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IMIDAZOLONE DERIVATIVES AS INHIBITORS OF PROTEIN KINASES IN PARTICULAR DYRK1A, CLK1, AND/OR CLK4

Publication No.:  US2025340539A1 06/11/2025
Applicant: 
PERHA PHARMACEUTICALS [FR]
PERHA PHARMACEUTICALS
US_2025340539_PA

Absstract of: US2025340539A1

The present invention relates to a compound of formula (I) wherein R1 represents a (C3-C8)cycloalkyl group, a bridged (C6-C10)cycloalkyl group, a fused phenyl group, a substituted phenyl group, a R′-L- group, wherein L is either a single bond or a (C1-C3)alkanediyl group, and R′ represents, a (C3-C8)heterocycloalkyl group, or a (C3-C8)heteroaryl u group, or a R″-L- group wherein L is a (C1-C3)alkanediyl group, and R″ is an optionally substituted phenyl group; R2 is selected from the group consisting of a hydrogen atom and a (C1-C3)alkyl group; R5 represents a hydrogen atom, a (C1-C4)alkyl group or a (C3-C6)cycloalkyl group or any of its pharmaceutically acceptable salt. The present invention further relates to a composition comprising a compound of formula (I) and a process for manufacturing said compound as well as its synthesis intermediates. It also relates to said compound for use as a medicament, in particular in the treatment and/or prevention of cognitive deficits and neuroinflammation associated with Down syndrome; Alzheimer's disease; dementia; tauopathies; Parkinson's disease; CDKL5 Deficiency Disorder; Phelan-McDermid syndrome; autism; type 1 and type 2 diabetes; abnormal folate and methionine metabolism; osteoarthritis and tendinopathy; Duchenne muscular dystrophy; cancers and leukemias; neuroinflammation, anemia, infections caused by unicellular parasites, viral infections and for regulating body temperature.

IMIDAZOLONE DERIVATIVES AS INHIBITORS OF PROTEIN KINASES IN PARTICULAR DYRK1A, CLK1 AND/OR CLK4

Publication No.:  US2025340538A1 06/11/2025
Applicant: 
PERHA PHARMACEUTICALS [FR]
PERHA PHARMACEUTICALS
US_2025340538_PA

Absstract of: US2025340538A1

The present invention relates to a compound of formula (I) wherein A, B, C, D and E are selected from the group consisting of ═CH— and —N═, R2 is selected from a hydrogen atom, a (C1-C4)alkyl group and a (C3-C6)cycloalkyl group, R1 represents a (C4-C6)alkyl group, a (C3-C8)cycloalkyl group, a bridged (C6-C10)cycloalkyl group, a fused phenyl group, a substituted phenyl group, a R′-L- group, wherein L is either a single bond or a (C1-C3)alkanediyl group, and R′ represents, a (C3-C8)heterocycloalkyl group, or a (C3-C8)heteroaryl group, or a R′-L- group wherein L is a (C1-C3)alkanediyl group, and R′ is a an optionally substituted phenyl group or any of its pharmaceutically acceptable salt. The present invention further relates to a composition comprising a compound of formula (I) and a process for manufacturing said compound as well as its synthesis intermediates. It also relates to said compound for use as a medicament, in particular in the treatment and/or prevention of cognitive deficits and neuroinflammation associated with Down syndrome, Alzheimer's disease, dementia and/or tauopathies; Parkinson's disease; CDKL5 Deficiency Disorder; Phelan-McDermid syndrome; autism; type 1 and type 2 diabetes; abnormal folate and methionine metabolism; tendinopathy and osteoarthritis; Duchenne muscular dystrophy; several cancers; neuroinflammation, anemia, infections and for regulating body temperature.

4-AMINOPYRROLO2,1-F1,2,4TRIAZINES AND PREPARATION AND USES THEREOF

Publication No.:  US2025340575A1 06/11/2025
Applicant: 
BIOSPLICE THERAPEUTICS INC [US]
Biosplice Therapeutics, Inc
US_2025340575_A1

Absstract of: US2025340575A1

4-Aminopyrrolo2,1-f1,2,4triazine compounds for treating various diseases and pathologies are disclosed. More particularly, the present disclosure concerns the use of 4-aminopyrrolo2,1-f1,2,4triazine compounds or analogs thereof, in the treatment of disorders characterized by overexpression of DYRK1A (e.g., cancer, Down syndrome, Alzheimer's disease, diabetes, and osteoarthritis).

