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TREATMENTS FOR AMYOTROPHIC LATERAL SCLEROSIS USING DAZUCORILANT

Publication No.:  US2025288580A1 18/09/2025
Applicant: 
CORCEPT THERAPEUTICS INC [US]
Corcept Therapeutics Incorporated
US_2025288580_PA

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.

BAG3 AND PROTEIN QUALITY CONTROL IN THE BRAIN

Publication No.:  US2025289858A1 18/09/2025
Applicant: 
UNIV TEMPLE [US]
TEMPLE UNIVERSITY OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION
US_2025289858_A1

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.

VARIANT RNAi

Publication No.:  US2025290075A1 18/09/2025
Applicant: 
GENZYME CORP [US]
Genzyme Corporation
US_2025290075_PA

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.

PATHOLOGY-RESPONSIVE RECOMBINANT CELLS AND USES THEREOF

Publication No.:  US2025290037A1 18/09/2025
Applicant: 
UNIV CALIFORNIA [US]
THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
US_2025290037_PA

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.

SELECTIVE LIGANDS FOR TAU AGGREGATES

Publication No.:  US2025289803A1 18/09/2025
Applicant: 
SENTONIX INC [US]
Sentonix, Inc
US_2025289803_PA

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.

PEPTIDE TO TREAT ALPHA-SYNUCLEIN AMYLOID BASED DISORDERS

Publication No.:  US2025289849A1 18/09/2025
Applicant: 
COUNCIL SCIENT IND RES [IN]
COUNCIL OF SCIENTIFIC & INDUSTRIAL RESEARCH
US_2025289849_PA

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.

TREATMENT OF PARKINSON'S DISEASE IN A PATIENT USING A GLUCOCEREBROSIDASE ACTIVATOR

Publication No.:  AU2024237252A1 18/09/2025
Applicant: 
BIAL - R&D INVESTMENTS S A
BIAL - R&D INVESTMENTS, S.A
AU_2024237252_A1

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.

HUMANISED ANTIBODY AGAINST AMYLOID BETA 42

Publication No.:  AU2024238598A1 18/09/2025
Applicant: 
ALZINOVA AB
ALZINOVA AB
AU_2024238598_A1

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.

Pulsatile drug delivery system for treating morning akinesia

Publication No.:  AU2025223847A1 18/09/2025
Applicant: 
CONTERA PHARMA AS
Contera Pharma A/S
AU_2025223847_A1

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

AUTOPHAGY ENHANCERS

Publication No.:  WO2025194036A1 18/09/2025
Applicant: 
WASHINGTON UNIVERSITY ST LOUIS [US]
WASHINGTON UNIVERSITY
WO_2025194036_PA

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.

REAGENT, PHARMACEUTICAL COMPOSITION AND METHODS FOR DETECTING, EVALUATING THE PROGRESSION, AND TREATING DISEASES ASSOCIATED WITH B-AMYLOID PLAQUES

Publication No.:  US2025288701A1 18/09/2025
Applicant: 
UNIV CHILE [CL]
UNIV PONTIFICIA CATOLICA CHILE [CL]
UNIVERSIDAD DE CHILE,
PONTIFICIA UNIVERSIDAD CAT\u00D3LICA DE CHILE
US_2025288701_PA

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.

INFLAMMATORY CYTOKINE PRODUCTION INHIBITOR

Publication No.:  US2025288615A1 18/09/2025
Applicant: 
SAISEI PHARMA CO LTD [JP]
SAISEI PHARMA CO., LTD
US_2025288615_A1

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.

DELIVERY OF TAU REPRESSORS USING THE BLOOD-BRAIN BARRIER PENETRANT CAPSID

Publication No.:  WO2025193848A1 18/09/2025
Applicant: 
SANGAMO THERAPEUTICS INC [US]
SANGAMO THERAPEUTICS, INC
WO_2025193848_PA

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.

A TRISOMY LINKED HEMATOPOIETIC GENE VARIANT FOR TREATING ALZHEIMER'S DISEASE

Publication No.:  WO2025193743A1 18/09/2025
Applicant: 
UNIV RUTGERS [US]
RUTGERS, THE STATE UNIVERSITY OF NEW JERSEY
WO_2025193743_PA

Absstract of: WO2025193743A1

Provided herein are systems and methods for introducing an A455D mutation into CSF2RB to neurodegenerative diseases, such as Alzheimer's disease.

PHARMACEUTICAL COMPOSITION FOR PREVENTING OR TREATING ALZHEIMER'S DISEASE, COMPRISING NEURAL CREST-DERIVED NASAL TURBINATE STEM CELLS EXPRESSING SSEA3 AND CD105 AS ACTIVE INGREDIENT

Publication No.:  WO2025192800A1 18/09/2025
Applicant: 
CATHOLIC UNIV KOREA IND ACADEMIC COOPERATION FOUNDATION [KR]
\uAC00\uD1A8\uB9AD\uB300\uD559\uAD50 \uC0B0\uD559\uD611\uB825\uB2E8
WO_2025192800_A1

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.

TREM2 ANTIGEN BINDING PROTEINS AND USES THEREOF

Publication No.:  EP4617286A2 17/09/2025
Applicant: 
AMGEN INC [US]
Amgen Inc
EP_4617286_PA

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.

