Foundation News » Other Retinal Diseases
Foundation-Funded Gene Therapy the Focus at International Biotech Conference
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The success of vision-restoring gene therapy clinical trials continues to get outstanding recognition from a variety of media, research, and advocacy organizations. Most recently, key leaders in the biotechnology community and the federal government have been enthusiastically telling the story. At the 13,000-attendee annual international conference of BIO, the world's largest biotechnology advocacy, development, and communications organization, BIO Chairman Dr. Stephen Sherwin spoke glowingly of the success in Foundation-funded gene therapy studies and the potential gene therapy holds for treating a broad range of diseases. Specifically, he told the story of nine-year-old Corey Hass, who has realized dramatic vision restoration after receiving gene therapy for Leber congenital amaurosis (LCA). Dr. Sherwin’s keynote address on May 5 included a touching video about Corey’s story — a video created specifically for his remarks at the event. Also at the BIO event, the Foundation’s Bill Schmidt, chief executive officer, and Dr. Stephen Rose, chief research officer, participated in a satellite media tour on the gene therapy. They spoke with 21 television and radio outlets from around the country about the Foundation and the success of the clinical trials. Earlier this year, Dr. Francis Collins, the new director of the National Institutes of Health, recently highlighted the success of LCA gene therapy studies during his annual testimony to Congress. He’s using these studies to underscore the importance of research funding and the dramatic results it can bring. Thus far, 25 people have had one eye treated in three different LCA gene therapy clinical trials. Researchers are working with the FDA to obtain approval to treat the participants’ other eyes. Success in these clinical trials has opened the door for using gene therapy to treat a variety of retinal degenerative diseases. Oxford BioMedica, a U.K.-based commercial partner of the Foundation is planning fall launches of clinical studies for two gene therapies — one for Stargardt disease (StarGen®) and the other for wet age-related macular degeneration (RetinoStat®). Dr. José Sahel, the director of the Foundation grant center in Paris, will be leading the Europe-based study of StarGen. |
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