USC-backed startup awarded grant for clinical trial of eye treatment

This is a milestone in the development of a stem cell patch to treat advanced dry age-related macular degeneration. (Image Credit: ©Giovanni Cancemi - Adobe.Stock.com)

A stem cell patch developed by a team of USC researchers for patients with macular degeneration will soon be tested in a phase 2b clinical trial, according to the Keck School of Medicine of USC.

In a news release, it was noted the latest milestone in the patch’s development was driven by a combined $21 million in support from the university, a state organization, and a nonprofit foundation.

In July, the California Institute for Regenerative Medicine (CIRM) awarded an estimated $12.4 million to the USC-supported startup Regenerative Patch Technologies (RPT) to test the safety and efficacy of the patch for treating advanced dry age-related macular degeneration (AMD), also known as geographic atrophy (GA).

According to the news release, the award from CIRM, a state institute that supports research to address unmet medical needs, is the latest round of funding for the startup. USC and The Marcus Foundation were also influential in the award.

The Keck School of Medicine of USC noted in its news release in order to qualify for CIRM funds, applicants must have a substantial amount of co-funding. USC Research and Innovation provided a $2 million promissory note to partially fulfill the co-funding requirement. The Marcus Foundation in Atlanta also played a critical role, providing USC researcher Mark Humayun and the university with more than $7 million in a peer-reviewed grant for this novel, transformational stem cell research program at USC that is aimed at curing blindness.

USC researcher Mark Humayun offered his thanks for the support from USC.

“I want to thank USC President Carol L. Folt for supporting this important research, as well as USC Research and Innovation for all the work they did to make this possible,” he said in the news release. “I would also like to thank The Marcus Foundation for their generous support after a thorough review process.”

According to the news release, USC Research and Innovation’s award was from a first-of-its-kind research program that backs USC faculty partnerships with small businesses to plan competitive funding applications.

“We are thrilled to support this critically important medical innovation by fostering the university’s ongoing technology transfer and commercialization efforts through faculty partnerships with small businesses,” Ishwar K. Puri, senior vice president for USC Research and Innovation, said in the news release.

A patch with promise

According to the news release, geographic atrophy affects more than 1 million Americans ages 40 and older, according to data from the Centers for Disease Control and Prevention. There currently is no therapy to improve vision in patients who have the condition.

According to researchers, work on the patch so far has provided positive results. It appears to restore the structure and function of the retina to improve vision. The phase 2b clinical trial will involve evaluating the efficacy of the implant in 24 patients who have suffered vision loss from geographic atrophy.

The disease, according to researchers, affects a vital group of cells: retinal pigment epithelium (RPE) cells. When healthy, these cells enable photoreceptor cells of the retina to detect light. With geographic atrophy, RPE cells degenerate over time. This can lead to photoreceptor loss, impaired vision and, for many, blindness, forcing some patients to lose their independence.

In an announcement of the award, Maria T. Millan, president and CEO of CIRM, pointed out the institute was pleased to continue to fund this groundbreaking stem cell therapy that has the potential to improve outcomes for the millions of people suffering from geographic atrophy.

“This investment is follow-on funding to CIRM’s previous support to develop this therapy,” Millan said in a statement. “It reflects our commitment to advancing cutting-edge science and underscores our dedication to addressing the unmet medical needs of those affected by degenerative diseases.”

Realizing the vision

Humayun and his fellow co-founders of Regenerative Patch Technologies — Dennis Clegg, PhD, professor in the department of molecular, cellular and developmental biology at the University of California, Santa Barbara; and the late David Hinton, MD, FARVO, professor of ophthalmology and pathology at USC — had developed the core technology for the stem cell-based subretinal implant.

The implant, named the “California Project to Cure Blindness-Retinal Pigment Epithelium 1” or CPCB-RPE1, is licensed by the USC Stevens center for Innovation exclusively to Regenerative Patch Technologies.

According to the news release, the phase 2b clinical trial at USC will be led by Sun Young Lee, MD, and Rodrigo Antonio Brant Fernandes,MD, both faculty members at the USC Roski Eye Institute.

The initial development of the implantable patch received its first support from the Alfred E. Mann Institute at USC as an incubator for new USC-related companies, the news release concluced.