The Foundation Fighting Blindness (FFB), the world’s leading private funder of research on treatments and cures for vision-robbing inherited retinal diseases, strongly opposes recent U.S. Senate and House of Representatives actions which would slow or stop research on treatments for Americans impacted by rare diseases.
Though the U.S. Food & Drug Administration (FDA) is more tightly regulating U.S. clinics that tout stem-cell trials and treatments for inherited retinal diseases (IRDs) and a host of other conditions, many significant loopholes and enforcement limitations remain.
ProQR, a biotech company in the Netherlands, has treated its first patient 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) announced that it has selected Benjamin Yerxa, a biotechnology and drug development executive, as its Chief Executive Officer, effective immediately.
Ashley and Cole Carper traveled from Little Rock, AR, to tell their family’s story at the FDA hearing.
Approval of the gene therapy will allow treatment for people with vision-loss due to RPE65 mutations and will help advance other investigational studies with potential to help millions, FFB tells the Committee.
With five gene-therapy clinical trials underway or soon to begin, Applied Genetic Technologies Corporation (AGTC) is generating tremendous excitement for the potential to overcome vision loss from several inherited retinal diseases.
The Foundation Fighting Blindness (FFB), the world’s leading private funder of research on preventions, treatments and cures for vision loss due to inherited retinal diseases is accepting applications for its next round of funding grants to be awarded May 2018.
MeiraGTx, a gene-therapy company in London and New York City, has treated its first patient in a gene-therapy clinical trial for people with X-linked retinitis pigmentosa (XLRP) caused by mutations in the gene RPGR. The Phase I/II study is taking place at Moorfields Eye Hospital in London. The safety-oriented trial will enroll 36 participants. Three dose levels of the therapy will be evaluated.
The first patient has been enrolled in a four-year natural history study of people with mutations in the USH2A gene.