Usher III Initiative is developing BF844, a first-in-class small molecule therapy, for the treatment of Usher syndrome type III (USH3), a rare and debilitating genetic disorder that results in progressive loss of hearing and vision. The Initiative is delighted to announce the award of a Translational Research Acceleration Program (TRAP) grant for 1 million dollars from the Foundation Fighting Blindness to complete pre-IND toxicity studies.
The TRAP program was established to advance promising therapeutic candidates towards clinical trials. “Our TRAP grantees are exceptional scientists developing therapies that have strong potential to reach patients with retinal diseases,” says Chad Jackson, PhD, director of the Foundation’s TRAP program. The grant awarded to the Initiative is designed to support late-stage projects requiring one or more critical experiments to satisfy a transition requirement in order to reach human clinical trials.
USH3 is an inherited disease caused by a single mutation in the CLRN1 gene. Most individuals with USH3 are profoundly deaf and legally blind by age 50. There is no existing cure for the disease. “We are so appreciative of this critical support from the Foundation Fighting Blindness,” commented Cindy Elden, President and Co-Founder of the Initiative and an USH3 patient, “We have worked long and hard to get to this point and these funds will make a significant contribution to advancing BF844 to the clinic and the patients that so desperately need this drug.”