The Latest Breaking News from Gene Therapy

The treatment is the first to be licensed using the gene-editing tool known as Crispr, for which its inventors were awarded the Nobel prize in 2020.

A new gene therapy targeting an enzyme involved in familial hypercholesterolemia slashes bad cholesterol in a small clinical trial.

‘It’s really important that we get the first gene therapy treatment right’

Sarepta Therapeutics' gene therapy, Elevidys, faces an uphill regulatory journey, analysts said Tuesday on mixed test results.

There soon may be a new cure for sickle cell disease that attacks the disorder at its genetic source

The big names behind biotech stocks no longer want to merely treat diseases, they want to cure them. But it's going to be costly.

US research linked to dopamine release finds macaques decreased consumption by up to 90% after treatment

A small, proof-of-concept study in monkeys reveals the potential of a one-off gene therapy to treat people with alcohol use disorder who haven't responded to other treatments.

Narayana Nethralaya to start human clinical trials of indigenous gene therapy for ocular diseases; first such effort in India. Cost of therapy to reduce from ₹7 crore to 1/20th-1/50th of international costs. Meeting of 25 clinicians & scientists held to come out with consensus & establish national registry for rare…

A teen with dystrophic epidermolysis bullosa, or "butterfly disease," was legally blind but has now had his sight restored with gene therapy.

Antonio Vento Carvajal has been legally blind for much of his 14 years