Absstract of: WO2025136887A1
The present disclosure describes compounds and methods for disrupting the amyloid cascade.
Absstract of: WO2025134068A1
The invention provides antibody conjugate compositions comprising an antibody targeting a pathological protein covalently attached to one or more brain penetrant, pathological protein binding small molecule entities by a linker. The invention further provides methods of treating neurodegenerative diseases and disorders such as Alzheimer's disease with the antibody conjugate compositions.
Absstract of: US2025205239A1
The present invention provides a composition for preventing or treating a neurodegenerative disease containing a phosphodiesterase 5 inhibitor (PDE5 inhibitors) and an acetylcholinesterase inhibitor (AChEI) and a method using thereof, wherein the PDE5 inhibitor is selected from among mirodenafil, sildenafil, vardenafil, tadalafil, udenafil, dasantafil, avanafil, pharmaceutically acceptable salts, solvates, hydrates, and a mixture thereof; and the AchEI is selected from among donepezil, rivastigmine, galantamine, physostigmine, tacrine, metrifonate, phenserine, tolserine, eseroline, huperizine A and B, galangin, cardanol, donepezil-AP2238, donepezil-tacrine, tacrine-ferulic acid hybrid, tacrine-hydroxyquinoline, ladostigil, indenyl derivatives, pharmaceutically acceptable salts, solvates, hydrates and a mixture thereof; and the neurodegenerative disease is dementia, Parkinson's disease (PD), Alzheimer's disease (AD), Huntington's disease (HD) or Multiple sclerosis (MS).
Absstract of: US2025205281A1
The present invention relates to a pharmaceutical composition for preventing and treating a degenerative brain disease, specifically dementia, comprising an activated natural killer cell as an active ingredient. The pharmaceutical composition comprising a mouse-derived activated natural killer cell as an active ingredient, according to the present invention, regulates the activity of microglial cells and inhibits the deposition of amyloid β plaques. In addition, the pharmaceutical composition showed an excellent effect on cognitive function improvement in an animal model of dementia. Furthermore, when a human peripheral blood mononuclear cell-derived natural killer cell and a human induced pluripotent stem cell-derived natural killer cell were activated, it was confirmed that the expression of some genes involved in restoring the activity of microglial cells was similar to or higher than that of a mouse-derived activated natural killer cell. Therefore, the pharmaceutical composition of the present invention can be effectively used for preventing or treating a degenerative brain disease, such as dementia, Parkinson's disease, and Huntington's disease, and improving cognitive impairment.
Absstract of: US2025205368A1
The present invention relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated. for example. for treatment of lower motor neuron diseases such as SMA and ALS as well as Pompe disease and lysosomal storage disorders.
Absstract of: WO2025130951A1
The present invention provides crystalline forms of 6-2-1-(2-pyridylmethyl)-4-piperidylethylspiro1,3dioxolo4,5-fisoindole-7,1'-cyclopropane-5-one phosphate (AD-35), and preparation methods therefor. The invention further provides a use of the crystalline forms of AD-35 in the preparation of a drug for treating Alzheimer's disease.
Absstract of: US2025205321A1
The present invention relates to therapeutical uses of non-classical human major histocompatibility complex (MHC) molecules (also named MHC class Ib molecules) in combination with peptide antigens for the treatment of Parkinson's disease. The invention more specifically relates to recombinant polypeptides comprising peptide antigens and one or more domains of a non-classical MHC class Ib molecule. The invention also relates to methods of producing such recombinant polypeptides, pharmaceutical compositions comprising the same, as well as their uses for treating Parkinson's disease.
Absstract of: US2025205305A1
The present disclosure provides to systems, methods, and compositions for rescuing protein misfolding and preventing protein aggregation. Particularly the present disclosure provides methods and compositions comprising DNAJB6 or variants thereof, or polynucleotides encoding DNAJB6 or variants thereof. The present disclosure also provides methods for treating protein misfolding and/or protein aggregation diseases (e.g., multiple amyotrophic lateral sclerosis and frontotemporal dementia) by administering the systems or compositions to a subject in need thereof.
Absstract of: WO2025131275A1
The present invention relates to pharmaceutical compositions and medicaments comprising the compound of formula (I); or a stereoisomer, tautomer, pharmaceutically usable solvate or salt thereof, and dosage regimens for the administration thereof to human patients.
Absstract of: WO2025137210A1
The present disclosure relates to a compound of formula (1) as an anhydrate which is in a crystalline Form 1, characterized by having a powder-X-ray diffractogram displaying a peak expressed as degree 2-Theta angles at about 8.3 and a solid form thereof. The present disclosure also relates to processes for its preparation, as well as a medicament and a pharmaceutical composition comprising it. The present disclosure further concerns the anhydrate crystalline Form 1 of compound of formula (1) for use as a medicine and more particularly in the treatment of Alzheimer disease, multiple sclerosis, and amyotrophic lateral sclerosis (ALS).
Absstract of: WO2025137077A1
Compositions and methods are disclosed herein for the treatment of Alzheimer's disease with allogeneic mesenchymal stem cells. The methods of treatment involve the administration of a composition of allogeneic mesenchymal stem cells to a subject in need thereof, wherein the efficacy of the treatment methods can be determined through the measurement of specific biomarkers and improved cognitive function and/or quality of life.
