Gene therapy is an emerging class of treatment that targets disease at its source by repairing or replacing a faulty gene. Unlike traditional drugs that require ongoing use, many gene therapies are designed as one‑time interventions with the potential for long‑lasting – sometimes even curative – results.
✔️ Recent FDA Approvals (2025-2026):
• Kresladi - first FDA-approved therapy for a ultra-rare immune disorder (severe leukocyte adhesion deficiency type 1 (LAD-1)
• Casgevy - Sickle Cell Disease, Transfusion-Dependent Beta-Thalassemia
• Roctavian - Hemophilia A
• Elevidys - Duchenne Muscular Dystrophy
Why do these approvals matter for coverage strategy?
• Ultra-high cost – often $1-$3 million per treatment
• One-time therapies that may be curative, reshaping long-term cost models
• Expansion into more common diseases
• Importance of monitoring durability of outcomes
Major Categories of Gene Therapy Products:
• Gene Replacement Therapies – these therapies deliver a functional gene into a patient’s cells using a modified virus.
Common uses include: Inherited blood disorders, Hemophilia, Inherited retinal diseases, Neuromuscular disorders
Examples: Luxturna, Zolgensma, Hemgenix, Roctavian
• Gene-Edited Therapies – these therapies edit a patient’s DNA rather than adding a new gene.
Common uses include: Sick Cell Disease, Beta-Thalassemia
Examples: Casgevy
• CAR-T Gene Therapies (Genetically Modified Immune Cells) – these are autologous T-cells engineered to attack cancer cells.
Common uses include: Leukemia, Lymphoma, Multiple Myeloma
Examples: Kymriah, Yescarta, Abecma, Carvykti
• Stem Cell Gene Therapies – patient stem cells are modified outside the body, then reinfused.
Common uses include: Rare metabolic disorders, Immune deficiencies, Neurologic genetic diseases
Examples: Syksona, Zynteglo, Lyfgenia
