The U.S. FDA marked the 40th anniversary of the Orphan Drug Act with Rare Disease Day 2023 as Robert Califf, the agency’s commissioner of food and drugs, opened the day by expressing his wonder and accompanying concern regarding gene editing and gene therapy.
The Rare Disease Day, which takes place at the end of February each year, is designed to focus global attention on the need for therapies to treat patients suffering from devastating rare diseases. The most recent event represented the 13th year it has been held. Over that period, research and development in the area has come a long way, and there are now 420 companies around the world that are active in developing regenerative medicines and advanced therapies for the treatment of rare diseases, according to a new report released by the Alliance for Regenerative Medicine (ARM).