The gene therapy field progresses amid regulatory challenges and promising clinical data. Stealth BioTherapeutics resubmitted its investigational drug application for Barth syndrome after addressing FDA concerns, seeking accelerated review. Concurrently, UMass Chan Medical School reported encouraging Phase I/II trial results of a dual-vector gene therapy correcting biochemical deficits in GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases. Additionally, Kriya Therapeutics secured a $313 million funding round to advance its pipeline targeting neurology, metabolic health, and ophthalmology, underscoring ongoing investor confidence despite broader sector headwinds.