| 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 |