Researchers at the University of London and collaborating institutions have developed a gene and cell therapy approach that enables sustained systemic frataxin protein delivery, improving motor performance and tissue pathology, and supporting a promising translational strategy for long-term disease stabilization in Friedreich’s ataxia patients.
A new strategy aims to improve gene therapy for Pompe disease by optimizing both the genetic component that restores the function of a deficient lysosomal enzyme and the vector that delivers it to the target tissue while avoiding the liver. The findings suggest that combining an optimized transgene with a targeted capsid could significantly enhance the effectiveness of gene therapy for Pompe disease.
Nxera Pharma UK Ltd. has identified new uracil nucleotide/cysteinyl leukotriene receptor (GPR17; P2Y-Like) antagonists potentially useful for the treatment of multiple sclerosis.
Researchers from the University of Copenhagen and collaborating institutions aimed to develop a therapy for chronic neuropathic pain based on gene therapy delivered with adeno-associated viral (AAV) vectors.
Increasing evidence supports metabotropic glutamate mGlu7 receptor as a promising target in psychiatric disorders. Neurosterix Pharma Sarl has presented data on NTX-819, a potential first-in-class, potent and selective negative allosteric modulator (NAM) of mGlu7. NTX-819 was tested in vivo in rats using behavioral tests that are relevant to disorders such as obsessive-compulsive disorder (OCD), agitation and mania/psychosis.
Camp4 Therapeutics Corp. has highlighted new preclinical data for CMP-002, the company’s lead investigational antisense oligonucleotide (ASO) therapeutic candidate for SYNGAP1-related disorder. CMP-002 administration resulted in a statistically significant improvement in seizure phenotypes and parameters in a SYNGAP1 haploinsufficient mouse model.
Andzonbio2 has signed agreements with the Alborada Drug Discovery Institute (ADDI) at the University of Cambridge and Cambridge Enterprise to advance a new class of therapeutics targeting neuroinflammation, a central driver of multiple neurodegenerative and neurological conditions.
Universitätsklinikum Hamburg-Eppendorf has synthesized new transient receptor potential cation channel subfamily M member 4 (TRPM4) antagonists potentially useful for the treatment of neurodegeneration, multiple sclerosis and inflammatory disorders.
RSS
