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Posts tagged clinical trials

Foundation Fighting Blindness and 4D Molecular Therapeutics Partner to Boost Retinal Gene Therapy Development

Promising gene therapies for inherited retinal diseases (IRDs) — many of which have moved into clinical trials — use viral delivery systems (aka vectors) to get copies of the therapeutic gene to the retinal cells that need them. Viruses are well suited when used for gene delivery, because they’re adept at penetrating cells with their genetic cargo. While some viruses in their natural state can cause illness, therapy developers remove the native genes and replace them with those that are therapeutic.

The Foundation Fighting Blindness and 4D Molecular Therapeutics (4DMT), an innovative leader in viral vector development, have formed a partnership to help companies and researchers quickly obtain and implement high-quality vectors for their retinal gene-therapy development efforts.
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FFB-Funded Scientists Report on Nine Promising Translational Research Efforts

Translational research — moving promising science out of laboratories and into clinical trials — is essential to getting vision-saving, retinal-disease treatments out to the millions who need them. With that said, translational research is also costly and high risk and requires extensive clinical development and regulatory knowledge.

The Foundation Fighting Blindness has taken the translational challenge head on by investing more than $75 million in therapy-development projects with strong clinical-trial potential through its Translational Research Acceleration Program (TRAP), which includes Gund-Harrington Scholar Awards.
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ID Your IRD: A Free Genetic Testing Program for Eligible People with Inherited Retinal Diseases


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, inherited retinal diseases are caused by mutations in single genes.
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Unregulated Stem-Cell Therapy Causes Severe Vision Loss for Three Florida Women

A report today in The New England Journal of Medicine (NEJM) describes the cases of three women with age-related macular degeneration (AMD) who lost much of their eyesight after receiving ocular injections of stem cells derived from their own fat tissue. All of the women had good enough eyesight to drive before the procedures. Each paid $5,000 to receive the injections from a private clinic in Sunrise, Florida. The New York Times and other major media outlets have published news stories on the NEJM report.
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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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Optogenetic Therapy Takes First Step Forward in Clinical Trial

Retrosense logoRetroSense Therapeutics has reported that three participants have received injections of its potential optogenetic therapy, known as RST-01, in a Phase I/II clinical trial. The patients were given the lowest dose of RST-01, and no adverse ocular events were observed. Furthermore, the treatment showed some biological activity, though RetroSense did not provide details about what that activity was or what it meant.

More information on safety and efficacy will likely be reported about the RetroSense trial after more trial participants have been observed over a longer period of time, and after discussions with the U.S. Food and Drug Administration. Continue Reading…

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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The Challenge Continues: FFB Chairman Gordon Gund is Interviewed on CNBC

Gordon Gund on CNBC in NantucketGordon Gund, who’s held in extremely high esteem by people inside and outside the Foundation Fighting Blindness (FFB), has been completely blind for decades. He lost his eyesight to a disease called retinitis pigmentosa in his thirties. Not that it slowed him down much. Among other accomplishments, he’s been a financier, venture capitalist, sports-team owner and sculptor. And, of course, he’s a co-founder of FFB and its chairman of the board.
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Need-to-Know Information about Clinical Trials

clinical trial patientWith about 15 clinical trials underway for inherited retinal diseases, and several more poised to begin in a few years, patients are eager to sign up for access to potential vision-saving therapies.
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Low-Hanging Fruit: Repurposing Drugs to Treat Retinal Diseases

Low-hanging fruitMany years ago, while picking apples with my uncle, he advised me to “pick the low-hanging fruit.” That way, he told me, you can fill your basket faster and not be as tired at the end of the day. I think the same can be said of drug repurposing—the process of evaluating a drug prescribed for one disease to see if it can safely and effectively treat another disease.
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