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Scientists Receive $25 Million to Develop a Vision-Restoring System that Connects to the Brain

Labels in English are (clockwise): Intelligent neuromorphic camera/LED; Matrix for optical stimulation of neurons/electronic; Implant for data processing/external; Antenna for energy transfer and wireless communication; External module for visual information transfer

Labels in English are (clockwise from upper left): Intelligent neuromorphic camera/LED; Matrix for optical stimulation of neurons/electronic; Implant for data processing/external; Antenna for energy transfer and wireless communication; External module for visual information transfer

While researchers around the world are developing numerous drugs and biological therapies for ocular diseases and injuries, an international scientific team coordinated in France is developing an innovative approach to bring back eyesight to the blind that bypasses the eye entirely. Their high-tech, vision-restoring system interfaces with the visual cortex, the back of the brain where visual input is processed to create the images we see.

The CorticalSight Consortium, led by Jose-Alain Sahel, MD, at the Institut de la Vision and the University of Pittsburgh, and Serge Picaud, PhD, at the Institut de la Vision, was recently awarded a contract of up to $24.9 million by the Defense Advanced Research Projects Agency (DARPA) for development of their system, which combines optogenetics and advanced image-capturing and -processing technologies. A major advantage of the approach is it is designed to provide vision restoration for people regardless of their eye disease or condition.
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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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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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SparingVision Formed to Advance Sight-Saving Protein for RP

L to R: Florence Allouche Ghrenassia, PharmD, President, SparingVision; Frédérique Vidal, French Minister of Higher Education, Research and Innovation; José-Alain Sahel, MD, Co-Founder, SparingVision and Fondation Voir & Entendre; David Brint, and Chairman, Foundation Fighting Blindness; and Laure Reinhardt, Deputy CEO, Bpifrance

L to R: Florence Allouche Ghrenassia, PharmD, President, SparingVision; Frédérique Vidal, French Minister of Higher Education, Research and Innovation; José-Alain Sahel, MD, Co-Founder, SparingVision and Fondation Voir & Entendre; David Brint, Chairman, Foundation Fighting Blindness; and Laure Reinhardt, Deputy CEO, Bpifrance

The development of a vision-saving treatment for people with retinitis pigmentosa (RP) is getting a major boost thanks to the formation of the French biotech SparingVision to move it into a clinical trial and out to the international marketplace.

A spin-off of the Institut de la Vision, SparingVision was established to clinically develop and commercialize a protein known as rod-derived cone-viability factor (RdCVF). The emerging therapy performed well in several previous lab studies funded by the Foundation Fighting Blindness. SparingVision’s goal is to launch a clinical trial for the protein in 2019.
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Researchers Find Mutation as Frequent Cause of RP in American Hispanics

A Foundation-funded research collaboration identified a mutation in the gene SAG as a frequent cause of autosomal dominant retinitis pigmentosa (adRP) in the American Hispanic population. Eight of the 22 Hispanic families with adRP in their whole-exome-sequencing study had the mutation. The discovery can help genetic experts diagnose more patients with adRP, and it gives researchers a target for developing potential therapies. Results of the SAG study were published in the journal Investigative Ophthalmology and Visual Science (IOVS).
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Valproic Acid’s Effect Too Small in One-Year Clinical Trial

However, researchers identify a potentially powerful endpoint for evaluating emerging therapies in future studies.

Results from a clinical trial sponsored by the Foundation Fighting Blindness Clinical Research Institute (FFB-CRI) indicate that valproic acid, a drug approved by the U.S. Food & Drug Administration for seizure disorders, did not sufficiently preserve vision in people with autosomal dominant retinitis pigmentosa (adRP). FFB-CRI launched the 90-person study in 2010, because previous lab research, and a published clinical report involving a few patients, had suggested the drug might slow vision loss in people with adRP.
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Forty-Four High-Impact Retinal-Research Efforts Highlighted at FFB-Casey Innovation Summit

For anyone wanting to know about the latest clinically focused research for inherited retinal diseases (IRDs), the Innovation Summit for Retinal Cell and Gene Therapy was the place to be.

In its fourth year, the meeting is becoming the world’s most comprehensive overview of the promising research underway for emerging IRD treatments. Hosted by the Foundation Fighting Blindness and Casey Eye Institute (Oregon Health & Science University), the Innovation Summit featured 44 information-packed presentations from research and industry leaders. Summit co-hosts were Casey’s Mark Pennesi, MD, PhD, and Trevor McGill, PhD, and Patricia Zilliox, PhD, chief drug development officer of FFB’s Clinical Research Institute. More than 250 researchers and industry leaders were in attendance. Spark Therapeutics was a sponsor of the event.
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Clinical Trial Authorized in the U.S. for Emerging LCA 10 Therapy

ProQR, a biotechnology company in the Netherlands, has received authorization from the U.S. Food and Drug Administration to start a Phase I/II clinical trial for its therapy known as QR-110, which is being developed for Leber congenital amaurosis type 10 (LCA 10). The genetic retinal condition causes severe vision loss in children. QR-110 targets the specific mutation p.Cys998X in the CEP290 gene, also known as c.2991+1655A>G mutation. It is estimated that it affects about 2,000 people in the Western world.

QR-110 was a featured topic at the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) in Baltimore, May 7-11. About 12,000 eye researchers and industry professionals attended the event.
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FFB Funding Helps Retinal Genetics Lab Secure $2 Million Investment

This is a great story of how the Foundation Fighting Blindness (FFB) provided timely funding of $155,000 to help my lab at the University of California, San Diego (UCSD), leverage a $2 million retinal-gene discovery project.

What is very rewarding for me is that FFB’s support helped us find the retinal-disease gene mutation in 33 families, who were otherwise left without a clear diagnosis. Now these families are better able to understand their prognosis and which clinical trials and future therapies may be most relevant to them.
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