Absstract of: EP4573926A1
The present invention relates to a composition for preventing, ameliorating, or treating Parkinson's disease comprising osmotin protein as effective component, and, by having the effects of alleviating behavior and motor skill deficits induced by MPTP/α-synuclein, protecting dopaminergic neurons, regulating the expression level of neuroinflammation-related proteins, inhibiting apoptotic cell death induced by MPTP/α-synuclein, alleviating cell damage caused by overexpression of α-synuclein (A53T), reducing the accumulation of α-synuclein caused by activation of AMPK and subsequent autophagy, regulating the dendritic complex structure, increasing the spine density in pyramidal neurons, alleviating the cognitive deficits, and restoring the expression of synaptic markers, the osmotin protein can be advantageously used for a functional health food or a pharmaceutical product for preventing, ameliorating, or treating Parkinson's disease.
Absstract of: CN119907664A
A method of treating Parkinson's disease in a patient that accepts N doses per day of levodopa to provide X mg total daily dose of levodopa and that begins to experience motion fluctuations or begin to show a sign of "hypoefficacy", the treatment comprises administering more than N doses per day of levodopa to provide X mg of a total daily dose of levodopa, and administering Y mg of opirapone in a single daily dose, where X is from 100 to 1000, N is from 2 to 10, and Y is from 25 to 50.
Absstract of: WO2025128777A1
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, osteoarthritis, atherosclerosis, heart failure, idiopathic pericarditis, myocarditis, atopic dermatitis, hidradenitis suppurativa, inflammatory bowel disease, cancer, Alzheimer's Disease, Parkinson's Disease, dementia with Lewy bodies (DLB), and traumatic brain injury.
Absstract of: WO2025125071A1
Cathepsin D peptides are provided that are capable of interacting with and/or inhibiting the formation of beta amyloid aggregates, thereby leading to a competitive reduction of amyloid- amyloid interactions. Also provided are recombinant expression vectors encoding said peptides as well as of pharmaceutical formulations comprising said peptide-analogues. Said peptides, compositions and recombinant vectors are useful as therapeutic agents in the treatment and/or amelioration of the symptoms of amyloidoses such as Alzheimer's disease.
Absstract of: WO2025126158A2
SnRNA systems targeting SOD1 RNA sequences are disclosed herein. Further disclosed are methods of treating Amyotrophic Lateral Sclerosis.
Absstract of: JP2025023319A
To provide compositions and methods for treating and preventing amyotrophic lateral sclerosis.SOLUTION: Dosage regimens for SOD1-targeting antisense oligonucleotides and salts thereof are provided. These dosage regimens find use in the treatment of subjects having or at risk of developing amyotrophic lateral sclerosis. In a first aspect, the disclosure provides a method of treating or preventing amyotrophic lateral sclerosis associated with a mutation in the human superoxide dismutase 1 (SOD1) gene in a human subject in need thereof.SELECTED DRAWING: None
Absstract of: WO2025128134A1
A composition and a method of treatment for treatment of diseases related to overexpression of SNCA gene in a subject such as Parkinson's disease comprising one or more clustered regularly interspaced short palindromic repeats associated protein (Cas9) or a variant thereof and one or more guide ribonucleic acids (RNAs). The present invention also provides a nanoparticle encapsulating any embodiment of the composition of the present invention.
Absstract of: AU2023403437A1
The present disclosure relates to compositions comprising trehalose and methods of using same for the treatment of Huntington's disease.
Absstract of: US2025195511A1
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, osteoarthritis, atherosclerosis, heart failure, idiopathic pericarditis, myocarditis, atopic dermatitis, hidradenitis suppurativa, inflammatory bowel disease, cancer, Alzheimer's Disease, Parkinson's Disease, dementia with Lewy bodies (DLB), and traumatic brain injury.
Absstract of: US2025197862A1
Provided herein are artificial microRNA (miRNA) molecules for treating tauopathies. In some embodiments, the miRNA molecules target expression of Tau protein. Further provided herein are expression constructs, vectors (e.g., rAAV), cells, viral particles, and pharmaceutical compositions containing the artificial miRNA molecules. Yet further provided herein are methods and kits related to the use of the miRNA molecules, for example, to treat tauopathies including is Alzheimer's disease, progressive supranuclear palsy, corticobasal degeneration, frontotemporal dementia with parkinsonism-17, Pick's Disease, argyrophilic grain disease, globular glial tauopathy, chronic traumatic encephalopathy and post-encephalitic parkinsonism.
Absstract of: US2025197375A1
Disclosed are compounds comprising pyrimidinyl core and pharmaceutical compositions useful in the treatment of neurological disorders. The compounds described herein, alone or in combination with other pharmaceutically active agents, can be used for treating or preventing neurological diseases, including FTLD-TDP, chronic traumatic encephalopathy, ALS, Alzheimer's disease, LATE, or frontotemporal lobar degeneration.
Absstract of: US2025195554A1
A gene expression regulator containing microparticles containing miRNA which targets a gene related to the expression of at least one kind of protein of amyloid precursor protein (APP), β-secretase (BACE1), an NMDA-activating protein, glycogen synthase kinase-3β (GSK-3β) and polyglutamine-binding protein-1 (PQBP1) can provide a novel gene expression regulator which can regulate (suppress, inhibit or the like) the expression of a protein related to amyloid β-related or tau protein-related dementia or brain inflammation such as Alzheimer's disease; an agent for preventing or treating Alzheimer's disease; and a method for improving dementia.
Absstract of: US2025195457A1
Disclosed is a pharmaceutical composition for treatment of Parkinson's disease comprising N-(1S)-2,2,5,7-tetrafluoro-2,3-dihydro-1H-inden-1-ylsulfamide represented by formula (1) or a pharmaceutically acceptable salt thereof.
Nº publicación: WO2025128781A1 19/06/2025
Applicant:
MERCK SHARP & DOHME LLC [US]
MERCK SHARP & DOHME LLC
Absstract of: WO2025128781A1
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.