PriZm Therapeutics, a late-stage clinical biotech company with a focus on rare pediatric orphan diseases and BIO’s Seed-Stage Start-Up Stadium winner was featured nationally and Internationally in BIO News.
PriZm Therapeutics co-founder, Kiran Avancha, PhD, presents at BIO’s Start-Up Stadium in June, 2023. (Image Credit: BIO News)
AZBio Board Member Kiran Avancha, PhD andPriZm Tx cofounders, Samuel Refetoff, M.D. and Roy Weiss, M.D., Ph.D., identified SRW101 as a potential treatment for MCT8 deficiency.
The company launched in 2020 with its lead asset, SRW101.
As a synthetic T3 analogue, SRW101 is a novel small molecule, also known as diiodothyropropionic acid (DIPTA). Its mechanism of action is similar to that of triiodothyronine (T3).
Monocarboxylate transporter 8 (MCT8) deficiency is an X-linked genetic disorder resulting from a defective gene (SLC16A2). The consequence of this defect is a deficiency in this transporter. Without the transporter, thyroid hormone (TH), more specifically, triiodothyronine (T3) is unavailable in the brain during crucial periods of neonatal development.