Alerta

Method

Resumen de: 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

PRODUCTS AND METHODS FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS

Resumen de: 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

Resumen de: 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.

PYRIDINE DERIVATIVES WITH N-LINKED CYCLIC SUBSTITUENTS AS CGAS INHIBITORS

Resumen de: 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

Resumen de: 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

TRIAZOLOPYRIDINYL ETHER LINKED COMPOUNDS AS KINASE INHIBITORS

Resumen de: TW202521108A

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

PIPERIDINE SUBSTITUTED PYRAZOLOPYRIMIDINE DERIVATIVES AS INHIBITORS OF SGK1

Resumen de: 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.

TRIAZOLOPYRIDINYL COMPOUNDS AS KINASE INHIBITORS

Nº publicación: EP4784245A1 05/08/2026

Solicitante:

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

Resumen de: 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.