Alerta

Aav vector for use

Absstract of: GB2703823A

AAV vectors which encode for DNAJB1, particularly of a AAV9 serotype or variant thereof are claimed. The AAV vectors are used in a method of treatment of a disease, wherein the disease is ALS and/or FTD, a disease characterised by production of one or more dipeptide repeat proteins, or a disease characterised by TDP-43 mislocalization or TDP-43 proteinopathy. none

Novel compounds and uses thereof

Absstract of: GB2703800A

The invention relates to eight synthetic compounds related to naturally occurring flavonoids such as 7,8-dihydroxyflavone (7,8-DHF): The invention further relates to the synthesis of these piperidinyl chromeno7,8-dimidazol-6(3H)-one and piperidinyl chromeno7,8-dimidazol-6(1H)-one compounds, the use of these piperidinyl compounds as research tools, their use as pharmaceuticals, and to intermediates in their synthesis. These compounds may be useful in the treatment of conditions such as depression and neurodegenerative diseases such as Alzheimer’s disease, Parkinson’s disease and amyotrophic lateral sclerosis (ALS). See Table 1

LACTAMS FOR USE AS SARM1 INHIBITORS

Absstract of: WO2025076088A1

This invention relates to lactam compounds of Formula (I), as further detailed herein, which are used for inhibition of SARM1 proteins, as well as compositions comprising these compounds and methods of treatment by their administration.

METHODS AND COMPOUNDS FOR MODULATING HUNTINGTON'S DISEASE

Absstract of: TW202530208A

The present disclosure relates to transcription modulator molecules having a first terminus, a second terminus, and an oligomeric backbone and methods for treating Huntington's disease (HD).

METHODS AND COMPOUNDS FOR MODULATING HUNTINGTON'S DISEASE

Absstract of: WO2025076181A1

The present disclosure relates to transcription modulator molecules having a first terminus, a second terminus, and an oligomeric backbone and methods for treating Huntington's disease (HD).

METHODS AND COMPOUNDS FOR MODULATING HUNTINGTON'S DISEASE

Absstract of: WO2025076219A1

The present disclosure relates to transcription modulator molecules having a first terminus, a second terminus, and an oligomeric backbone and methods for treating Huntington's disease (HD).

GENE EXPRESSION-BASED TESTS FOR ALZHEIMER'S DISEASE

Absstract of: WO2025076156A1

This invention provides a method for determining whether a human subject is afflicted with Alzheimer's disease ("AD") or non-Alzheimer's dementia ("non-ADD") when the subject is suspected of being afflicted with AD or non-ADD, comprising the steps of (a) synchronizing a population of suitable cells derived from the subject, wherein the suitable cells are cultured skin cell fibroblasts or cultured B lymphocytes; and (b) in the resulting synchronized cell population, measuring the expression levels of two or more genes selected from the group consisting of FAM149B, NHLH1, SHISA5, URB2, and WASF2, whereby (i) the subject is afflicted with AD if the expression levels measured in step (b) are consistent with those genes' expression levels in corresponding synchronized cells derived from AD patients, and (ii) the subject is afflicted with non-ADD if the expression levels measured in step (b) are consistent with those genes' expression levels in corresponding synchronized cells derived from non-ADD patients. This invention also provides related diagnostic and therapeutic methods, including diagnostic methods based on NDS subject gene expression levels.

NEGATIVE ALLOSTERIC MODULATORS OF METABOTROPIC GLUTAMATE RECEPTOR 2

Absstract of: US20260226019A1

0000 Described are 6-aryl isoindolin-1-ones as negative allosteric modulators of metabotropic glutamate receptor 2 (mGlu<2>), pharmaceutical compositions including the compounds, and methods of using the compounds and compositions for treating depression, anxiety, obsessive-compulsive disorder, cognitive disorders, Alzheimer's disease, or autism spectrum disorders in a subject.

