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Best Read Gene Therapy Stories of 2023


FDA Approves First Cellular Therapy for Type 1 Diabetes

In June, the FDA approved Lantidra (donislecel), the first allogeneic (donor) pancreatic islet cellular therapy to treat patients with type 1 diabetes. In two small studies, patients were able to be free from insulin injections for a year or more.

First Gene Therapy for Duchenne Priced at $3.2 Million

Elevidys is a one-time therapy that delivers to muscle a gene that codes for a shortened, functional form of dystrophin, which is mutated in Duchenne muscular dystrophy.

State Medicaid Programs Look to Value-Based Contracts for Gene Therapies

Magellan Rx Management has begun providing a multi-state solution in which they negotiate with drug manufacturers for value-based contracts for high-cost gene and cell therapies.

FDA Approves Two Gene Therapies for Sickle Cell Disease

The FDA has approved the gene therapies Lyfgenia and Casgevy to treat patients with sickle cell disease. Casgevy is the first FDA-approved gene therapy to use the CRISPR gene editing technology.

FDA Approves $2.9 Million Gene Therapy for Hemophilia A

BioMarin is offering an outcomes-based warranty that will reimburse payers up to 100% of the cost if a patient does not respond to Roctavian.

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