COMPOUNDS AND METHODS FOR REDUCING TAU EXPRESSION

Publication No.:  US2025340872A1 06/11/2025
Applicant: 
IONIS PHARMACEUTICALS INC [US]
Ionis Pharmaceuticals, Inc
US_2025340872_A1

Absstract of: US2025340872A1

Provided are RNAi agents, methods, and pharmaceutical compositions for reducing the amount or activity of tau RNA in a cell or animal, and in certain instances reducing the amount of tau protein in a cell or animal. Such RNAi agents, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease, including a tauopathy, Alzheimer's disease, fronto-temporal dementia (FTD), FTDP-17, progressive supranuclear palsy (PSP), chronic traumatic encephalopathy (CTE), corticobasal ganglionic degeneration (CBD), epilepsy, or Dravet's Syndrome.

OLIGONUCLEOTIDE TARGETING AMYLOID PRECURSOR PROTEIN GENE AND USE THEREOF

Publication No.:  WO2025228429A1 06/11/2025
Applicant: 
ANLONG BIOPHARMACEUTICAL CO LTD [CN]
\u5317\u4EAC\u5B89\u9F99\u751F\u7269\u533B\u836F\u6709\u9650\u516C\u53F8
WO_2025228429_A1

Absstract of: WO2025228429A1

An RNAi agent targeting an amyloid precursor protein, such as a double-stranded small interfering RNA (siRNA) agent. A method for inhibiting the expression of the APP gene by using the RNAi agent and a method for preventing and treating APP-related diseases, such as cerebral amyloid angiopathy (CAA) or Alzheimer's disease (AD), including early-onset familial Alzheimer's disease (EOFAD). The siRNA significantly inhibits the expression level of the APP gene and has a long-lasting drug effect.

METHODS FOR PREVENTING, TREATING, AND/OR DIAGNOSING NEURODEGENERATIVE DISEASES

Publication No.:  WO2025229644A1 06/11/2025
Applicant: 
LEUMIT LAATID LTD [IL]
LEUMIT LAATID LTD
WO_2025229644_PA

Absstract of: WO2025229644A1

The present invention relates to pharmaceutical compositions and diagnostic methods for neurodegenerative diseases, particularly Parkinson's disease. The invention includes compositions comprising anti-Clostridium tetani agents such as tetanus vaccines, anti-tetanus immunoglobulins, and antibiotics. Methods for diagnosis and monitoring Parkinson's disease comprising detecting C. tetani-derived nucleic acids, proteins, or analogues thereof, in biological specimens, are further provided.

PHOSPHO-TAU AGGREGATION-BASED BIOMARKERS FOR ALZHEIMER'S DISEASE DIAGNOSIS, DIFFERENTIATION, AND TREATMENT

Publication No.:  WO2025231348A1 06/11/2025
Applicant: 
NORTH CAROLINA CENTRAL UNIV [US]
DUKE UNIV [US]
NORTH CAROLINA CENTRAL UNIVERSITY,
DUKE UNIVERSITY
WO_2025231348_PA

Absstract of: WO2025231348A1

Provided are methods of phospho-tau aggregation-based biomarker discovery, and new utilities for discovered biomarkers in Alzheimer's disease (AD) diagnosis, differentiation, treatment, and identification of the presence of pretangles in a subject. Novel p-tau sites, p-tau198, p-tauS356, p-tau396, and p-tau422, identified through such methods showed comparable or superior characteristics with established p-tau biomarkers, and identified biomarkers were capable of differentiating AD or mild cognitive impairment (MCI) from cognitively normal controls.

4-AMINOPYRROLO2,L-FL,2,4TRIAZINES AND PREPARATION AND USES THEREOF

Publication No.:  WO2025231425A1 06/11/2025
Applicant: 
BIOSPLICE THERAPEUTICS INC [SA]
BIOSPLICE THERAPEUTICS, INC
WO_2025231425_PA

Absstract of: WO2025231425A1

4-Aminopyrrolo2,l-fl,2,4triazine compounds for treating various diseases and pathologies are disclosed. More particularly, the present disclosure concerns the use of 4-aminopyrrolo2,l-fl,2,4triazine compounds or analogs thereof, in the treatment of disorders characterized by overexpression of DYRK1A (e.g., cancer, Down syndrome, Alzheimer's disease, diabetes, and osteoarthritis).

