Applied Genetics Technology Corporation (AGTC) reported that its gene therapy for X-linked retinoschisis (XLRS) has performed encouragingly in a Phase I/II, safety-oriented clinical trial taking place at seven sites in the U.S.
XLRS is an inherited disease that leads to significant vision loss due to splitting of the layers of the retina. The condition affects about 35,000 males in the U.S. and Europe. XLRS is caused by mutations in the gene retinoschisin. AGTC’s gene therapy uses a human-engineered virus — and adeno-associated virus or AAV — to deliver normal copies of retinoschisin to the patient’s retina.
Continue Reading…
For anyone wanting to know about the latest clinically focused research for inherited retinal diseases (IRDs), the 
A 29-year-old British man is the first person to be treated in a gene therapy clinical trial for X-linked 
Company Builds on FFB’s Initial Investment to Garner $265 Million in Therapy Development Funding
A retinal researcher can develop the best vision-saving stem-cell or gene therapy ever imagined, but if the doctor or surgeon can’t deliver it to the retina safely and effectively, the treatment is worthless. It could even be harmful.
We all know from the nursery rhyme “Mary Had a Little Lamb” that “everywhere that Mary went, the lamb was sure to go.” But what if the lamb had a vision-robbing retinal disease, making it challenging to find Mary prancing around a sunlit meadow, or catching some rays on the Jersey Shore?
The annual meeting of the 
Steve is highly respected for his expertise and tireless commitment to finding treatments and cures for vision-robbing retinal diseases. 
As the Foundation's Director, Science Communica- tions, Ben writes science and research articles for the Foundation’s website, newsletters and Eye on the Cure blog. 