Fourth Gene Therapy Program Targets the Most Common Inherited Juvenile Macular Dystrophy

LEXINGTON, MA, USA and LONDON, UK I November 08, 2017 I Nightstar Therapeutics (NASDAQ:NITE), a clinical-stage gene therapy company developing treatments for rare inherited retinal diseases, today announced an expansion of its pipeline with an exclusive global license for a gene therapy program for the treatment of Stargardt disease from Oxford University Innovation.

The gene therapy program utilizes a novel technology developed by the University of Oxford. Preclinical proof-of-concept studies in the Abca4-/- murine model of Stargardt disease have demonstrated the expression, localization and function of the ABCA4 protein.

Stargardt disease is the most common form of inherited juvenile macular dystrophy with a prevalence of one in 10,000. This autosomal recessive disease is linked to mutations in the ABCA4 gene that are inherited from both parents of an affected individual. The progressive central vision loss associated with Stargardt disease is caused by the degeneration of photoreceptor cells in the central portion of the retina called the macula. Stargardt disease typically develops during childhood and adolescence, leading to blindness by the age of twenty. There are currently no treatment options for this disease.

“The licensing of this novel gene therapy program exemplifies our commitment to developing treatments for patients suffering from inherited retinal diseases that would otherwise lead to blindness,” said Dave Fellows, chief executive officer of Nightstar. “We plan to leverage our clinical experience with choroideremia and X-linked retinitis pigmentosa to accelerate the development of this exciting program for the unmet medical need in Stargardt disease.”

Financial terms of the agreement were not disclosed.

About Nightstar

Nightstar is a leading clinical-stage gene therapy company focused on developing and commercializing novel one-time treatments for patients suffering from rare inherited retinal diseases that would otherwise progress to blindness. Nightstar’s lead product candidate, NSR-REP1, is being developed as a treatment for patients with choroideremia, a rare, degenerative, genetic retinal disorder that has no current treatments and affects approximately one in every 50,000 people. Positive results from a Phase 1/2 trial of NSR-REP1 were published in The Lancet in 2014 and in The New England Journal of Medicine in 2016. Nightstar plans to commence a Phase 3 registrational trial of NSR-REP1 for choroideremia at sites in the United States, Europe and Canada in the first half of 2018. Nightstar’s second product candidate, NSR-RPGR, is currently being evaluated in a Phase 1/2 clinical trial for the treatment of patients with X-linked retinitis pigmentosa, an inherited X-linked recessive retinal disease that affects approximately one in every 40,000 people.

For more information about Nightstar or its clinical trials, please visit www.nightstartx.com.

About Oxford University Innovation

Oxford University Innovation (OUI) supports innovation activities across all divisions of the University of Oxford, managing technology transfer and consulting activities, and providing an innovation management service to clients around the world. OUI provides access to technology from Oxford researchers through intellectual property licensing, spinout company formation and material sales, and to academic expertise through its Consulting Services team. The New Venture Support & Funding team of OUI supports investors or donors with an interest in early-stage ventures, and manages the Oxford Angels Network. OUI’s Startup Incubator supports members and ex-members of the University who wish to start or grow entrepreneur-driven ventures that are not University spinouts.

SOURCE: Nightstar

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