CRYSTALLINE FORM OF A PYRIDAZINE NLRP3 INHIBITOR

Publication No.:  WO2025229146A1 06/11/2025
Applicant: 
SANOFI [FR]
SANOFI
WO_2025229146_A1

Absstract of: WO2025229146A1

The present disclosure relates to a compound of formula (I) which is in crystalline Form 1, characterized by having a powder X-ray diffractogram displaying peaks expressed as degree 2-Theta angles at about 3.4, 6.8, 10.3, 13.7, and 20.5. 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 crystalline Form 1 of compound of formula (I) for use as a medicine and more particularly in the prevention and/or in the treatment of Parkinson's disease, frontotemporal dementia, multiple system atrophy, Alzheimer's disease, multiple sclerosis, amyotrophic lateral sclerosis, or brain injury.

PHARMACEUTICAL COMBINATION OF DONEPEZIL AND CEVIMELINE

Publication No.:  WO2025229537A1 06/11/2025
Applicant: 
LUKA ASSETS INC [US]
LUKA ASSETS INC
WO_2025229537_A1

Absstract of: WO2025229537A1

The present invention relates to combination of donepezil or a pharmaceutically acceptable salt thereof and cevimeline or a pharmaceutically acceptable salt thereof for use in the treatment of Alzheimer ́s disease. Furthermore, a pharmaceutical formulation containing donepezil or a pharmaceutically acceptable salt thereof, cevimeline or a pharmaceutically acceptable salt thereof, and at least one pharmaceutically acceptable excipient is provided.

USE OF SUBSTITUTED 1,4 BENZOQUINONES TO TREAT ALPHA-SYNUCLEINOPATHIES

Publication No.:  WO2025231245A1 06/11/2025
Applicant: 
STEALTH BIOTHERAPEUTICS INC [US]
STEALTH BIOTHERAPEUTICS INC
WO_2025231245_A1

Absstract of: WO2025231245A1

The present disclosure provides methods, compounds, compositions, formulations, or medicaments for treating, preventing, inhibiting, ameliorating, or delaying the onset of a- synucleinopathies (e.g., Parkinson's disease (PD), PD with dementia (PDD), dementia with Lewy bodies (LBD), or Multiple System Atrophy (MSA)) as well as methods for ameliorating, inhibiting, or delaying the onset of signs or symptoms of an a-synucleinopathy in a subject. The disclosed methods, compounds, compositions, formulations, or medicaments are also useful for addressing the related signs and symptoms of a- synucleinopathies. The methods comprise administering to the subject the compounds, mixtures of compounds, or compositions, formulations, or medicaments derived from said compounds or mixtures thereof to thereby produce the aforementioned therapeutically beneficial effect(s).

COMPOSITIONS AND METHODS FOR TREATING PARKINSON'S DISEASE

Publication No.:  EP4642905A1 05/11/2025
Applicant: 
MOTIGENIX SINGAPORE PTE LTD [SG]
RELIABLE HOLDINGS CO LTD [SC]
Motigenix Singapore Pte. Ltd,
Reliable Holdings Co., Ltd
KR_20250121452_PA

Absstract of: CN120435551A

Provided herein are compounds, compositions, uses, and methods for increasing cell viability of dopaminergic neurons, or for preventing or treating dopaminergic neuronal death. In certain examples, methods are provided for reducing symptoms and/or for preventing or treating Parkinson's disease in an individual in need thereof, which can include the step of treatment with a GDP-binding form of Rab1a (Rab1aGDP), one or more manifestation nucleic acids encoding Rab1aGDP, or a combination thereof.

