Peboctocogene camaparvovec - Bayer
Alternative Names: AAV-mediated Factor VIII gene therapy; BAY-2599023; DTX-201Latest Information Update: 23 Apr 2023
At a glance
- Originator Dimension Therapeutics
- Class Antihaemorrhagics; Gene therapies
- Mechanism of Action Gene transference
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
- Available For Licensing Yes
Highest Development Phases
- Phase I/II Haemophilia A
Most Recent Events
- 31 Dec 2022 Ultragenyx Pharmaceutical has patent protection for peboctocogene camaparvovec
- 11 Dec 2021 Updated adverse events data from a phase-I/II clinical trial in Haemophilia A presented at the 63rd American Society of Hematology Annual Meeting and Exposition (ASH-Hem-2021)
- 05 Mar 2021 Phase-I/II development is still ongoing in Haemophilia A in Bulgaria, Germany, France, Netherlands, United Kingdom and USA (IV) (NCT03588299)