SAN DIEGO, Nov. 05, 2015 (GLOBE NEWSWIRE) -- Retrophin, Inc. (NASDAQ:RTRX) today announced a research collaboration with the Grace Wilsey Foundation and the Warren Family Research Center for Drug Discovery and Development at the University of Notre Dame surrounding the development of a novel therapeutic for patients with NGLY1 deficiency, a rare genetic disorder. NGLY1 deficiency is believed to be caused by a deficiency in an enzyme called N-glycanase-1, which is encoded by the gene NGLY1. The condition is characterized by a variety of symptoms, including global developmental delay, movement disorder, seizures, and ocular abnormalities.
Under this collaboration, the Grace Wilsey Foundation will provide support and funding to Retrophin to enable discovery efforts that aim to validate and address a new molecular target that may be relevant to NGLY1 deficiency. In addition, the Warren Family Research Center for Drug Discovery and Development at the University of Notre Dame will also provide funding and in-kind research support to help Retrophin advance this program.
"This collaboration with Retrophin will add momentum to our pursuit of a cure for NGLY1 deficiency," said Matt Wilsey, President and co-founder of the Grace Wilsey Foundation. "Retrophin brings a
wealth of scientific and drug development expertise to our work, and together we'll be able to make a significant impact on the lives of patients and families affected by this condition."
The initial research effort will focus on the hypothesis that a specific novel molecular target may compensate for the lack of NGLY1 in affected cells. Retrophin will apply its scientific and drug development capabilities to lead efforts towards the validation of this proposed target.
"This collaboration exemplifies Retrophin's commitment to working with patient advocacy groups and academic institutions to develop therapeutics for patients suffering from rare diseases," said Alvin Shih, M.D., Executive Vice President and Head of Research
and Development at Retrophin. "We appreciate the support from the Grace Wilsey Foundation, which is leading the charge to find a cure for NGLY1 deficiency. Our team is also excited to begin collaborating with the University of Notre Dame, an emerging leader in the rare disease research community."
About the Grace Wilsey Foundation
Kristen and Matt Wilsey founded the Grace Wilsey Foundation when their daughter Grace was diagnosed with a rare genetic disease called NGLY1 deficiency. This complex and devastating neuromuscular disease affects less than 50 known patients worldwide. Very few resources existed devoted to NGLY1 deficiency, either in terms of funding, public awareness, active research, or existing
knowledge. Undeterred, the Wilseys decided to apply their passion and entrepreneurial spirits to finding a cure for Grace and other NGLY1 deficiency patients. Over the last 6 years, the foundation has assembled a world-class team of over 60 scientists across 20 teams in the United States, Japan, and Germany. They work not only to understand the disease and develop a cure, but also to drive innovations in the world of genetic research in general. Their work has been featured by CNN.com, the San Francisco Chronicle, Der Spiegel, and The New Yorker. To learn more, please visit www.gracewilsey.org.
About the Warren Family Research Center for Drug Discovery and Development at the University of Notre Dame
The University of Notre Dame is a private research university inspired by its Catholic character to be a powerful force for good in the world. Notre Dame's Warren Family Research Center for Drug Discovery and Development is a collaborative research program, well aligned with the University's overarching Catholic mission, which seeks to enhance all stages of the process from basic discovery, to applied and translational research, and directs its efforts towards the
identification of new therapeutic leads for the treatment of unmet clinical needs in cancer, infectious diseases, and rare diseases.
Retrophin is a pharmaceutical company focused on the development, acquisition and commercialization of drugs for the treatment of serious, catastrophic or rare diseases for which there are currently no viable options for patients. The Company's approved products include Chenodal®, Cholbam®, and Thiola®, and its pipeline includes compounds for several catastrophic diseases, including focal segmental glomerulosclerosis (FSGS), pantothenate kinase-associated neurodegeneration (PKAN), nephrotic syndrome and others. For
additional information, please visit www.retrophin.com.
This press release contains "forward-looking statements" as that term is defined in the Private Securities Litigation Reform Act of 1995. In addition, expressions of our strategies, intentions or plans are also forward-looking statements. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statement can be guaranteed. Among the factors
that could cause actual results to differ materially from those indicated in the forward-looking statements are risks and uncertainties associated with the Company's business and finances in general, as well as risks and uncertainties associated with the Company's research pre-clinical and clinical stage pipeline. You are cautioned not to place undue reliance on these forward-looking statements as there are important factors that could cause actual results to differ materially from those in forward-looking statements, many of which are beyond our control. The Company undertakes no obligation to publicly update forward-looking statement, whether as a result of new information, future events, or otherwise. Investors are referred to the full discussion of risks and uncertainties as included in the Company's filings with the Securities and Exchange Commission.
Chris Cline, CFA
Director, Investor Relations
Source: Retrophin, Inc.
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