Transformative therapy

Collaboration focuses on molecular target for unmet medical need

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SAN DIEGO—Retrophin Inc. is collaborating with the Grace Wilsey Foundation and the Warren Family Research Center for Drug Discovery and Development at the University of Notre Dame to develop a novel therapeutic for patients with NGLY1 deficiency, a rare genetic disorder. 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.
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 complex and devastating neuromuscular disease is characterized by a variety of symptoms, including global developmental delay, movement disorder, seizures and ocular abnormalities.
Under the terms of 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 will also provide funding and in-kind research support to help Retrophin advance this program.
Over the past 12 months, Retrophin. 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, has sought to initiate collaborations with patient advocacy organizations and academic institutions to advance drug development in areas of high unmet medical need, according to Dr. Alvin Shih, the company’s executive vice president and head of research and development. Retrophin and the leadership team of the Grace Wilsey Foundation began discussions to explore a potential discovery collaboration focused on the development of a novel molecular target that may be relevant to NGLY1 deficiency, which resulted in further dialogue on the structure of a potential collaboration.
At the same time, Retrophin was in active discussion with the Warren Family Research Center for Drug Discovery and Development at the University of Notre Dame to explore avenues to collaborate in drug development. NGLY1 had already been identified as a potential area of interest for Notre Dame, so it became clear that a three-way collaboration would be a great way of leveraging each party’s strengths and resources in an effort to find a cure for this devastating disease, Shih said.
NGLY1 deficiency is so nascent, having been described less than five years ago, that there is a limited understanding of the natural history and underlying disease processes, according to sources at Retrophin. These issues are being worked on by academia and the patient advocacy foundations, and will enable drug development to proceed more quickly.
Retrophin brings experience in early-stage drug discovery, including target validation, assay development and compound screening and optimization. The Retrophin R&D team includes experienced senior scientists who have worked on numerous successful drug development campaigns in the rare disease space.
Retrophin will apply its scientific and drug development capabilities to lead efforts towards the validation of a new molecular target. The company will be working hand-in-hand with the Grace Wilsey Foundation and the Warren Family Research Center for Drug Discovery and Development at the University of Notre Dame to advance the science as quickly as possible. “We hope the ultimate product of this collaboration is a transformative therapy for patients with NGLY1 deficiency,” said Shih.
“This collaboration with Retrophin will add momentum to our pursuit of a cure for NGLY1 deficiency,” commented Matt Wilsey, president and co-founder of the Grace Wilsey Foundation, who, along with his wife, founded the organization when their daughter Grace was diagnosed with NGLY1 deficiency. “Together, we’ll be able to make a significant impact on the lives of patients and families affected by this condition.”
“This collaboration exemplifies Retrophin’s commitment to working with patient advocacy groups and academic institutions to develop therapeutics for patients suffering from rare diseases,” added Shih. “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.”




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