ANTIBODIES FOR TREATING NEUROLOGICAL AND NEUROVASCULAR DISORDERS

Absstract of: US20260226177A1

0000 The present disclosure provides humanized anti-GluN1 receptor antibodies and antigen-binding fragments or derivatives thereof, which are effective in inhibiting the deleterious effects of tissue-type plasminogen activator (t-PA) mediated by N-methyl-D-aspartate (NMDA) receptors, as well as pharmaceutical compositions and medical uses thereof, particularly for the treatment of neurological, neurovascular or neurodegenerative disorders, such as stroke, multiple sclerosis, Parkinson's disease, and others.

OLIGONUCLEOTIDES FOR MODULATING APOLIPOPROTEIN E (APOE) EXPRESSION AND METHODS OF USE THEREOF

Absstract of: US20260226463A1

0000 The present disclosure relates to oligonucleotides, in particular antisense oligonucleotides (ASOs) and pharmaceutically acceptable salts thereof, that can hybridize and reduce the expression of APOE pre-mRNA or mRNA. ASOs disclosed herein can reduce translation of APOE protein in mammals (e.g., humans). The present disclosure further relates to methods of treating a disease or disorder in a subject in need thereof by administration of an antisense oligonucleotide disclosed herein. In particular, methods and ASOs described herein can be used for preventing and/or treating human diseases in which the reduction of APOE amount or its activity would be beneficial, including but not limited to neurodegenerative diseases such as Alzheimer's disease (AD), dementia with Lewy bodies (DLB), Parkinson disease dementia (PDD), and those broadly defined as tauopathies and synucleinopathies.

FULLERENE GABA DOPA AND METHODS

Absstract of: US20260224713A1

0000 A novel dual neurotransmitter nanoparticle composition is provided to store and transport protons and cations into neural cell membranes and to disassemble salt-bridge stabilized toxic protein plaques. These properties function to mitigate cognitive deficits in neurological diseases such as Parkinson's disease and Alzheimer's disease, as well as to reduce the severity of Inflammatory Bowel Syndrome, and aging related reactive oxygen species damage by promoting the sequestration and termination of free radicals and reactive oxygen species. The composition comprises C60 bonded to one or more gamma amino butyric acid molecules and one or more molecules of either levodopa or dopamine. The composition can be produced at low temperatures through reactive shear milling. This composition therapeutically improves and prophylactically preserves cognitive performance, memory, and mental acuity on aging to promote mental performance and health-span improvement.

ARYL HETEROCYCLIC KV1.3 INHIBITOR, PREPARATION METHOD THEREFOR, AND USE THEREOF

Absstract of: US20260224536A1

A novel Kv1.3 channel (or KV1.3) inhibitor, which can be used for preventing and/or treating diseases related to the Kv1.3 channel (or Kv1.3), including immune and inflammatory diseases, such as: multiple sclerosis, inflammatory bowel disease, ulcerative colitis, Crohn's disease, rheumatoid arthritis, type I diabetes, psoriasis and asthma, spondylitis, and periodontitis; as well as obesity, type 2 diabetes, renal fibrosis, Alzheimer's disease, ischemic stroke, etc.

INHIBITORS OF CYCLIC ADP RIBOSE HYDROLASE AND METHODS OF USE THEREOF

Absstract of: US20260224561A1

0000 The disclosure provides compounds, in part, compounds of Formula I or Formula II and their use in treating medical diseases or disorders, such as neurodegenerative diseases, e.g., Parkinson's disease. Pharmaceutical compositions and methods of making compounds of the disclosure are provided. The compounds are contemplated to be modulators, e.g., inhibitors, of cyclic ADP ribose hydrolase (CD38).

ALS treatment using induced regulatory T (IT) cells

Absstract of: AU2026207969A1

ALS TREATMENT USING INDUCED REGULATORY T (iTREG) CELLS The present disclosure provides methods for treating ALS using pentostatin and cyclophosphamide treatment followed by TREG and/or TREG/Th2 hybrid cells from dedifferentiated T cells. The present disclosure further provides methods for producing TREG and TREG/Th2 hybrid cells from de-differentiated T cells, said TREG and TREG/Th2 hybrid cells, populations thereof and compositions thereof. Methods for producing de-differentiated T cells, said de-differentiated T cells, populations thereof and compositions thereof are also provided. ul u l

PHARMACEUTICAL FORMULATIONS OF GENE DELIVERY VEHICLES

Absstract of: US20260224740A1

The invention relates to formulations comprising miRNA that have improved stability for the treatment of diseases including neurodegenerative diseases such as Huntington's disease.

