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 moreFirst Gene Therapy for Hearing Loss
The first gene therapy for hearing loss, a dual adeno-associated viral (AAV) vector-based gene therapy, is currently being evaluated in the phase 1/2 AK-OTOF-101 clinical trial (NCT05821959). It seeks to replace the nonfunctioning OTOF gene in the cochlea with a working copy of the OTOF gene.
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 moreIt Costs How Much?!
Most plans have a precertification process in place for high-dollar drugs that addresses the appropriateness for diagnosis as well as provider network status; however, this is where it stops.
Read moreMetachromatic Leukodystrophy (MLD) and Gene Therapy
Metachromatic Leukodystrophy (MLD) is a rare recessive genetic disorder caused by a mutation in the arylsulfatase-A (ARSA) gene, which normally breaks down sulfatide, resulting in the toxic buildup of lipids and other storage materials in cells in the white matter of the central nervous system and peripheral nerves.
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 moreSickle Cell Disease: Hope for the Future
Every June 19, we observe World Sickle Cell Day to raise international awareness of sickle cell disease and the challenges patients and families face when handling the disease. Sickle cell disease is a group of inherited red blood cell (RBC) disorders that affect hemoglobin, the protein that carries oxygen through the body.
Read moreGene Therapy Update: SRP-9001 delandistrogene moxeparvovec for DMD
The FDA has pushed back the decision on the experimental gene therapy for Duchenne Muscular Dystrophy (DMD) until June 22.
Read moreApproval of Gene Therapy Treatment for CALD Anticipated in September 2022
Lenti-D (elivaldogene autotemcel or eli-cel) is an investigational gene therapy being developed by bluebird bio for the treatment of childhood cerebral adrenoleukodystrophy (CALD). The FDA advisory committee reviewed it in June 2022 and unanimously approved drug/therapy 15-0. The FDA’s scheduled decision date on the therapy is September 16, 2022.
Read moreGene Therapy Update: Beti-cel
This week a decision should be made by the FDA on a new gene therapy, betibeglogene autotemcel (beti-cel). Beti-cel is a one-time autologous product designed to treat transfusion-dependent beta thalassemia.
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