Another day, another about-face by the U.S. FDA on Uniqure NV’s Huntington’s disease gene therapy. But this latest shift brings good news for the company’s AMT-130, for which the FDA says three-year analysis data from the phase I/II study will be acceptable for an accelerated BLA filing, now expected to be submitted in the third quarter.

Opus Genetics Inc. is advancing a pipeline of gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases, with three programs expected to enter clinical testing over the next 12-18 months.

Autism spectrum disorder (ASD), developmental epileptic encephalopathies and other neurodevelopmental disorders are driven by the disruption of genes regulating neuronal proliferation, differentiation and synaptic maturation. Researchers from Shanghai Jiao Tong University School of Medicine generated Csnk2b haploinsufficient (Csnk2b+/-) mice mimicking the most relevant disease features to investigate the effects of reduced gene dosage.

Scientists at Columbia University have used base editing to make precise changes in the genomes of human embryos, avoiding the damage to chromosomes that occurs following two-stranded DNA cuts with conventional CRISPR/Cas9 editing.

Sensorion SA has selected SENS-601 (GJB2-GT) as its lead program and has filed clinical trial applications to study its use for GJB2-related hearing loss. SENS-601 is an AAV-based gene therapy program, utilizing a gene therapy platform codeveloped with Institut Pasteur.

Scientists at Columbia University have used base editing to make precise changes in the genomes of human embryos, avoiding the damage to chromosomes that occurs following two-stranded DNA cuts with conventional Crispr-Cas9 editing.

Idefine, The Kleefstra Syndrome Foundation, has established a collaboration with UT Southwestern Medical Center to advance development of a potential gene therapy to treat Kleefstra syndrome, a rare neurodevelopmental disorder caused by changes or loss of the EHMT1 gene, which plays a critical role in brain development and function.

Rather than reinventing the wheel for every gene therapy that uses genome editing, the U.S. FDA is advising sponsors on leveraging existing knowledge, be it publicly available or platform-based, to more efficiently advance their products across multiple stages of development.

Voyager Therapeutics Inc. has obtained IND clearance from the FDA for VY-1706, the company’s investigational gene therapy for the treatment of Alzheimer’s disease.

X-linked Alport syndrome is an inherited kidney disease caused by pathogenic mutations in the COL4A5 gene. Patients develop hematuria, proteinuria and kidney function decline leading to end-stage renal disease. Nionyx Bio Inc. has developed ONYX-101, a novel kidney-targeting therapeutic designed to ensure durable COL4A5 restoration through dual-vector AAV delivery using NYX capsids that were optimized for kidney targeting.