Coave Therapeutics Preparing for Retinitis Pigmentosa Gene Therapy Registration Trial
In an ongoing Phase I/II trial, the gene therapy improved visual function in patients with early stages of the rare, inherited visual disorder.
Lupus Research Alliance Launches Landmark Study to Speed Personalized Drug Development
Investigators aim to enroll 3,500 patients into the prospective, observational study, which is supported by multiple drugmakers.
FDA Delays Decision for Sarepta Therapeutics Duchenne Muscular Dystrophy Gene Therapy
According to Sarepta, the federal agency has indicated it may grant accelerated approval to SRP-9001 as a treatment for a subgroup of patients based on age.
Therini Bio Kicks Off Human Testing of Fibrin-Targeting Alzheimer's Drug
THN391 is the firm's lead asset and first clinical-stage candidate, which it will initially test in healthy subjects and then in patients with dementia and higher levels of fibrin.
Locanabio Gets CureDuchenne Investment to Support Gene Therapy Development
The equity investment of an undisclosed amount will fund therapeutic development efforts that use AAV vectors to deliver snRNAs that promote exon skipping.