THIOUREA ANTIOXCOMPOUNDS WITH NEUROPROTECTIVE ACTIVITY

Absstract of: US20260226036A1

Provided herein are compounds of the formula wherein the variables are as defined herein. Pharmaceutical compositions of the compounds are also provided. In some aspects, the compounds or compositions of the present disclosure may be used for the treatment of diseases or disorders, such as an addiction or neurodegenerative disease including Alzheimer's disease or Parkinson's disease.

ALPHA-SYNUCLEIN BINDERS AND METHODS OF USE

Absstract of: US20260224758A1

The invention is directed to compounds of Formula I (I) or their pharmaceutically acceptable salts, which may be suitable for imaging alpha-synuclein pathology and hence are useful in binding and imaging alpha-synuclein aggregates in patients with Parkinson's Disease. More specifically, this invention relates to a method of using the compounds of this invention as tracers in positron emission tomography (PET) imaging to study alpha-synuclein in brain in vivo to allow diagnosis of Parkinson's Disease and other neurodegenerative diseases Specific characterized by alpha-synuclein pathology. The invention further relates to a method of measuring clinical efficacy of therapeutic agents for Parkinson's Disease and other neurodegenerative diseases characterized by alpha-synuclein pathology.

COMPOUND FOR RECOGNIZING α-SYNUCLEIN AGGREGATE, AND USE THEREOF

Absstract of: AU2025213908A1

A compound specifically binding to an α-synuclein aggregate, and a preparation method therefor and the use thereof. Specifically, the compound binding to the α-synuclein aggregate comprises compounds as shown in formula A or sub-general formulas thereof, or stereoisomers, pharmaceutically acceptable salts, solvates or stable isotope variants thereof. The compound is a small molecule tracer, which can specifically recognize the α-synuclein aggregate, and can be used for the preparation of a drug for the treatment or diagnosis of neurodegenerative diseases (such as Parkinson's disease, dementia with Lewy bodies, multiple system atrophy, Alzheimer's disease, amyotrophic lateral sclerosis, progressive supranuclear palsy and progressive muscular atrophy) related to the α-synuclein aggregate and other misfolded proteins.

PYRIDINE DERIVATIVES WITH N-LINKED CYCLIC SUBSTITUENTS AS CGAS INHIBITORS

Absstract of: EP4786463A2

0001 The invention relates to new proline derivatives of formula (I) as cGAS inhibitors, wherein wherein R1, R2, R3, R4, R5, R6, R7, R8, R9, R10, R11 and G are defined as in claim 1, and prodrugs or pharmaceutically acceptable salts of these compounds for the treatment of diseases such as systemic lupus erythematosus, systemic sclerosis (SSc), non-alcoholic steatohepatitis (NASH), interstitial lung disease (ILD) and idiopathic pulmonary fibrosis (IPF).

Fused heterocyclic compounds and uses thereof

Absstract of: GB2703562A

A phenoxazine, chromenothiazole, xanthene or thioxanthone compound of formula I or a pharmaceutically acceptable salt, hydrate or solvate thereof for use in a method of treatment or prophylaxis of a tauopathy or a disease of tau protein aggregation: wherein: R1 is -H, C1-4alkyl, halogenated C1-4alkyl, ORO1, C(=O)ORO2 or C(=O)NRN1RN2; R2 is H, halo, C1-4alkyl, NO2, OH or OC1-4alkyl; or R1 and R2, together with the atoms to which they are bound, form a fused phenylene group; R3 is H, halo, -ORO4, or -NRN3RN4; RN3 and RN4 are H, C1-4alkyl or -C(=O)R’, where R’ is C1-4alkyl; R4 is selected from H, halo, C1-4alkyl, halogenated C1-4alkyl, and -ORO5; RO1, RO2, RO4, RO5, RN1 and RN2 are H or C1-4alkyl; X is O; Y is N, N-oxide, or C-R5; and ring ‘A’ is a fused phenylene of formula A1 or a fused aminothiazole of formula A2; or X is S; Y is C=O; and ring A is a fused phenylene of formula A3: wherein W is O or N+ substituted with H or C1-4alkyl; and the remaining groups are as defined herein. The compounds may be useful in the treatment of Alzheimer’s disease. Compounds prepared include N-ethyl-N-2-(piperidin-1-yl)-6H-chromeno2,3-dthiazol-6-ylideneethanaminium perchlorate. See generic formula I at page 5

