02-28-2022

Gene Therapies Continue to Emerge as Effective Treatments for Rare Diseases, But Complex Challenges Remain

Genethon, a non-profit research organization focused on developing gene therapies for rare diseases, today recognizes International Rare Disease Day by reflecting on successes and highlighting challenges that remain in bringing hope to 300 million patients worldwide who suffer from more than 7,000 rare diseases.

“As a world leader in gene therapy, Genethon is making a unique contribution to finding treatments and cures for rare diseases,” said Frederic Revah, Ph.D., Genethon CEO. “Products emerging from our research make up a global pipeline of treatments for patients who have no therapeutic alternatives. Today, thousands of patients around the world are already benefiting from our research.”

Products from Genethon’s research include a marketed gene therapy for spinal muscular atrophy and 12 other treatments in clinical trials for rare diseases of the muscle, immune system, eye, and liver. Another seven products from Genethon’s research should enter clinic trials in the next few years.

However, Dr. Revah observes. “While therapeutic successes are multiplying, demonstrating the full relevance of this breakthrough technology, the development of gene therapy for rare diseases is coming up against scientific, technological, clinical, economic and financial obstacles, which jeopardize making it available to as many people as possible.”

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