Usher III Initiative Receives $1M Grant from Foundation Fighting Blindness to Advance Novel Treatment for Usher Syndrome Type 3

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. 

Usher III Initiative Receives $1M Grant from Foundation Fighting Blindness to Advance Novel Therapy for Usher Syndrome

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." 

About Usher III Initiative

The Usher III Initiative is a 501(c)(3) nonprofit medical research organization founded by Cindy Elden and the late Richard Elden, to address the unmet medical needs of individuals with USH3. Over the last 18 years, the Initiative's efforts led to the discovery and optimization of BF844, a novel compound that targets the underlying disease pathology of USH3. This non-invasive, first-in-class therapeutic candidate has the potential to vastly improve clinical outcomes for a patient population with no other options. 

About Foundation Fighting Blindness

Established in 1971, the Foundation Fighting Blindness is the world's leading private funding source for retinal degenerative disease research. The Foundation has raised more than $816 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the entire spectrum of blinding retinal diseases including: retinitis pigmentosa, macular degeneration, and Usher syndrome. Visit FightingBlindness.org for more information. 

For more information, contact us at info@usheriii.org


Previous
Previous

In the Spotlight: Michael and Jonathan Tutnauer

Next
Next

Newsletter - Winter 2020