Gene Therapy for Rare Diseases

Rocket Pharma specializes in finding cures for genetic ailments using gene therapy. They target rare diseases, such as Fanconi Anemia, LAD1, and pyruvate kinase deficiency, as well as certain cardiac conditions. Their therapies involve replacing faulty genes to restore normal function. The company works closely with the FDA, which plays a critical role in ensuring clinical safety and efficacy. Rocket Pharma's approach provides hope for patients and families facing devastating diseases, and their work may also have implications for the treatment of chronic lifestyle diseases in the future.