Achondroplasia (ACH)

Condition Description

Achondroplasia is a genetic disorder that affects bone growth, leading to short stature and characteristic physical features. 

Achondroplasia is caused by a mutation in the fibroblast growth factor receptor 3 (FGFR3) gene. This mutation disrupts the process of cartilage formation and conversion into bone, resulting in abnormal bone growth. 

Status

Active and Recruiting Patients

Active Trials

  • SPONSOR: Tyra Biosciences, Inc.

    INDICATION: Achondroplasia

    PROTOCOL: TYR300-201

    PHASE: Phase 2

    DESCRIPTION: This is a Phase 1, multi-cohort trial studying TYRA-300-B01, a novel, potent fibroblast growth factor receptor (FGFR) 3-selective tyrosine kinase inhibitor, in healthy, adult participants. The purpose of this study is to evaluate the relative bioavailability of capsule and tablet formulations of TYRA-300-B01, and to evaluate the safety, tolerability, and food effect of TYRA-300-B01 tablets in healthy adult participants.

    STATUS: Recruiting

    RECRUITING PATIENTS: Yes
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Past Trials