gene therapies

News

A third fatality has been recorded among patients receiving treatment with a Sarepta gene therapy, adding to the troubles being faced by the company.

News

Sanofi has been granted a speedy review from the FDA for a gene therapy for geographic atrophy, a major cause of blindness.

News

The FDA has launched a formal investigation into two liver failure-related deaths in patients treated with Duchenne gene therapy Elevidys.

News

Another death from liver failure has been seen with Roche and Sarepta's Duchenne muscular dystrophy therapy Elevidys, prompting a pause on treatment.

News

Three studies of Sarepta's Elevidys Duchenne gene therapy have been placed on hold as the death of a 16-year-old boy is investigated.

News

Boehringer's cystic fibrosis gene therapy is poised to start clinical testing, hoping to offer a one-shot alternative to costly CFTR modulator drugs.