PHARMACEUTICAL COMBINATION OF DONEPEZIL AND CEVIMELINE

Publication No.:  EP4643857A1 05/11/2025
Applicant: 
LUKA ASSETS INC [US]
Luka Assets Inc
EP_4643857_A1

Absstract of: EP4643857A1

The present invention relates to combination of donepezil or a pharmaceutically acceptable salt thereof and cevimeline or a pharmaceutically acceptable salt thereof for use in the treatment of Alzheimer's disease. Furthermore, a pharmaceutical formulation containing donepezil or a pharmaceutically acceptable salt thereof, cevimeline or a pharmaceutically acceptable salt thereof, and at least one pharmaceutically acceptable excipient is provided.

PRIDOPIDINE FOR TREATING JUVENILE HUNTINGTON'S DISEASE

Publication No.:  EP4642457A1 05/11/2025
Applicant: 
PRILENIA NEUROTHERAPEUTICS LTD [IL]
Prilenia Neurotherapeutics Ltd
CN_120569199_A

Absstract of: CN120569199A

Provided herein is a method of treating teenager Huntington's disease in a subject in need thereof, the method comprising orally administering a pharmaceutical composition comprising pridopidine and/or an analog thereof or a pharmaceutically acceptable salt thereof.

COMBINATION TREATMENT OF SCYLLO-INOSITOL AND VITAMIN D AND/OR OTHER VITAMINS OR ACTIVE INGREDIENTS TO TREAT COGNITIVE DISORDERS

Publication No.:  MX2025002654A 03/11/2025
Applicant: 
EIRGEN PHARMA LTD [IE]
EIRGEN PHARMA, LTD
CN_120826223_PA

Absstract of: MX2025002654A

The disclosure relates to a combination of active ingredients/adjuvants for the treatment of neurological disorders and diseases such as Alzheimer's disease and mild cognitive impairment (MCI) and memory and cognitive disorders and conditions. In particular, combinations of scyllo-inositol and treatments for Alzheimer's disease such as aducanumab and/or combinations with essential fatty acids such as mixtures of linolenic acid/linoleic acid or vitamin D or vitamin D compounds such as calcifediol are disclosed as useful. The combinations may be in the form of separate dosage forms of each active ingredient or may be an oral dosage form having multiple active ingredients in a single capsule or tablet or oral solution. The invention also relates to a method of treating patients having mild cognitive impairment with MMSE criteria of between 22 to 26 with a pharmaceutically effective amount of scyllo-inositol to treat the disease and to slow down progression to Alzheimer's disease.

MIVELSIRAN COMPOSITIONS AND METHODS OF USE THEREOF

Publication No.:  MX2025012729A 03/11/2025
Applicant: 
ALNYLAM PHARMACEUTICALS INC [US]
ALNYLAM PHARMACEUTICALS, INC
AU_2024260719_A1

Absstract of: MX2025012729A

The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting the APP gene, as well as methods of inhibiting expression of an APP gene and methods of treating subjects having an APP-associated disease or disorder, such as Alzheimer's disease (e.g., early onset Alzheimer's disease), using such dsRNAi agents and compositions.

GENE THERAPY VECTORS FOR USE IN PARKINSON'S DISEASE

Publication No.:  MX2025009097A 03/11/2025
Applicant: 
JANSSEN PHARMACEUTICA NV [BE]
JANSSEN PHARMACEUTICA NV
KR_20250142386_PA

Absstract of: MX2025009097A

Disclosed herein are recombinant gene therapy vectors comprising a PTEN-induced kinase 1 (PINK1) encoding gene that is operatively linked to a promoter and methods of using the recombinant therapy vectors for inhibiting, reducing, or delaying degeneration or death of neurons of a subject.

ANTI-CD2 ANTIBODIES FOR AMYOTROPHIC LATERAL SCLEROSIS

Publication No.:  MX2025009971A 03/11/2025
Applicant: 
ZELARION MALTA LTD [MT]
ZELARION MALTA LIMITED
WO_2024180185_PA

Absstract of: MX2025009971A

Provided herein is an anti-CD2 antibody or antigen binding fragment thereof for treating and/or preventing ALS in a subject in need thereof.

