Particle.news

Download on the App Store

Bluebird Bio Files for FDA Approval of Gene Therapy Treatment for Sickle Cell Disease

  • Bluebird Bio has submitted an application to the FDA for approval of its gene therapy to treat sickle cell disease.
  • The company is seeking priority review, which could speed up the approval process.
  • The therapy aims to treat sickle cell disease by delivering a correct copy of the gene into patients' cells.
  • In a clinical trial, the treatment resolved pain crises for all 25 evaluated subjects.
  • While the therapy shows promise, it still faces questions about potential risks and safety concerns.
Hero image