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 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.
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 moreMelanoma Vaccine Update
The FDA has granted breakthrough therapy designation for mRNA-4157 and Pembrolizumab combination for melanoma.
Read moreHumira Biosimilars Now Available in the US
On January 31, 2023, Amgen introduced a biosimilar for one of the pharmaceutical’s industry’s most lucrative products. Amjevita became first of nine biosimilar lookalikes scheduled to launch this year to compete with Humira (adalimumab).
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