Date: 2017-09-05
Type of information: Research agreement
Compound: small molecule therapeutics for NGLY1 deficiency
Company: Retrotope (USA - CA) National Institutes of Health’s National Center for Advancing Translational Sciences (NCATS) (USA) NGLY1.org (USA - UT)
Therapeutic area: Rare diseases - Genetic diseases - Neurodevelopmental diseases
Type agreement: research
Action mechanism:
Disease: NGLY1 deficiency
Details:
- • On September 5, 2017, Retrophin announced that it has entered into a three-way Cooperative Research and Development Agreement (CRADA) with the National Institutes of Health’s National Center for Advancing Translational Sciences (NCATS) and patient advocacy foundation NGLY1.org to collaborate on research efforts aimed at the identification of potential small molecule therapeutics for NGLY1 Deficiency. This rare and debilitating monogenic disease is believed to be caused by a deficiency in an enzyme called N-glycanase-1, which is encoded by the gene NGLY1. The condition has been characterized by symptoms such as developmental delays, seizures, complex hyperkinetic movement disorders, diminished reflexes and an inability to produce tears. There are no approved therapeutic options for NGLY1 deficiency, and current therapeutic strategies are limited to symptom management.
- The research collaboration will focus on the development of assays for small molecule high-throughput screening in an effort to better understand the biology of the disorder, and identify potential small molecules to be developed as a therapeutic for patients living with NGLY1 deficiency. Retrophin will seek to continue development of compounds through pre-clinical and clinical phases, with the ultimate goal of delivering a treatment option for the NGLY1 community.
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