PriZm Therapeutics
PriZm Therapeutics is a late-stage clinical biotech company focused on developing therapies for ultra-rare pediatric orphan diseases. Driven by science, knowledge, and compassion, the company aims to provide first-in-class solutions for patients with conditions like AHDS and MCT8 deficiency. Their lead program, SRW101, is licensed from the University of Miami and is in development as a treatment for these rare disorders.
Industries
biotechnology
therapeutics
Products
Small-molecule thyroid hormone analogue for MCT8 deficiency (oral formulation)
A synthetic thyroid-hormone analogue developed as an oral formulation intended to partially restore central nervous system thyroid hormone activity in patients with MCT8 deficiency; previously administered under compassionate use with reported beneficial results and advanced toward a planned Phase III registration trial.
Small-molecule thyroid hormone analogue for MCT8 deficiency (oral formulation)
A synthetic thyroid-hormone analogue developed as an oral formulation intended to partially restore central nervous system thyroid hormone activity in patients with MCT8 deficiency; previously administered under compassionate use with reported beneficial results and advanced toward a planned Phase III registration trial.
Expertise Areas
- Clinical trial management
- Rare pediatric disease drug development
- Translational medicine
- Preclinical pharmacology and mechanism-of-action studies
Key Technologies
- Small-molecule therapeutics
- Oral formulation development
- Blood–brain barrier transport assays
- Animal disease models (Mct8-deficient mice)