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Posts tagged retinal

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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Stem-Cell Therapy for Retinitis Pigmentosa Safe Thus Far in Early Human Study

Dr. Klassen in his labAn emerging stem-cell-derived treatment designed to preserve and potentially restore vision in people with retinitis pigmentosa (RP) has demonstrated a favorable safety profile in an ongoing Phase I/II clinical trial at the University of California, Irvine. The therapy is being developed by the regenerative medicine company jCyte with trial funding from the California Institute for Regenerative Medicine. Earlier research funded by the Foundation Fighting Blindness helped advance this therapeutic approach toward a human study.

Given this trial is one of the first-ever for a stem-cell-derived therapy for RP, this safety report is good news and an important step in the right direction. We at the Foundation look forward to additional reports from this study in the coming years as the trial advances.

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