ProQR, a biotechnology company in the Netherlands, has received authorization from the U.S. Food and Drug Administration to start a Phase I/II clinical trial for its therapy known as QR-110, which is being developed for Leber congenital amaurosis type 10 (LCA 10). The genetic retinal condition causes severe vision loss in children.
The Foundation Fighting Blindness (FFB), the world’s leading private funder of research aimed at preventing, treating and curing blindness caused by inherited retinal diseases will hold its Fashion and Finance Ball on Tuesday, May 16 at New York’s Plaza Hotel. A cocktail reception and silent auction will open the evening at 6:00 p.m.; the dinner and awards program will begin at 7:00 p.m.
This is a great story of how the Foundation Fighting Blindness (FFB) provided timely funding of $155,000 to help my lab at the University of California, San Diego (UCSD), leverage a $2 million retinal-gene discovery project.
What is very rewarding for me is that FFB’s support helped us find the retinal-disease gene mutation in 33 families, who were otherwise left without a clear diagnosis. Now these families are better able to understand their prognosis and which clinical trials and future therapies may be most relevant to them.
Genes are like the blueprint or code for determining who we are. We all have about 23,000 pairs of genes in most cells in our bodies. Many of our physical attributes — such as height, eye and hair color, and complexion — are determined by our genes.
However, certain misspellings, also known as mutations, in our genetic code can cause diseases or increase our risk for them. In fact, each inherited retinal disease (IRD) is caused by a mutation in a single gene.
The Association for Research in Vision and Ophthalmology (ARVO)’s annual conference – Baltimore, May 7-11 – gathers the field’s leading researchers to report on advancements in vision science including the search for cures for retinal degenerative diseases.
These are retinal progenitors.
The stem-cell therapy company jCyte is launching a Phase IIb clinical trial of its therapy for people with retinitis pigmentosa (RP). The trial is taking place at University of California, Irvine, and Retina-Vitreous Associates Medical Group in Los Angeles. The 70-participant study is being led by Henry Klassen, MD, PhD. Participant enrollment is scheduled to begin this month.
The Foundation Fighting Blindness (FFB) will hold its annual For the Love of Sight dinner on Tuesday, May 2, 2017 at the Ritz Carlton, Washington – reception 6:00 p.m., dinner 7:00 p.m.
Sai Chavala, MD
The Foundation Fighting Blindness (FFB), a national nonprofit focused on funding cutting-edge retinal disease research, and the Casey Eye Institute at Oregon Health & Science University will co-host the fourth annual Retinal Cell and Gene Therapy Innovation Summit in Baltimore, May 4–5, 2017.