Some in the gene therapy field lament lost time while Capsida kept silent about the brain receptor it was targeting when things went wrong.
Photo illustration: STAT; Photo: Adobe
By Jason Mast
May 12, 2026
General Assignment Reporter
Jason Mast
With new data, Regenxbio to seek FDA approval of Duchenne gene therapy
After an apparent cure, a gene editing company struggles to repeat the magic
A year after Vertex’s big launch, pain drug research faces a pivotal moment
Boy’s brain tumor tied to gene therapy
Updated: Biogen won’t be ‘impulsive’ with anti-tau Alzheimer’s drug
Encoded Therapeutics gives a heads-up to other gene therapy company about the trade-offs with a drug meant to improve safety.
The FDA will reconsider approving Regenxbio's experimental gene therapy for a deadly and rare childhood brain disorder it…
Doctors have linked a boy's tumor to a gene therapy, but say the rare risks need to be weighed against the profound benefits such…
Regenxbio plans to submit its Duchenne gene therapy for FDA approval, just a month after it said that regulators wanted to see…
Grace Science CEO Matt Wilsey says FDA demands could kill its gene therapy for the rare and fatal NGLY1 deficiency by September…
Some boys had astonishing recoveries. But other boys died. Now Astellas is returning to revised gene therapy for XLMTM.