CRYSTAL FORMS OF TETRAHYDRO-N,N-DIMETHYL-2,2-DIPHENYL-3-FURANMETHANAMINE HYDROCHLORIDE, PROCESSES OF MAKING SUCH FORMS, AND THEIR PHARMACEUTICAL COMPOSITIONS

Publication No.:  EP4616852A2 17/09/2025
Applicant: 
ANAVEX LIFE SCIENCES CORP [US]
Anavex Life Sciences Corp
EP_4616852_PA

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).

PEPTIDE FOR TREATMENT OF COGNITIVE DISEASES

Publication No.:  WO2025186220A1 12/09/2025
Applicant: 
PRIAVOID GMBH [DE]
PRIAVOID GMBH
WO_2025186220_PA

Absstract of: WO2025186220A1

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.

VECTORIZED ANTI-TDP-43 ANTIBODIES

Publication No.:  WO2025186332A1 12/09/2025
Applicant: 
AC IMMUNE SA [CH]
AC IMMUNE SA
WO_2025186332_A1

Absstract of: WO2025186332A1

The present invention is in the field of AAV vectorized TDP-43 specific antibodies and uses thereof. The present invention further relates to means and methods to prevent, alleviate and/or treat a disease, disorder and/or abnormality associated with TDP-43, or TDP-43 proteinopathy, including but not limited to Frontotemporal dementia (FTD), amyotrophic lateral sclerosis (ALS), Alzheimer's disease (AD), Parkinson's disease (PD), Chronic Traumatic Encephalopathy (CTE), and limbic-predominant age-related TDP-43 encephalopathy (LATE).

METHODS OF TREATING PARKINSON'S DISEASE WITH T-TYPE CALCIUM CHANNEL MODULATORS

Publication No.:  WO2025188619A1 12/09/2025
Applicant: 
PRAXIS PREC MEDICINES INC [US]
PRAXIS PRECISION MEDICINES, INC
WO_2025188619_PA

Absstract of: WO2025188619A1

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.

METHODS OF USE OF (4R,5R)-5-(2-CHLOROPHENYL)-4-(5-(PHENYLETHYNYL)PYRIDIN-3-YL)OXAZOLIDIN-2-ONE

Publication No.:  WO2025188734A1 12/09/2025
Applicant: 
ALLYX THERAPEUTICS INC [US]
ALLYX THERAPEUTICS, INC
WO_2025188734_PA

Absstract of: WO2025188734A1

The present disclosure provides methods of treating Alzheimer's disease and other disorders using (4R,5R)-5-(2-chlorophenyl)-4-(5-(phenylethynyl)pyridin-3-yl)oxazolidin-2-one (Compound 1).

FUSED AMINO PYRIMIDINE COMPOUNDS AND PRIDOPIDINE FOR TREATMENT OF HUNTINGTON'S DISEASE

Publication No.:  WO2025188979A1 12/09/2025
Applicant: 
OVID THERAPEUTICS INC [US]
OVID THERAPEUTICS INC
WO_2025188979_PA

Absstract of: WO2025188979A1

Compositions and methods for treating Huntington's disease with a compound of Formula (I), or a pharmaceutically acceptable salt thereof, in combination with pridopidine, or a pharmaceutical salt thereof, are provided. The compositions and methods may be used to improve one or more symptoms of Huntington's disease. Formula (I): (I) or pharmaceutically acceptable salts thereof, wherein R1, R2, R7 and ring A have any of the meanings herein defined in the description.

ANTI-GALECTIN 3 ANTIBODIES AND THEIR USE IN EPILEPSY AND RELATED DISEASES

Publication No.:  WO2025185724A1 12/09/2025
Applicant: 
SUNMED THERAPEUTIC LTD [CN]
SUNMED THERAPEUTIC LIMITED
WO_2025185724_PA

Absstract of: WO2025185724A1

Provided herein are antibodies that target Galectin-3. Such antibodies are used in methods of treating epilepsy and related neurological disorders, such as Alzheimer's disease (AD) and Parkinson's disease (PD).

2,3,5-TRIMETHYL-6-NONYLCYCLOHEXA-2,5-DIENE-1,4-DIONE FOR SUPPRESSING AND TREATING a-SYNUCLEINOPATHIES, TAUOPATHIES, AND OTHER DISORDERS

Publication No.:  MY210324A 11/09/2025
Applicant: 
PTC THERAPEUTICS INC [US]
PTC THERAPEUTICS, INC
CN_119185268_PA

Absstract of: MY210324A

Disclosed herein are methods of treating or suppressing a disorder selected from the group consisting of a-synucleinopathies, tauopathies, ALS, traumatic brain injury, and ischemic reperfusion related injuries.ury, comprising administering to a subject in need thereof a therapeutically effective amount of a compound of the formula: or the hydroquinone form thereof; or a solvate or hydrate thereof. Fig. 1A

FUSED AMINO PYRIMIDINE COMPOUNDS AND PRIDOPIDINE FOR TREATMENT OF HUNTINGTON’S DISEASE

Nº publicación: US2025281500A1 11/09/2025

Applicant:

OVID THERAPEUTICS INC [US]
Ovid Therapeutics Inc

Absstract of: US2025281500A1

Compositions and methods for treating Huntington's disease with a compound of Formula (I), or a pharmaceutically acceptable salt thereof, in combination with pridopidine, or a pharmaceutical salt thereof, are provided. The compositions and methods may be used to improve one or more symptoms of Huntington's disease.Formula (I):or pharmaceutically acceptable salts thereof, wherein R1, R2, R7 and ring A have any of the meanings herein defined in the description.

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