With dozens of experimental gene therapies floating down the research and development pipeline, here are five candidates grabbing headlines:
1. An FDA advisory meeting for Sarepta Therapeutics' SRP-9001 (delandistrogene moxeparvovec) is set for May 12. The gene therapy is for Duchenne muscular dystrophy, a rare disorder the CDC says affects 1 in 5,000 boys between 5 and 9 years old.
2. BioMarin Pharmaceutical's Roctavian (valoctocogene roxaparvovec), a gene therapy for adults with severe hemophilia A, is scheduled to receive the FDA's final decision by June 30. It is one of the four drugs Optum is keeping an eye on.
3. Bluebird Bio submitted an application in late April for its lovotibeglogene autotemcel gene therapy for sickle cell disease, which the Institute for Clinical and Economic Review said might be worth up to $1.9 million. If the FDA approves the application for priority review, the agency would plan to make a decision before November.
4. Vertex Pharmaceuticals and CRISPR Therapeutics filed an application for exagamglogene autotemcel in April and, if priority review is accepted, its decision date would fall in early December. The ICER priced this sickle cell disease therapy around the same as Bluebird Bio's candidate. Read about its potential for patients here.
5. Pfizer found strong phase 3 results for fidanacogene elaparvovec as a hemophilia B gene therapy in late 2022, and the company said it would discuss the results with regulators in early 2023.