Pipeline Report

Updated February 1st, 2026

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Showing results 4,121–4,128 of 4,419
Manufacturer ▼ Drug Name Indication Stage Status Route of Administration Drug Class
Tvardi Therapeutics Inc. TTI-101 - (REVERT IPF) Idiopathic pulmonary fibrosis (IPF) Phase 2 Data Released oral Respiratory
Tyra Biosciences Inc. TYRA-300 - (BEACH301) Achondroplasia Phase 2 Enrollment Initiation Oral Genetic Disorder
Tyra Biosciences Inc. TYRA-300 - (SURF302) Non-Muscle Invasive Bladder Cancer Phase 2 Ongoing oral Oncology
Ultragenyx Pharmaceutical Inc. DTX401 Glycogen Storage Disease Type Ia (GSDIa) PDUFA Ongoing Intravenous Genetic Disorder
Ultragenyx Pharmaceutical Inc. UX111 - (ABO-102) Sanfilippo syndrome type A (MPS IIIA) BLA Filing Ongoing Intravenous Genetic Disorder
Ultragenyx Pharmaceutical Inc. GTX-102 - (Aspire) Angelman Syndrome Phase 3 Ongoing Intrathecal Genetic Disorder
Ultragenyx Pharmaceutical Inc. GTX-102 - (Aspire) Angelman Syndrome Phase 3 Ongoing Intrathecal Genetic Disorder
Ultragenyx Pharmaceutical Inc. DTX301 - (Enh3ance) Ornithine Transcarbamylase (OTC) Deficiency Phase 3 Ongoing Intravenous Genetic Disorder