November ushered in two new therapies, AUCATZYL® (obecabtagene autoleucel), a CAR-T therapy which was approved on 11/8/2024, and Kebilidi™ (eladocagene exuparvovec-tneq), a gene therapy which was approved on 11/13/2024.
Read moreSickle Cell Gene Therapies and CMS Cell and Gene Therapy Access Model
The federal government has created a program called the Cell and Gene Therapy Access Model, the goal of which is to provide people with Medicaid who have rare and severe diseases access to potentially transformative treatments.
Read moreGene Therapy Approved for Transfusion-Dependent Thalassemia
With the approval of Casgevy, there are now two gene therapies, Casgevy and Lyfgenia, for the treatment of transfusion dependent thalassemia (TDT).
Read moreBlood Cancer: Advancements in CAR-T Cell Therapies
September is Blood Cancer Awareness Month, a global event to help raise awareness of one of the world’s most prevalent and dangerous cancers. Accordingly, we examine advances in research that have the potential to improve treatments and outcomes.
Read moreCell & Gene Therapy Update: July 2023
June was a busy month for the FDA with the approval of two gene therapies, Elevidys (delandistrogene moxeparvovec-rokl) and Roctavian (valoctocogene roxaparvovec-rvox), and one cellular therapy Lantidra (donislecel-jujn). See more about these therapies.
Read moreFirst Allogeneic Cellular Therapy Approved for Type 1 Diabetes
On June 28, 2023, the U.S. Food and Drug Administration approved Lantidra, the first allogeneic (donor) pancreatic islet cellular therapy made from deceased donor pancreatic cells for the treatment of type 1 diabetes.
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