further, the present invention is directed to the compound golexanolone or a pharmaceutically acceptable salt thereof, for use in the treatment of Parkinson's Diseases (PD) patients, in particular PD patients exhibiting a L-dopa Induced Dyskinesia (LID).
Absstract of: US2025213507A1
Tramiprosate and derivatives thereof are provided herein for treating neurodegenerative disorders such as Alzheimer's disease (AD).
Absstract of: US2025213619A1
A composition for treating intractable neurological diseases contains culture supernatant obtained by culturing deciduous dental pulp stem cells in serum-free medium under ultrasound irradiation. A pharmaceutical product for the treatment of amyotrophic lateral sclerosis (ALS) and Alzheimer's disease contains the composition for the treatment of intractable neurological diseases. In a method for producing a composition for treating intractable neurological diseases, the composition contains a culture supernatant obtained by culturing deciduous dental pulp stem cells under ultrasound irradiation using serum-free medium.
Absstract of: US2025213622A1
The use of extract from rabbit skin inflamed by vaccinia virus for treating Parkinson's disease or restoring neurological function of the brain or alleviating damage to neurological function of the brain in a patient suffering from Parkinson's disease is provided. The extract from rabbit skin inflamed by vaccinia virus can be Lepalvir.
Absstract of: US2025213564A1
Methods and compositions that treat Alzheimer's disease and other neurodegenerative diseases and/or to ameliorate or improve symptoms associated with Alzheimer's disease. In some aspects, the compositions and methods use a serotonin 4 receptor (5-hydroxytryptamine (serotonin) receptor 4, or 5-HT4R) agonist in combination with: (R,S)-ketamine, a (R,S)-ketamine analog, or a pharmaceutically acceptable salt, derivative, or metabolite thereof; an antagonist of the glutamate N-methyl-D-aspartate (NMDA) receptor (NMDAR); or an agonist of the α-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid (AMPA) receptor (AMPAR).
Absstract of: US2025213611A1
The present disclosure provides, among other things, methods for the treatment of neurodegenerative diseases (ND) and other mitochondrial disorders, and compositions related thereto. Described herein are in vitro (cell culture) and in vivo (animal model) experimental examples demonstrating mitochondrial organelle transplantation (MOT) for the treatment of NDs such as amyotrophic lateral sclerosis (ALS). Furthermore, as discussed herein, MOT has been performed in five human ALS patients with positive results—measurable improvement of their conditions has been observed, with no adverse events.
Absstract of: US2025214948A1
There is provided herein a compound of formula I,wherein R1, R2, n, X, Q, L, m, R3 and p are as defined herein, which compounds are useful in the treatment of treatment of diseases characterised by impaired signalling of neurotrophins and/or other trophic factors, such as Alzheimer's disease and the like.
Absstract of: US2025214984A1
Disclosed are compounds of following formula (I):in which all symbols have the same meanings as the definitions described in the specification; or a salt thereof. The compounds or a salt thereof are useful as a prophylactic and/or therapeutic agent for cancer, Huntington's disease, Alzheimer's disease and the like.
Absstract of: AU2025204524A1
Abstract The invention provides novel dosing regimens for Leuco-Methylthioninium (LMT) compounds which maximise the proportion of subjects in which the MT concentration will exceed concentrations in which therapeutic efficacy in relation to treatment of neurodegenerative disorders such as Alzheimer's disease and rontotemporal dementias can be achieved, while maintaining a desirable clinical profile. Also provided are LMT- containing dosage units and other compositions. Abstract The invention provides novel dosing regimens for Leuco-Methylthioninium (LMT) compounds which maximise the proportion of subjects in which the MT concentration will exceed concentrations in which therapeutic efficacy in relation to treatment of neurodegenerative disorders such as Alzheimer's disease and rontotemporal dementias can be achieved, while maintaining a desirable clinical profile. Also provided are LMT- containing dosage units and other compositions. un b s t r a c t u n h e i n v e n t i o n p r o v i d e s n o v e l d o s i n g r e g i m e n s f o r e u c o - e t h y l t h i o n i n i u m ( ) c o m p o u n d s w h i c h m a x i m i s e t h e p r o p o r t i o n o f s u b j e c t s i n w h i c h t h e c o n c e n t r a t i o n w i l l e x c e e d c o n c e n t r a t i o n s i n w h i c h t h e r a p e u t i c e f f i c a c y i n r e l a t i o n t o t r e a t m e n t o f n e u r o d e g e n e r a t i v e d i s o r d e r s s u c h a s l z h e i m e r ' s d i s e a s e a n d r o n t o t e m p o
Absstract of: WO2025143100A1
The present invention addresses the problem of providing a medicine that, despite of being a non-ergot dopamine agonist (DA), reduces the risk of the drowsiness side effect, and exhibits an excellent therapeutic effect on neurodegenerative disorders such as Parkinson's disease (PD). The present invention relates to a pharmaceutical composition for treating neurodegenerative diseases such as Parkinson's disease, the pharmaceutical composition comprising 1-{(4aR,6R,8aR)-2-amino-3-cyano-8-methyl-4,4a,5,6,7,8,8a,9-octahydrothieno3,2-gquinolin-6-ylcarbonyl}-3-2-(dimethylamino)ethyl-1-propylurea or a pharmacologically acceptable salt thereof. The pharmaceutical composition is characterized by being orally administered at a daily dose of 0.25 mg to 2 mg in terms of free form.
