‘It’s a vote for hope’: first gene therapy for muscular dystrophy nears approval, but will it work?
By Sara Reardon,
Nature
| 06. 02. 2023
The road to gene therapies for genetic disorders has been long — and expensive — but the field could soon get some good news. On 22 June, the US Food and Drug Administration will decide whether to grant a fast-track approval to the first gene therapy for Duchenne muscular dystrophy (DMD), a genetic disorder that affects around 1 in 3,500 boys. Children with DMD can’t make a protein called dystrophin, resulting in progressive muscle degeneration and death in their twenties due to heart or respiratory failure.
The therapy, known as SRP-9001, is made by Sarepta Therapeutics based in Cambridge, Massachusetts. It would be the 13th gene therapy that the FDA has approved since 2017, and the first to target a prevalent genetic disease in children. The accelerated approval would allow the drug to reach the market before large clinical trials have been completed, on the basis of evidence that the therapy allows boys to make an engineered form of dystrophin.
The decision date was delayed late in May after FDA officials and advisers raised concerns about the strength of Sarepta’s...
Related Articles
By Fyodor Urnov, Time | 08.12.2024
After a lifetime in the field of epigenetics, and nearly 20 years after my colleagues and I coined the term “genome editing,” I will be the first to admit that describing the “epigenome”—a marvelous biological process that guides...
By Azeen Ghorayshi and Sarah Kliff, The New York Times | 08.12.2024
An emerging movement against in vitro fertilization is driving some doctors and patients in red states to move or destroy frozen embryos.
The embryo migration is most striking in Alabama, where the State Supreme Court ruled in February that embryos...
By Gemma Conroy, Nature | 06.27.2024
Image by Robina Weermeijer from Unsplash
A molecular-editing tool that’s small enough to be delivered to the brain shuts down the production of proteins that cause prion diseases, a rare but deadly group of neurodegenerative disorders.
The system — known...
By Staff, AP-NORC | 07.12.2024
Image by Dr. Jayesh Amin from Wikimedia Commons licensed under CC by-SA 3.0
Most adults support protecting access to in vitro fertilization, or IVF, a type of fertility treatment where eggs are combined with sperm outside the body in a...