METHODS AND COMPOSITIONS FOR REDUCING SYMPTOMS OF PARKINSON'S DISEASE

Publication No.:  MX2025012236A 03/11/2025
Applicant: 
NEURODERM LTD [IL]
NEURODERM, LTD
TW_202448421_A

Absstract of: MX2025012236A

Disclosed is a method for the treatment of a neurological or movement disorder, e.g., Parkinson's disease, in a patient in need thereof, by parenteral administration of levodopa and a dopa decarboxylase inhibitor (DDCI), such as carbidopa, benserazide or any combination thereof.

Methods and compositions for reducing symptoms of parkinson's disease

Publication No.:  IL323682A 01/11/2025
Applicant: 
NEURODERM LTD [IL]
ADAR LIAT [IL]
LOPES NELSON FELIX [IL]
SALIN LAURENCE [IL]
YARDENI TAMAR [IL]
SASSON NISSIM [IL]
OREN SHEILA [IL]
YARITA HIKARI [JP]
HIMIZU MIKIO [JP]
VOSTOKOVA NATALIA [IL]
NEURODERM LTD,
ADAR Liat,
LOPES Nelson Felix,
SALIN Laurence,
YARDENI Tamar,
SASSON Nissim,
OREN Sheila,
YARITA Hikari,
HIMIZU Mikio,
VOSTOKOVA Natalia
IL_323682_A

Absstract of: MX2025012236A

Disclosed is a method for the treatment of a neurological or movement disorder, e.g., Parkinson's disease, in a patient in need thereof, by parenteral administration of levodopa and a dopa decarboxylase inhibitor (DDCI), such as carbidopa, benserazide or any combination thereof.

Methods of treatment and prevention of alzheimer's disease

Publication No.:  IL323583A 01/11/2025
Applicant: 
EISAI R& D MAN CO LTD [JP]
EISAI R& D MANAGEMENT CO. LTD
IL_323583_A

Absstract of: CN118924896A

The invention relates to a method for treating and preventing Alzheimer's disease. The present invention provides methods of reducing clinical decline in a subject suffering from early Alzheimer's disease; a method of converting an amyloid-positive subject suffering from early Alzheimer's disease to an amyloid-negative subject; methods of reducing brain amyloid levels in a subject; and methods of preventing Alzheimer's disease comprising administering a composition comprising a therapeutically effective amount of at least one anti-A beta profibril antibody. In some embodiments, the subject is ApoE4 positive. In some embodiments, the at least one anti-A beta protofibril antibody is BAN2401.

Phosphatidylserine taurine direct drinking powder for improving memory and Alzheimer's disease and preparation method thereof

Publication No.:  CN120860039A 31/10/2025
Applicant: 
WANG MING
\u738B\u660E
CN_120860039_PA

Absstract of: CN120860039A

The invention provides phosphatidylserine and taurine direct drinking powder for improving memory and Alzheimer's disease and a preparation method, and relates to the field of direct drinking powder. The phosphatidylserine taurine direct drinking powder for improving memory and Alzheimer's disease is prepared from the following raw materials in percentage by mass: 30-40% of erythritol, 10-20% of taurine, 10-20% of maltodextrin, 10-20% of 28%-38% of resistant dextrin; 12% to 15% of phosphatidylserine; 5%-10% of theanine in tea leaves; 2%-8% of rhizoma polygonati powder; 2%-6% of menthol; 0%-1% of citric acid; 0%-1% of taurine; 10%-1% of vitamin B; and 20%-1% of vitamin B. Core active components are phosphatidylserine and taurine, a compound of citric acid and menthol can promote absorption of phosphatidylserine and taurine, resistant dextrin and polygonatum polysaccharide form an eutectic network as a carrier, phosphatidylserine and taurine are subjected to spray drying under the condition that the pH value is 4.5 to form an ionic bonding body, and the antibacterial activity of the product is improved. The direct drinking powder can rapidly dissolve out and release active ingredients through the acid-base property of oral mucosa, and the dosage form is rapidly released through the oral mucosa, so that the bioavailability is greatly improved.

