Absstract of: WO2025199248A1
Applicants have discovered differential expression and activity of various miRNAs in neurodegenerative diseases. These miRNAs can serve as therapeutic targets for prevention and treatment of neurodegenerative diseases. Accordingly, embodiments of the invention include formulations and methods for treating neurodegenerative diseases such as dementia and Alzheimer's disease. The formulations and methods can include five microRNAs and two small molecule therapeutics. In aspects, the miRNAs are MIR-145-3p, let-7c-3p, MIR-383-5p, MIR-548aj-3p and MIR-548x-3p. In aspects, the small molecule therapeutics are GATC-49, fucosterol and fucoidan. In aspects, the compounds work synergistically with one another.
Absstract of: WO2025199495A1
The present invention provides methods and compositions for reducing internalization and/or trafficking of tan in neuronal cells comprising contacting the cells with an effective amount of VLDL receptor antagonist. The invention further provides a method of treating or preventing Alzheimer's disease in a subject in need thereof, comprising administering to the subject an effective amount of a VLDL receptor antagonist.
Absstract of: KR20250140146A
본 발명의 일 예는 락티플란티바실러스 플란타룸(Lactiplantibacillus plantarum) KACC 11451 균주, 락티플란티바실러스 파라플란타룸(Lactiplantibacillus paraplantarum) KACC 12373 균주 또는 락티플란티바실러스 펜토수스(Lactiplantibacillus pentosus) KACC 12428 균주에서 선택되는 1종 이상의 균주 및 L-티로신을 포함하는 파킨슨병 예방, 개선 또는 치료용 조성물을 제공한다. 본 발명의 일 예에 따른 특정 유산균은 L-티로신의 레보도파로의 전환 활성이 매우 높다. 또한, 본 발명의 일 예에 따른 특정 유산균 및 L-티로신을 포함하는 조성물은 생체내에서 레보도파를 생산할 수 있도록 제형화될 수 있고, 파킨슨병의 예방, 개선 또는 치료를 위한 건강기능식품 또는 의약품으로 사용될 수 있다.
Absstract of: WO2025199206A1
Provided herein are medical uses of CD11a-enriched T regulatory cells for treating amyotrophic lateral sclerosis (ALS) or ameliorating a symptom of ALS. Such uses may include longitudinal analysis of biomarkers, such as inflammatory cytokines.
Absstract of: WO2025199234A1
This disclosure relates to, inter alia, certain compounds, compositions, and pharmaceutical compositions thereof, that modulate the activity of the transporter protein vesicular monoamine transporter- 2 (VMAT2) and are directed to methods useful in the treatment of transporter protein vesicular monoamine transporter-2 mediated disorders, such as, neurological or psychiatric disease or disorders, including but not limited to, hyperkinetic movement disorders (e.g., tardive dyskinesia, Tourette's syndrome, Huntington's disease, tics, ataxia, chorea (such as, chorea associated with Huntington's disease), dystonia, hemifacial spasm, myoclonus, restless leg syndrome, and tremors). The disclosure further relates to synthetic methods and intermediates useful in the preparation of compounds.
Absstract of: WO2025199451A2
Compositions and methods are disclosed herein for the treatment of Alzheimer's disease with allogeneic mesenchymal stem cells (MSCs). The methods of treatment involve an administration of a composition of allogeneic mesenchymal stem cells to a subject in need thereof, wherein the effectiveness of the treatment methods can be determined through the measurement of specific biomarkers and improved cognitive or quality-of-life function.
Absstract of: EP4620959A1
The present invention discloses cyclic compounds as multi-target kinase inhibitors and preparation methods thereof. The multi-target kinase inhibitors of the present invention are as shown in general formula I, wherein R<sup>1</sup>, R<sup>2</sup>, R<sup>3</sup>, R<sup>3a</sup>, L<sup>1</sup>, L<sup>2</sup>, L<sup>3</sup>, ring A, and ring B are as shown in the Specification and Claims. The present invention also discloses preparation methods of general formula I and its inhibitory activity against multiple kinases. The compounds of general formula I described in the present invention can be used for treating cancers and neurodegenerative diseases such as Parkinson's disease, etc.
Absstract of: EP4620471A2
Pridopidine or a pharmaceutically acceptable salt thereof for use in treating a subject afflicted with amyotrophic lateral sclerosis (ALS).
Absstract of: US2025288701A1
The present invention relates to the technical field of nanotechnology and pharmaceutics, particularly, it relates to a reagent and pharmaceutical composition containing gold nanorods conjugated to D1 and Angiopep-2 peptides, and methods for detecting, diagnosing, evaluating the progression, and treating diseases related to β-amyloid plaques, such as Alzheimer's disease. Surprisingly, with the reagent and pharmaceutical composition of the present invention, a dose of gold per kg of body weight well below those previously reported is required, hence their applications are more economical, in addition to decreasing the likelihood of toxic effects, thanks to the low doses required to exert their function.