Absstract of: AU2025204414A1
Abstract Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SNCA mRNA in a cell or animal, and in certain instances reducing the amount of alpha-synuclein protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include motor dysfunction, aggregation of alpha-synuclein, neurodegeneration, cognitive decline and dementia. Such neurodegenerative diseases include Parkinson's disease, dementia withLewy bodies, diffuse Lewy body disease, pure autonomic failure, multiple system atrophy, neuronopathic Gaucher's disease and Alzheimer's disease. Abstract Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SNCA mRNA in a cell or animal, and in certain instances reducing the amount of alpha-synuclein protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include motor dysfunction, aggregation of alpha-synuclein, neurodegeneration, cognitive decline and dementia. Such neurodegenerative diseases include Parkinson's disease, dementia withLewy bodies, diffuse Lewy body disease, pure autonomic failure, multiple system atrophy, neuronopathic Gaucher's disease and Alzheimer's disease.
Absstract of: AU2024208288A1
Methods of treating a subject having parasomnias related to Rapid Eye Movement (REM) sleep and attenuating disease progression into synucleinopathies and Parkinson's disease by administering a formulation comprising an effective amount of piromelatine to the subject. Methods of treating a subject having parasomnias related to Rapid Eye Movement (REM) sleep and attenuating disease progression of parasomnias into chronic post-traumatic stress disorder (PTSD) by administering a formulation comprising of an effective amount of piromelatine to a subject.
Absstract of: EP4578461A2
Methods and pharmaceutical unit dosage forms for treating Parkinson's disease in a subject (e.g., an "off" episode in a subject having Parkinson's disease) are described. The pharmaceutical unit dosage forms are films having a first portion including particles containing an acid addition salt of apomorphine and a second portion containing a pH neutralizing agent. The pharmaceutical unit dosage forms can be flexible and have toughness greater than 100 g × mm. The methods can involve administering to a subject having Parkinson's disease a therapeutic dose sufficient to produce an apomorphine plasma concentrate of at least 2.64 ng/mL within 45 minutes after the administration. The subject may be identified as having low uptake, medium uptake, or high uptake of apomorphine administered via oral mucosa.
Absstract of: WO2024044355A2
Methods and compositions that treat Alzheimer's disease and other neurodegenerative diseases and/or to ameliorate or improve symptoms associated with Alzheimer's disease. In some aspects, the compositions and methods use a serotonin 4 receptor (5-hydroxytryptamine (serotonin) receptor 4, or 5-HT4R) agonist in combination with: (R,S)-ketamine, a (R,S)-ketamine analog, or a pharmaceutically acceptable salt, derivative, or metabolite thereof; an antagonist of the glutamate N-methyl-D-aspartate (NMDA) receptor (NMDAR); or an agonist of the α-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid (AMPA) receptor (AMPAR).
Absstract of: AU2023330511A1
The present disclosure relates to an sgRNA and its application in the preparation of a product for the treatment of Huntington's disease. The present disclosure was designed and screened to obtain an sgRNA targeting exon 1 of the human HTT gene as shown in SEQ ID NO: 1 or SEQ ID NO: 2. The CRISPR/Cas9 system mediated HTT gene knockout strategy based on this sgRNA and its high homologue sgRNA can efficiently knock out the human Huntingtin gene and achieve gene therapy for Huntington's disease.
Absstract of: WO2025136936A1
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.
Absstract of: AU2025204068A1
Abstract Provided herein are sulfopropanoic acid derivatives or pharmaceutically acceptable salts thereof, for treating a disease characterized by amyloid and amyloid-like aggregates, e.g., Alzheimer's disease.
Nº publicación: WO2025136898A1 26/06/2025
Applicant:
MERCK SHARP & DOHME LLC [US]
MERCK SHARP & DOHME LLC
Absstract of: WO2025136898A1
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.
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