Achondroplasia is a rare genetic disorder of bone growth that causes short-limbed dwarfism. It occurs due to mutations in a single gene called the FGFR3.

The drug has demonstrated clinical POC in exudative age-related macular degeneration. ABOUT Achondroplasia Achondroplasia is disease in which a genetic mutation of the fibroblast growth factor ...

while being agnostic for the FGFR3 gatekeeper mutations. TYRA-300 is in development for the treatment of cancer in the SURF301 Phase 1/2 study and for achondroplasia in the BEACH301 Phase 2 study.