Foundation Partners with Harrington Discovery Institute to Drive Clinical Research

July 16, 2015

The biggest challenge in developing a vision-saving treatment for people with inherited retinal diseases is moving it out of the laboratory and into a human study, otherwise known as a clinical trial. The process requires strong business-development skills to raise money, expertise to manufacture the therapy and in-depth knowledge of regulatory standards established by the U.S. Food and Drug Administration. It is also a risky proposition. Most projects fail before a therapy is ever tested in a person. Launching a clinical trial is so difficult, industry pundits often refer to it as “crossing the Valley of Death.”

To address these formidable challenges, the Foundation Fighting Blindness has teamed up with the Harrington Discovery Institute to launch a national initiative that provides the expertise to boost commercial viability of promising therapies. The partners plan to co-fund three projects every year with Gund-Harrington Scholarship Awards. The “Gund” refers to FFB co-founder Gordon Gund.

“While the Foundation funds outstanding researchers developing therapies with exciting potential, the scientist’s expertise is in the science,” says Brian Mansfield, Ph.D., deputy research officer at the Foundation. “Harrington brings invaluable know-how for translating research from the lab to the clinic. They place strong focus on the commercial end product, the design and formulation that will be most suitable for patients.”

Dr. Mansfield adds that the Harrington Discovery Institute collaboration provides drug-development experts with a strong track record of success at all phases of the translational research process. “There’s nothing like working with experts who know what it takes to succeed,” he says. “That really attracted us to the partnership.”

The first three investigators to be supported by the partnership are:

Albert La Spada, M.D., Ph.D., University of California, San Diego, who is developing a therapy to halt production of a toxic protein that leads to retinal degeneration for people with spinocerebellar ataxia type 7. In addition to blindness, the condition leads to loss of muscle control as well as difficulties with speech and swallowing. The emerging treatment uses oligonucleotides (small pieces of DNA) to “knock down” the harmful protein in the retina.

Konstantin Petrukhin, Ph.D., Columbia University, who is advancing a drug that stops the accumulation of toxic by-products in the retina, which cause vision loss in people with Stargardt disease, the most common form of juvenile macular degeneration.

Donald Zack, M.D., Ph.D., Johns Hopkins University, who is developing an innovative gene-editing tool known as CRISPR/Cas9 to treat autosomal dominant retinitis pigmentosa. Unlike conventional gene therapies—in which whole, therapeutic genes are delivered to the retina— CRSPR/Cas9 targets and repairs the defective region of the patients’ existing genes.

“The Gund-Harrington Awards are illustrative of the collaborative model the institute fosters,” says Mukesh K. Jain, M.D., scientific director, Harrington Discovery Institute. “We all want to accelerate the development of treatments and cures that tangibly impact lives. This impressive roster of Gund-Harrington Scholars was selected because their work shows great promise. Over the next three years, with oversight provided by the Harrington Discovery Institute’s Innovation Support Center panel of drug development experts, these projects will advance closer to the clinic and to becoming medicines that will improve sight.”