RE-034 & Discovery Programs
We have conducted early-stage research for RE-034, a novel synthetic analog of a hormone produced by the adrenal gland, known as ACTH.1, 2 This hormone regulates levels of cortisol that contribute to chemical homeostasis in the body.2
We are exploring strategic options to further develop RE-034, including external partnerships and out-licensing opportunities. Learn more about partnering with us.
Early-stage research exploring assets across several rare diseases is ongoing.
Among these therapeutic targets is NGLY1 deficiency, a disorder characterized by a variety of debilitating and isolating symptoms, including global developmental delay, movement disorders, seizures, ocular abnormalities, chronic constipation, and life-threatening liver disease.3 We are working with the Grace Science Foundation and the Warren Family Research Center for Drug Discovery and Development at the University of Notre Dame on the development of a novel therapeutic for patients with NGLY1 deficiency. This partnership exemplifies our aspirations to work with patient advocates, academia and independent researchers to advance research supporting new solutions that may transform the lives of patients and shape the future of our company. Learn more about this partnership.
- Data on file. San Diego, CA: Retrophin, Inc.; July 2016.
- Mayo Clinic. Adrenocorticotropic Hormone (ACTH), Plasma. Available at: http://www.mayomedicallaboratories.com/test-catalog/Clinical+and+Interpretive/8411. Last accessed July 28, 2016.
- Grace Science Foundation. What is NGLY1 Deficiency? Available at: https://gracescience.org/our-work/#section. Last accessed July 28, 2016.