Gene Therapy Transforms Rare Disease Care

Gene therapy is changing the game for patients with rare diseases, offering hope and tangible results for those who once had few, if any, treatment options. This groundbreaking technology works by repairing faulty genes in the body, targeting the root cause of many rare diseases and offering the potential for life-changing benefits.

Unlike traditional treatments, gene therapies have the potential for long-term, even curative effects. This means that patients can enjoy longer, healthier lives while avoiding the immense burden and high costs that often come with current standards of care. For many, gene therapy isn’t just a treatment; it’s a new lease on life.

As research continues to advance, gene therapy will likely extend its reach, with the potential to treat even more diseases with genetic roots. In fact, there are currently more than 2,000 gene therapy drugs in development. This growing pipeline offers hope for the approximately 6,500 rare diseases that still lack an FDA-approved treatment, providing the promise of better, more effective options for patients in the near future.¹