gene therapy

News

uniQure has revealed that the FDA has said it is not prepared to review its Huntington's gene therapy in what seems to be a change in policy.

News

Rocket Pharma has withdrawn a filing in the US for its experimental gene therapy for Fanconi anaemia, but may consider partnering the programme.

News

uniQure's gene therapy AMT-130 has cleared a trial in Huntington's, setting up filings and raising the prospect of a first treatment for the disease.

News

The FDA has added a warning label to bluebird bio's Skysona, after launching a probe into cases of blood cancer linked to the gene therapy.

News

Sarepta has restarted distribution of its Duchenne muscular dystrophy gene therapy Elevidys, but only for patients who can walk.

News

Sangamo plans to file for approval of its gene therapy for Fabry disease next year, but a launch could depend on finding a commercial partner.