Partnering

NGLY1 Collaboration & Discovery Efforts
We are collaborating with the NIH National Center for Advancing Translational Sciences (NCATS) Therapeutics for Rare and Neglected Diseases (TRND) Biology Laboratory, and patient advocacy foundation NGLY1.org on a research plan to develop different assays for small molecule high-throughput screening. This is being done in both an effort to better understand the biology of the disease, as well as identify potential small molecules to be developed as a therapeutic for patients suffering from NGLY deficiency.

Learn more about this partnership.

Alagille Syndrome Collaboration & Discovery Efforts
We are participating in a three-way Cooperative Research and Development Agreement (CRADA) with NIH NCATS and patient advocacy foundation Alagille Syndrome Alliance (ALGSA) to collaborate on research efforts aimed at the identification and development of potentially novel therapeutic targets for Alagille syndrome (ALGS). ALGS is a rare and debilitating disease characterized by severe liver and cardiovascular abnormalities.

Learn more about this partnership.

Business Development
We seek opportunities to acquire late-stage clinical assets or commercial-ready medicines that fit within our strategy of addressing rare diseases with significant unmet medical needs.

We invite potential partners who share our goals to contact us at partnering@retrophin.com.