Nightstar Expands Pipeline with Novel Gene Therapy for the Treatment of Stargardt Disease
November 8, 2017
Fourth Gene Therapy Program Targets the Most Common Inherited Juvenile Macular Dystrophy
The gene therapy program utilizes a novel technology developed by the
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
Financial terms of the agreement were not disclosed.
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
For more information about Nightstar or its clinical trials, please visit www.nightstartx.com.
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to: statements about our plans to develop and commercialize our product candidates, our planned clinical trials for NSR-REP1 and NSR-RPGR, the clinical utility of our product candidates, the prevalence of patient populations for our targeted indications, and the utility of prior preclinical and clinical data in determining future clinical results. These forward-looking statements are based on management's current expectations of future events and are subject to a number of involve substantial known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to be materially different from the information expressed or implied by these forward-looking statements, including the risks and uncertainties set forth in the "Risk Factors" section of our prospectus filed pursuant to Rule 424(b)(4) under the U.S. Securities Act of 1933, as amended, on
Source: Nightstar Therapeutics