Absstract of: US2025290037A1
Modified cells that express and present or secrete at least one therapeutic molecule that can treat or ameliorate a disease of interest such as but not limited to Alzheimer's disease. In the modified cells, expression of the therapeutic molecule is induced when the modified cells are proximate to or in contact with pathology related to the disease of interest. The present disclosure also relates to compositions and kits comprising the disclosed cells. The present disclosure also relates to methods of using the disclosed cells for treating disease.
Absstract of: AU2024237252A1
Methods for preventing, limiting or delaying clinical motor progression in a subject with Parkinson's disease with low GCase activity, such as a PD patient with a pathogenic variant in the glucocerebrosidase 1 (GBA1) gene (GBA-PD) is provided, said methods comprising administering a therapeutically effective amount of 5,7-dimethyl-N-((1R,4R)-4- (pentyloxy)cyclohexyl)pyrazolol1,5-apyrimidine-3-carboxamide (Compound A), or a pharmaceutically acceptable salt thereof, to said subject.
Absstract of: WO2025192800A1
The present invention relates to a pharmaceutical composition for preventing or treating Alzheimer's disease, the composition comprising, as an active ingredient, neural crest-derived nasal turbinate stem cells (NTSCs) expressing SSEA3 and CD105. Treatment with the NTSCs expressing SSEA3 and CD105 or with an NTSC cell line including at least a predetermined proportion of the NTSCs was found to result in remarkably good therapeutic activity against Alzheimer's disease. Therefore, the present invention is expected to be effectively used not only as a composition for preventing or treating Alzheimer's disease in which the composition includes, as an active ingredient, NTSCs expressing SSEA3 and CD105 or an NTSC cell line including at least a predetermined proportion of the NTSCs, but also for uses such as screening of NTSC formulations that can be used to treat Alzheimer's disease, or prediction of the therapeutic efficacy thereof.
Absstract of: WO2025193743A1
Provided herein are systems and methods for introducing an A455D mutation into CSF2RB to neurodegenerative diseases, such as Alzheimer's disease.
Absstract of: WO2025193848A1
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.
Absstract of: US2025288615A1
Provided are inflammatory cytokine production inhibitors which inhibit the production of the inflammatory cytokines TNF-α and IL-1β and can be applied to drugs effective against inflammatory diseases caused by overproduction of these inflammatory cytokines, such as chronic inflammation (e.g., rheumatoid arthritis, ulcerative colitis) and Crohn's disease, as well as type 2 diabetes, depression, obesity, sepsis, atherosclerosis, dermatitis, dementia, schizophrenia, and Parkinson's disease, and also to foods and drinks such as health foods. The inflammatory cytokine production inhibitors contain as an active ingredient an enzyme-treated whey obtained by contacting whey with β-galactosidase. The enzyme-treated whey is obtained by further contacting with sialidase.
Absstract of: US2025288580A1
Applicant discloses methods and compositions for treating a patient suffering from amyotrophic lateral sclerosis (ALS) comprising administration of a heteroaryl ketone fused azadecalin compound. In embodiments, the heteroaryl ketone fused azadecalin compound is dazucorilant: (R)-(1-(4-fluorophenyl)-6-((4-(trifluoromethyl)phenyl) sulfonyl)-4,4a,5,6,7,8-hexahydro-1-H-pyrazolo3,4-gisoquinolin-4a-yl) (pyridin-2-yl)methanone, having the chemical structure illustrated asSuitable doses include daily administration of 150 milligrams and 300 milligrams of dazucorilant. Suitable doses include daily administration of dazucorilant with food, or with water, or with food and water. Daily administration of dazucorilant is effective to increase dazucorilant exposure up to about 2-fold when continued for seven days or more. Administration of such a heteroaryl ketone fused azadecalin compound may comprise oral administration, enteral administration, or other administration. Pharmaceutical compositions comprising dazucorilant are useful in the treatment of patients suffering from ALS. Suitable pharmaceutical compositions comprising dazucorilant include, e.g., pharmaceutical compositions for oral administration and pharmaceutical compositions for enteral administration.
Absstract of: US2025290075A1
Provided herein are RNAi molecules for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.
Absstract of: AU2024238598A1
The present invention provides a humanised antibody comprising an antigen-binding domain capable of binding specifically to Aβ42 prefibrillar oligomers with β structure, said antigen-binding domain comprising: (iii) a heavy chain variable region (VH) comprising the sequence of SEQ ID NO.1; or (iv) a light chain variable region (VL) comprising the sequence of SEQ ID NO.2; or a combination thereof. Also provided is the use of the antibody in the treatment of an amyloid disease, and particularly Alzheimer's disease, conjugates and pharmaceutical compositions comprising the antibody, and nucleic acid molecules encoding the antibody or a heavy or light chain polypeptide thereof, as well as vectors and host cells comprising such a molecule.
