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Posts tagged X-linked retinoschisis

FFB’s Investments Are Filling the Pipeline for Vision-Saving Therapies

GXM_7140With 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.

At the Foundation’s Investing in Cures Summit on September 16 in Chicago, Sue Washer, AGTC’s chief executive officer, emphasized FFB’s crucial role in moving the company and its projects forward. “We as an organization would not be here today without FFB,” she said. “And that all started with the work that was funded by the Foundation in Bill Hauswirth’s lab at the University of Florida.” Bill Hauswirth, PhD, is one of AGTC’s scientific co-founders, and his groundbreaking gene-therapy research has been funded by FFB for 20 years.
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Twelve People Receive XLRS Gene Therapy in AGTC’s Clinical Trial

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.
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Two New Videos Highlight FFB’s Vital Role in Retinal Research

Dr. Boye in her lab, conducting a tourAs everyone knows, there is never just one side to a story. That’s certainly true in the case of Dr. Shannon Boye, whose FFB-funded research is the subject of not just one but two new Foundation videos.
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Retinal Researchers May Be Looking for You

A patient registers with My Retina Tracker.One of the biggest challenges in overcoming rare retinal diseases is, well, that they’re rare. There’s limited information about the conditions in humans, making it difficult for researchers to understand why they cause blindness and develop vision-saving treatments.
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CME – What It Is, and Why People with Retinal Diseases Should Know About It

Dr. FishmanIn this Q&A, Gerald Fishman, M.D., a world-renowned clinical researcher and longstanding member of FFB’s Scientific Advisory Board, discusses cystoid macular edema (CME), a potentially damaging accumulation of fluid in the retina affecting up to 32 percent of people with retinitis pigmentosa (RP) and related conditions. It’s a complication that can make matters worse for retinas already fragile from degenerative disease. While diagnosis isn’t always straightforward, CME can be treated to minimize vision loss.
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