Vision-Saving Research Highlighted at Foundation’s Orange County Symposium

March 7, 2014 – Continuing its role of fostering collaboration and knowledge-sharing to accelerate the clinical development of retinal disease therapies, the Foundation Fighting Blindness Clinical Research Institute (FFB CRI) co-hosted its Sixth Annual Orange County Innovation Symposium on February 27. This year’s event took place at the Gavin Herbert Eye Institute (GHEI), University of California, Irvine (UCI), which also served as co-host. William Link, Ph.D., managing director, Versant Ventures, and Roger F. Steinert, M.D., Irving H. Leopold Professor and chair director, GHEI, co-chaired the symposium.

The day-long event featured more than two dozen presentations and panel discussions from leaders in retinal research, ocular therapy development, the pharmaceutical industry and the venture capital community.

“It is critical that we inform the commercial sector about the innovative research funded by the Foundation and the potential it has for saving vision and changing lives,” explains Patricia Zilliox, Ph.D., chief drug development officer, FFB CRI. “Our goal is to attract investments and build relationships to move promising therapies into and through the costly clinical development process and out to the people who need them.”

“The symposium was notable for both the breadth and depth of the presentations,” adds Dr. Steinert. “Attendees ranging from bench scientists and clinical researchers to leaders in the vision industry stimulated each other with fresh insights into where we are and where we can go together. We all have a common goal of eradicating blindness and doing it sooner rather than later. The symposium was a key step in that process.”

The symposium featured a discussion of one of the most successful collaborations ever in the inherited retinal disease sector. The story began in the late 1990s, when FFB began funding cutting-edge gene therapy research in the lab. Over the next decade, that work led to early-stage clinical development of retinal gene therapies by Oxford BioMedica, an emerging biopharmaceutical company in the United Kingdom.

On February 19, Sanofi Fovea, a global leader in ocular therapy development, completed its acquisition of StarGen™ and UshStat® gene-therapy licenses from Oxford BioMedica, and is now committed to moving the Stargardt disease and Usher syndrome 1B treatments through the clinical trial process and out to the marketplace. Sanofi Fovea is also continuing its partnership with Oxford BioMedica in the clinical development of RetinoStat®, a gene therapy for wet age-related macular degeneration.

Soraya Bekkali, M.D., vice president, head of the ophthalmology unit at Sanofi Fovea, spoke about her company’s commitment to treatments for rare ocular diseases. She also acknowledged the challenge of developing therapies for populations for which there is a lack of disease knowledge, few patients for clinical trials and a limited regulatory track record. “We are very much willing to take on these challenges, because there are excellent opportunities to help patients, especially through partnerships and collaborations,” she said.

Retinal research highlights of the symposium also included presentations on emerging stem cell treatments targeting retinitis pigmentosa (RP) being developed by Michael Young, Ph.D., Harvard Medical School, and Henry Klassen, Ph.D., GHEI UCI.

Funded in part by FFB CRI, Dr. Young is conducting preclinical research for ReNeuron, a stem cell developer in the United Kingdom, which is planning a human study of its therapy for replacing lost photoreceptors in late 2014.

Dr. Klassen is working on cell manufacturing, toxicology research and clinical trial design in preparation for a human stem cell study he hopes to launch in about two years. A former recipient of an FFB Career Development Award, Dr. Klassen received a $17.3 million award in 2012 from the California Institute for Regenerative Medicine to advance the therapy.

On the pharmaceutical front, David Saperstein, M.D., a consultant to QLT, Inc., presented impressive data on the company’s Phase Ib clinical trial of retinoid replacement therapy for people with RP and Leber congenital amaurosis (LCA) caused by mutations in the genes LRAT or RPE65. A single, seven-day course of the oral treatment — which replaces11-cis-retinal, a protein essential to the biochemical process that makes vision possible — improved visual acuity and/or visual field in 79 percent of patients with LCA and 83 percent of those with RP.

In a press release issued a few hours before the symposium, QLT reported preliminary data from a re-treatment study showing that the therapy continued to be effective and well-tolerated in patients receiving as many as three additional seven-day regimens. QLT is now planning a Phase III clinical trial.

In addition to highlighting retinal disease therapies, researchers and company representatives also presented developments in overcoming glaucoma, including state-of-the art ocular stents and microsurgeries to release the fluid that causes increases in vision-damaging intraocular pressure.

Cornea-based solutions for presbyopia — a condition of aging characterized by the diminished ability to focus due to stiffening of the lens — were also presented.

Imaging researchers with expertise in adaptive optics and optical coherence tomography delivered updates on emerging technologies that are helping physicians better diagnose patients with ocular conditions and assessing therapies in human studies.

The symposium’s closing session featured remarks and discussion from an ophthalmic industry investor panel. It underscored the growing market opportunities in eye and vision care because of the world’s aging population, advancements in therapeutic and diagnostic technologies and the willingness of ophthalmologists to innovate in treating patients.

Eugene DeJuan, Jr., M.D., a founder of ForSight Labs and nine other ophthalmology companies, including the firm that began development of the first Argus retinal prosthesis, said that vision care will always be a high-demand, global market because of the critical role vision plays in our lives.

“I look at this from a physician’s standpoint. I am always thinking about the impact of what I am doing,” he said. “And the impact of reduced vision on quality of life is huge for everyone.”

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