WM-1229

WM-1229 is a small molecule readthrough inducer being developed by Wasserman Medic, in collaboration with Almirall, for the treatment of rare dermatological conditions, specifically Recessive Dystrophic Epidermolysis Bullosa (RDEB) and Junctional Epidermolysis Bullosa (JEB). These conditions are often caused by nonsense mutations that introduce premature termination codons (PTCs) in genes responsible for structural skin proteins, such as COL7A1 (for RDEB) or laminin genes (for JEB). WM-1229 works by binding to the ribosome and reducing the stringency of the decoding process, allowing the translation machinery to bypass the PTC and incorporate a near-cognate amino acid. This results in the production of full-length, functional proteins that are essential for anchoring the layers of the skin. The drug is formulated as a topical ointment and is currently in Phase 2 clinical development.

Other names

Readthrough inducer (Wasserman Medic)