RetroSense Therapeutics has received Orphan Drug designation from the Food and Drug Administration (FDA) for its lead product, RST-001, to treat retinitis pigmentosa (RP).
RP is a rare eye disease in which light receptors in the eye gradually die off, eventually leading to blindness. RP can be due to any one of over a hundred different gene defects. Currently there is no conventional treatment or cure, but nutritional therapies have been shown to slow the progress of the retinitis pigmentosa.
However, RetroSense is employing a new approach. They use gene therapy to install new light receptor genes in cells that previously had no ability to sense light. This approach may be useful in treating RP, no matter what the original cause of the disease.
RetroSense Therapeutics CEO Sean Ainsworth called the Orphan Drug designation a “significant milestone” and said, “We are pleased that the FDA has granted Orphan Drug status to our lead product, RST-001.”
Orphan Drug status is granted for drugs that treat rare diseases, in order to make it easier for companies to gain marketing approval. Research has been carried out at Wayne State University and Massachusetts General Hospital, and early studies have been promising.
Source: RetroSense Press Release