The AAV Innovation Summit 2025, hosted by Form Bio, will bring together top experts in gene therapy to discuss the latest ...

Organoids were cultured from healthy donor iPSC sources and differentiated using the human iPSC-derived Cerebral Organoid ...

AAV-Navβ1 gene therapy effectively reduces seizures and prolongs life in a mouse model of SCN1B-linked Dravet syndrome, ...

D Molecular Therapeutics is undervalued, with its lead candidate 4D-150 showing potential. Click here to find out why I rate ...

Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in ...

Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...

Day 90 biopsy data reported from first 3 participants dosed in Phase 1/2 INSPIRE DUCHENNE trial —— Average microdystrophin ...

The candidate in question is VY9323, an AAV gene therapy combining a highly potent pri-miRNA against superoxide dismutase 1 ...

Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...