Gene Therapy

December 24th 2024

Gene therapies are revolutionizing treatment for hemophilia A and sickle cell disease; however, they are saddled with hefty price tags and limited patient populations that are impacting uptake.

December 12th 2024

Research at the annual meeting of the American Society of Hematology evaluated patient and caregiver perspectives on gene therapies for sickle cell disease, which offer great potential but have had slow uptake.

November 22nd 2024

Gene therapies have a lot of potential to improve health outcomes for patients with life-threatening diseases but face a number of barriers that restrict access.

November 14th 2024

Kebilidi is a one-time gene therapy administered directly to the brain in patients with AADC deficiency, rare genetic disorder.