Composition for preventing and improving Alzheimer's disease and preparation method thereof

Publication No.:  CN120860174A 31/10/2025
Applicant: 
YIWOSEN BEIJING BIOTECHNOLOGY CO LTD
\u4EBF\u6C83\u68EE\uFF08\u5317\u4EAC\uFF09\u751F\u7269\u79D1\u6280\u6709\u9650\u516C\u53F8
CN_120860174_PA

Absstract of: CN120860174A

The invention belongs to the technical field of biological pharmacy, and particularly relates to a composition for preventing and improving Alzheimer's disease and a preparation method thereof. The composition is prepared by the following method: (S1) adding phosphatidylserine, an emulsifier and walnut peptide into walnut sauce to obtain an oil phase; (S2) dissolving xylitol, whole milk powder and taurine in water, and slowly adding the oil phase in the homogenizing process to obtain a mixed solution; and (S3) dissolving a stabilizer in water to obtain a stabilizer solution, adding the stabilizer solution into the mixed solution, adding water to a constant volume, uniformly mixing, and homogenizing to obtain the composition for preventing and improving the Alzheimer's disease. The precipitation phenomenon caused by the Maillard reaction of walnut peptides is avoided, the antioxidant components in the walnut sauce can prevent phosphatidylserine from being oxidized and degraded, the quality problems of oxidative rancidity, fat floating and layering and the like of the walnut sauce are solved, the stability of the composition is improved, the shelf life of the product is prolonged, and the competitiveness of the product is improved.

Probiotic preparation for preventing and treating Alzheimer's disease and preparation method thereof

Publication No.:  CN120860076A 31/10/2025
Applicant: 
THE FIRST AFFILIATED HOSPITAL OF BENGBU MEDICAL COLLEGE AFFILIATED TUMOR HOSPITAL OF BENGBU MEDICAL
\u868C\u57E0\u533B\u5B66\u9662\u7B2C\u4E00\u9644\u5C5E\u533B\u9662\uFF08\u868C\u57E0\u533B\u5B66\u9662\u9644\u5C5E\u80BF\u7624\u533B\u9662\uFF09
CN_120860076_PA

Absstract of: CN120860076A

The invention belongs to the technical field of biological medicines, and particularly relates to a probiotic preparation for preventing and treating Alzheimer's disease and a preparation method thereof. The probiotic powder comprises the following raw materials in parts by weight: 5 to 6 parts of probiotic freeze-dried powder, 5 to 7 parts of whey protein powder, 8 to 10 parts of pullulan, 2.5 to 3.5 parts of quaternary ammonium salt modified chitosan, 35 to 40 parts of trehalose, 10 to 12 parts of maltodextrin, 10 to 12 parts of prebiotics, 3 to 3.5 parts of glycine, 1.3 to 1.8 parts of glycerol, 4.5 to 6 parts of sodium alginate, 1.2 to 1.8 parts of L-glutamine, 3 to 3.5 parts of fructo-oligosaccharide and 1 to 1.2 parts of lecithin. The probiotic preparation provided by the invention contains lactobacillus rhamnosus, bifidobacterium longum and lactobacillus plantarum, and the inner layer is a vitrification freeze-drying protection matrix of trehalose/protein, so that freeze-drying stability and rehydration activity are ensured; the outer layer is a sodium alginate/quaternized chitosan electrostatic self-assembly delivery system, so that gastric acid protection and small intestine targeted release are realized.

Recombinant AAV vectors for treatment of neurodegenerative disorders

Nº publicación: CN120866361A 31/10/2025

Applicant:

SHANGHAI TIANZE YUNTAI BIOMEDICAL CO LTD
\u4E0A\u6D77\u5929\u6CFD\u4E91\u6CF0\u751F\u7269\u533B\u836F\u6709\u9650\u516C\u53F8

CN_120866361_A

Absstract of: CN120866361A

There is provided a recombinant adeno-associated virus (rAAV) vector comprising one or both of (a) to (c): (a) a nucleotide sequence encoding an aromatic L-amino acid decarboxylase (AADC); (b) a nucleotide sequence for encoding glucocerebrosidase (GBA1); and (c) a nucleotide sequence encoding a neurotrophic factor (NTF), such as brain dopamine neurotrophic factor (CDNF) or glial cell-derived neurotrophic factor (GDNF), for use in the treatment of neurodegenerative disorders, in particular Parkinson's disease (PD), Multi-System Atrophy (MSA), Gaucher's disease (GD) and other proteinopathies. Also provided herein are viral particles comprising the rAAV vector, pharmaceutical compositions comprising the viral particles, and uses thereof.

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