Method

Absstract of: GB2703561A

The present invention relates to a method of introducing a vector comprising a nucleotide sequence encoding a Kv7.3 ion channel subunit to a lower urinary tract (LUT) neuron and expressing or overexpressing the Kv7.3 ion channel subunit, such that it forms functional ion channels. The method can additionally include a step of administering a neuromodulatory drug. Also disclosed is a gene therapy vector, comprising a nucleotide sequence encoding a Kv7.3 ion channel subunit, wherein the vector selectively transduces the target LUT neuron. Further disclosed is an AAV viral particle gene therapy vector comprising an AAV capsid and a nucleotide sequence encoding a Kv7.3 ion channel subunit. The method can be used to treat a neurological disorder, selected from; spasticity, Parkinson's disease, multiple sclerosis, stroke, traumatic brain injury, spinal cord injury, motor neuron disease, spina bifida, transverse myelitis, HTLV-1 associated neurological conditions, and cerebral palsy. The neuromodulatory drug is selected from; Retigabine/Ezogabine and derivatives thereof, BHV-7000 and Xen496, Xen 1101, flupirtine, diclofenac, BMS-204352, meclofenamic acid, ETX-123 and linopirdine. A use of a neuromodulatory drug in the treatment of bladder musculature dysfunction (BMD), such as detrusor sphincter dyssynergia (DSD) or OAB-NC is also disclosed. Fig. 1

PIPERIDINE SUBSTITUTED PYRAZOLOPYRIMIDINE DERIVATIVES AS INHIBITORS OF SGK1

Absstract of: WO2025072438A1

The present invention provides compounds of Formula (I): or stereoisomers, tautomers, or pharmaceutically acceptable salts thereof, wherein all the variables are as defined herein. These compounds are selective SGK1 inhibitors. This invention also relates to pharmaceutical compositions comprising these compounds and methods of treating disorders associated with serum- and glucocorticoid-regulated kinase 1 (SGK1) activity, such as cardiovascular disorders, fibrotic diseases, metabolic diseases, immune and inflammatory diseases, neurological disorders, and cancer, by using the compounds and pharmaceutical compositions.

PRODUCTS AND METHODS FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS

Absstract of: US2021108209A1

0001 The present invention relates to RNA-based methods for inhibiting the expression of the superoxide dismutase 1 (SOD-1) gene. Recombinant adeno-associated viruses of the invention deliver DNAs encoding RNAs that knock down the expression of SOD-1. The methods have application in the treatment of amyotrophic lateral sclerosis.

COMPOUND AS GLUTAMINE CYCLASE INHIBITOR

Absstract of: EP4786464A1

Disclosed are a compound represented by formula (A-I) or a stereoisomer thereof or a pharmaceutically acceptable salt thereof as a glutamine cyclase inhibitor, a preparation method therefor, a pharmaceutical composition comprising the compound represented by formula (A-I) or the stereoisomer thereof or the pharmaceutically acceptable salt thereof, and a use of the compound represented by formula (A-I) or the stereoisomer thereof or the pharmaceutically acceptable salt thereof in preventing or treating diseases or conditions mediated by QPCT and/or QPCTL, including neurodegenerative diseases.

TRIAZOLOPYRIDINYL COMPOUNDS AS KINASE INHIBITORS

Nº publicación: EP4784245A1 05/08/2026

Applicant:

BRISTOL MYERS SQUIBB CO [US]
Bristol-Myers Squibb Company

Absstract of: AU2024353744A1

Compounds having formula (I), and enantiomers, and diastereomers, stereoisomers, pharmaceutically acceptable salts thereof, (I) are useful as kinase modulators, including RIPK1 modulation. All the variables are as defined herein.