NEI Pursues Innovation on Multiple Tracks

Since a congressional mandate required the National Institutes of Health to set aside 3% of its total funding for small business grants, the National Eye Institute has been funding approximately 70 small companies with about $20 million a year in grants, Mala Dutta, PhD, lead, office of translational research at the National Eye Institute told attendees at OIS@AAO 2016.

Speaking during the session “Ophthalmic Innovation 2016: A view from the NEI,” Dr. Dutta explained that the NEI has multiple programs for doling out its $700 million annual research budget, the biggest part of which is grants to individual investigators. She discussed two programs that have resulted in innovative translational breakthroughs.

The Small-Business Initiative focuses on development of new pharmaceuticals and low-vision devices, Dr. Dutta said. Among recent success stories of the small-business program are:

• VisionQuest Biomedical LLC, developer of a telemedicine system for real-time, large-scale eye screening at a sustainable cost.
• Photoswitch Biosciences Inc., developer of a small-molecule therapeutic photo-switch channel to restore vision in degenerative retinal disease
• Parion Sciences Inc., developer of an epithelial sodium channel antagonist that regulates salt and water transport as a treatment for dry eye.

The Translational Research Program involves multidisciplinary and collaborative research projects, and provides renewable grants totaling of $1.5 million a year for five years. “The current portfolio includes small-molecule photo switches for outer retinal blindness, gene therapy for a number of ocular disorders, and microRNAs for potent anti-angiogenesis,” said Dr. Dutta.

Among the success stories in this program are grants to Emory University for developing nanoparticle preparation of microneedles for injecting drugs into the suprachoroidal space. This program has since led to the formation of Clearside Biomedical, which has taken this platform into clinical trials. Grants to the University of Miami for gene therapy treatment of Leber’s hereditary optic neuropathy have led to a Phase I clinical trial.

Also promising is the NEI intramural program to develop cell and gene therapies. Dr. Dutta said the leading stem cell program for neurotransplantation of stem cells involves using induced pluripotent stem cells to create a patch of the retinal pigment epithelium as a treatment for age-related macular degeneration (AMD). “They are on track for human trials in two to three years,” she said. Other cell and gene programs involved a Phase I/IIa trial of gene therapy for x-linked retinoschisis and animal studies of gene therapy for x-linked retinitis pigmentosa.

In addition, NEI has committed $50 million this year to support clinical trials outside the institute, along the lines of the Diabetic Retinopathy Clinical Research Network, and is continuing a program to develop young investigators, Dr. Dutta said.

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