Drug intelligence / Profile preview

TAY-R1

Development stage
Preclinical
Lead developer
Tay Therapeutics
Modality
Small Molecules
Administration
Oral
01

Overview

TAY-R1 is an orally available small molecule being developed by Tay Therapeutics for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB). It is designed as a first-in-class translational read-through agent that targets nonsense mutations in the *COL7A1* gene, which affect approximately 30% of RDEB patients. By promoting read-through at premature stop codons, TAY-R1 enables the ribosome to complete translation and restore the production of full-length, functional Collagen VII. This protein is essential for forming anchoring fibrils that secure the epidermis to the dermis, and its restoration aims to address the underlying cause of skin fragility and blistering in RDEB patients. The program utilizes the Tay-Enable™ platform and is currently in the preclinical stage of development.

02

Targets

Eukaryotic ribosome translation termination machinery

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