Absstract of: AU2025223847A1
Abstract Provided herewith is a pharmaceutical composition comprising, separately or together, a pulsatile release component comprising levodopa and a DOPA decarboxylase inhibitor for the management of OFF-time episodes in patients with Parkinson’s disease. 5 Abstract Provided herewith is a pharmaceutical composition comprising, separately or together, a pulsatile release component comprising levodopa and a DOPA decarboxylase inhibitor for the management of OFF-time episodes in patients with Parkinson's 5 disease. ug b s t r a c t r o v i d e d h e r e w i t h i s a p h a r m a c e u t i c a l c o m p o s i t i o n c o m p r i s i n g , s e p a r a t e l y o r t o g e t h e r , u g a p u l s a t i l e r e l e a s e c o m p o n e n t c o m p r i s i n g l e v o d o p a a n d a d e c a r b o x y l a s e i n h i b i t o r f o r t h e m a n a g e m e n t o f - t i m e e p i s o d e s i n p a t i e n t s w i t h a r k i n s o n ' s d i s e a s e
Absstract of: WO2025194036A1
The present disclosure is generally directed to tetracyclic analogs that modulate autophagy in a subject suffering from alpha-1 antitrypsin deficiency (ATD) and possibly other autophagy associated diseases or disorders, such as Alzheimer's disease, Parkinson's disease, Huntington's disease, and amyotrophic lateral sclerosis.
Absstract of: US2025289849A1
The present invention relates to a cell-penetrating peptide and its derivatives to inhibit α-synuclein fibrillation. The present invention specifically relates to a peptide-based inhibitor of Parkinson's Disease. The present invention discloses the identification of the peptides permeable to blood brain barrier for inhibition. α-synuclein fibril formation is observed in the presence of peptides as indicated by SEQ. ID-7, SEQ. ID-8 wherein the SEQ. ID-7, SEQ. ID-8 are truncated versions of SEQ. ID-4 having the homology of 83% and 75% respectively. It provides a peptide having an amino acid sequence of Formula 1. The present invention also provides a pharmaceutical composition comprising a peptide of Formula 1 along with the pharmaceutically acceptable excipient(s) having inhibitory activity against β-sheet polymerisation of amyloidogenic proteins. The analysis showed that peptides corresponding to SEQ. ID-7, SEQ. ID-8 are better inhibitors than SEQ. ID-4 against α-synuclein fibrillation.
Absstract of: US2025289858A1
BAG3 protein is used in the treatment of, for example, is Parkinson's disease, Alzheimer's disease, Amyotrophic Lateral Sclerosis, Huntington disease, Lewy body disease, vascular dementia, mixed dementia and Traumatic Brain Injury, for example, complicated by Chronic Traumatic Encephalopathy (CTE). Therapeutically effective BAG3 compositions, BAG3 uses and BAG3 methods of treatment are described.
Absstract of: US2025289803A1
The invention provides a compound of formula (I), or a pharmaceutically acceptable salt, ester or carbamate thereof, or a salt of such an ester or carbamate,wherein either:R1 is OH, and R2 is H; orR1 is H, and R2 is OH.The invention further provides uses of the compounds of formula (I) and compositions comprising compounds of formula (I), including the use of such compounds for the detection of tau deposits, and the use of such compounds and compositions as diagnostic agents in the diagnosis or monitoring of the progression of a disease or disorder such as Alzheimer's disease, progressive supranuclear palsy and corticobasal degeneration, or for the prevention or treatment of a disease or disorder such as Alzheimer's disease, progressive supranuclear palsy and corticobasal degeneration.
Absstract of: EP4617286A2
The present invention relates to antigen binding proteins, such as monoclonal antibodies, that specifically bind to and activate human triggering receptor expressed on myeloid cells-2 (TREM2) and pharmaceutical compositions comprising such antigen binding proteins. The agonist antigen binding proteins (e.g. antibodies) of the invention are capable of activating TREM2/DAP12 signaling in myeloid cells in the absence of Fe-mediated crosslinking of the antigen binding proteins. Methods of treating or preventing conditions associated with TREM2 loss of function, such as Alzheimer's disease and multiple sclerosis, using the antigen binding proteins are also described.
Nº publicación: EP4616852A2 17/09/2025
Applicant:
ANAVEX LIFE SCIENCES CORP [US]
Anavex Life Sciences Corp
Absstract of: EP4616852A2
Polymorphic forms of tetrahydro-N,N-dimethyl-2,2-diphenyl-3-furanmethanamine hydrochloride (ANAVEX2-73) and a metabolite of tetrahydro-N,N-dimethyl-2,2-diphenyl-3-furanmethanaminehydrochloride (ANAVEX2-73) are disclosed and characterized. Compositions and method for treatment of Alzheimer's disease that includes the polymorphic forms and metabolite of tetrahydro-N,N-dimethyl-2,2-diphenyl-3-furanmethanamine hydrochloride (ANAVEX2-73).