News

FDA opens probe into mysterious death linked to gene therapy
The Food & Drug Administration is investigating the death of an 8‑year‑old following Elevidys gene therapy for Duchenne ...
Fierce Biotech1d
'We will crack gene therapy': Elevidys fallout doesn't dent Roche's hopes for modality
Roche’s move came against a backdrop of wider industry retreat from gene therapy, with Pfizer pulling its FDA-approved ...
Sarepta denies Elevidys is linked to latest patient death in Brazil
Shortly after the U.S. FDA announced an investigation into the death of a patient who had received Elevidys, a gene therapy ...
STAT1d
Europe moves to reject embattled Duchenne muscular dystrophy gene therapy
European regulators said that a Duchenne gene therapy should not be approved, imperiling the treatment that is also facing ...
Fierce Pharma1d
Roche won't throw in the towel after DMD gene therapy Elevidys' rebuff in Europe
Despite a new setback for Elevidys in Europe, Roche—which markets Sarepta’s gene therapy outside the U.S.—remains committed ...
BioPharma Dive1d
Sarepta woes mount as Duchenne gene therapy knocked back in Europe
The European Medicines Agency determined testing failed to prove Elevidys’ benefit and issued a negative opinion that could ...
STAT2d
For many Duchenne families, halt to gene therapy is heartbreak upon heartbreak
As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...
Sarepta Therapeutics laying off dozens in Ohio, won't comply with FDA request to halt therapy
After the FDA request, Cambridge, Massachusetts-based Sarepta said in a statement that it will continue to ship the therapy ...