Preclinical data of Ocugen's OCU400 genetic modifier published in Nature Gene Therapy

Reviewed by Kate Anderton, B.Sc. (Editor)Mar 3 2020

Ocugen, Inc. (NASDAQ: OCGN), a clinical-stage company focused on discovering, developing and commercializing transformative therapies to treat rare and underserved ophthalmic diseases, announced today the publication in Nature Gene Therapy of preclinical data of nuclear hormone receptor gene NR2E3 as a genetic modifier and therapeutic agent to treat multiple retinal degenerative diseases.

OCU400 (NR2E3-AAV) has received two orphan drug designations targeting two distinct inherited retinal diseases (IRDs): NR2E3 mutation-associated retinal diseases and CEP290 mutation-associated retinal diseases.

The publication details efficacy results in five unique mouse models of retinitis pigmentosa (RP) that underwent administration of NR2E3-AAV by subretinal injection.

The five RP models tested were rd1 (PDE6 β associated RP), Rho-/- and RhoP23H (both Rhodopsin associated RP), rd16 (Leber Congenital Amaurosis) and rd7 (Enhanced S-cone Syndrome). The study demonstrates the potency of a novel modifier gene therapy to elicit broadspectrum therapeutic benefits in early and intermediate stages of RP.

Please refer to Nature Gene Therapy's online publication for additional results from this study.

This represents an important milestone for the development of this therapy. I am impressed by the protection that was elicited in multiple animal models of degeneration caused by different mutations. A treatment for inherited retinal degenerations that is mutation independent would have wide reaching implications."

Mark Pennesi, M.D., Ph.D., Associate Professor of Ophthalmology, Oregon Health & Science University School of Medicine

Pennesi is also the Division Chief of the Ophthalmic Genetics Service at the OHSU Casey Eye Institute.

"Dr. Haider and her vision research team have successfully demonstrated proof of principle in their elegant study by rescuing 5 animal models of RP by resetting homeostasis. This is the foundation work for the development of the first broad spectrum therapy for inherited retinal degeneration diseases and is a game changer for rescue even after disease onset," said Cheryl Mae Craft, Ph.D., Professor of Ophthalmology, USC Keck School of Medicine at USC Roski Eye Institute Los Angeles, CA.

"One of the biggest advantages of our modifier gene therapy platform is that it has the potential to eliminate the need for individual gene replacement and gene editing strategies and may revolutionize gene therapy treatments for eye diseases.

"Inherited retinal degenerations such as RP affect over 1.5 million people worldwide. Over 150 gene mutations have been associated with RP and this number represents only 60% of the RP population. The remaining 40% of RP patients cannot be genetically diagnosed, making it difficult to develop individual treatments.

"Our modifier gene therapy has potential to eliminate the need for developing more than 150 individual products and provide one treatment option for all RP patients," said Rasappa Arumugham, Ph.D., Ocugen's Chief Scientific Officer. "We are completing preclinical studies for OCU400 and anticipate commencing a Phase 1/2a clinical trial in patients in 2021."