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Posts tagged AGTC

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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AGTC Leverages Funding from the Foundation to Move Promising Treatments into Clinical Trials

Seed becoming a plantCompany Builds on FFB’s Initial Investment to Garner $265 Million in Therapy Development Funding

In the early 1990s, scientists began discovering the genetic defects causing blinding, inherited retinal diseases and saw a unique opportunity to overcome them. They envisioned gene therapy — delivering healthy genes to the retina to replace the bad ones — as an elegant approach to saving and restoring vision. Furthermore, a single injection of gene therapy would likely halt or reverse the disease process and work effectively for several years, perhaps the patients’ lifetimes.

The Foundation Fighting Blindness, the world’s leading private, nonprofit retinal research organization, funded most of these genetic discoveries for retinal diseases and immediately recognized the enormous opportunity for gene therapy to beat blindness.
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Fighting Blindness Gets Sheepish

A sheep in tall grass.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?
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Israeli Research Group Receives $1.33 Million to Advance Stem Cell Treatment

Dr. Benjamin Reubinoff

I was heartened to just learn that researchers from the Hadassah-Hebrew Medical Center in Jerusalem are receiving a $1.33 million grant from the Israeli government to advance their development of a stem cell treatment for people with dry age-related macular degeneration (AMD Hadassah is also funded by the Foundation Fighting Blindness. We are providing the group with a three-year, $300,000 grant for development of stem cell therapies.

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