More than two decades ago, the Foundation Fighting Blindness (FFB) began funding RPE65 gene therapy research that led in late 2017 to LUXTURNA, the first FDA-approved gene therapy for the eye or an inherited condition. Ultimately, the Foundation provided more than $10 million in funding for the groundbreaking effort.
ProQR, a biotech company in the Netherlands, has reported vision improvements for patients in a Phase 1/2 clinical trial for QR-110, a therapy for people with Leber congenital amaurosis 10 (LCA10), which is caused by the p.Cys998X mutation in the CEP290 gene. The mutation is estimated to affect about 2,000 people in the Western world.
The Foundation Fighting Blindness (FFB), the world’s leading private funding source for inherited retinal disease research, announced major awards in two areas: promising research projects in retinal diseases and career development for clinical researchers.
“The first time I examined a person’s retina as a junior resident, something clicked. All the things I found interesting came together — surgery, patient care, genetics, regenerative medicine, and cell biology,” says Mandeep Singh, MD, PhD, assistant professor in ophthalmology, Wilmer Eye Institute, Johns Hopkins Medicine. “I knew retina was what I wanted to focus on for my career.”
The Foundation Fighting Blindness has announced funding for seven new research projects to advance the development of treatments and cures for retinal degenerative diseases. Each project will receive a total of $300,000 over a three-year period.
Hosted by the Foundation Fighting Blindness and Casey Eye Institute at Oregon Health & Science University, the Innovation Summit for Retinal Cell and Gene Therapy has emerged as one of the most essential events for researchers and companies developing treatments and cures for retinal degenerative diseases.
An important bill has been introduced to the US House of Representatives that has the potential to greatly increase research funding for all blinding eye conditions, including retinal diseases such as retinitis pigmentosa, Leber congenital amaurosis, Usher syndrome, and age-related macular degeneration.
Bill Introduced in U.S. House Would Speed Up Cures for Blindness
Four research and development leaders share the latest details of their groundbreaking work in therapies for retinal degenerative diseases