Retrophin Announces Cooperative Research and Development Agreement with NCATS and to Identify Potential Small Molecule Therapeutics for NGLY1 Deficiency

SAN DIEGO, CA, USA I September 05, 2017 I Retrophin, Inc. (NASDAQ:RTRX) today 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 to collaborate on research efforts aimed at the identification of potential small molecule therapeutics for NGLY1 Deficiency. This rare and debilitating monogenic disease is characterized by developmental delays, seizures and an inability to produce tears; there are currently no approved therapeutic options for NGLY1 Deficiency.

“This collaboration with Retrophin and NCATS represents an exciting step forward and a major milestone for the NGLY1 community,” said Cristina Casanova Might, president of “We are grateful to both NCATS and Retrophin for working with our community to establish this partnership which enables us to accelerate drug discovery efforts, and gives hope to our patients and their families.”

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.

“This CRADA is a great example of how we can strive to make advancements through collaborative research on new therapeutic approaches for people living with rare diseases, such as NGLY1 deficiency,” said Bill Rote, PhD, senior vice president and head of research and development for Retrophin. “This synergy between industry, public research, and advocacy gives us a great opportunity to share resources with the common goal of making a difference in the lives of patients and families affected by NGLY1 deficiency, and Retrophin is proud to work alongside NCATS and in this endeavor.”

About NGLY1 Deficiency

N-glycanase deficiency, or NGLY1 deficiency, is an extremely rare genetic disorder 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.

About is a nonprofit 501(c)(3) patient support, advocacy and research organization for N-Glycanase (NGLY1) Deficiency.’s mission is to eliminate the challenges of NGLY1 through research, awareness and support. improves the lives of patients through family support, diagnostic access, clinical and patient education, conferences, and grants. For more information, visit

About Retrophin

Retrophin is a fully integrated biopharmaceutical company dedicated to delivering life-changing therapies to people living with rare diseases who have few, if any, treatment options. The Company's approach centers on its pipeline featuring late-stage assets targeting rare diseases with significant unmet medical needs, including fosmetpantotenate for pantothenate kinase-associated neurodegeneration (PKAN), a life-threatening neurological disorder that typically begins in early childhood, and sparsentan for focal segmental glomerulosclerosis (FSGS), a disorder characterized by progressive scarring of the kidney often leading to end-stage renal disease. Research exploring the potential of early-stage assets in additional rare diseases is also underway. Retrophin's R&D efforts are supported by revenues from the Company's commercial products Thiola®, Cholbam® and Chenodal®.

SOURCE: Retrophin

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