Drug Profile

BBP 812

Alternative Names: BBP-812

Latest Information Update: 19 Nov 2024

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At a glance

Originator University of Massachusetts Amherst
Developer Aspa Therapeutics
Class Gene therapies
Mechanism of Action Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Canavan disease
New Molecular Entity No

Highest Development Phases

Phase I/II Canavan disease

Most Recent Events

12 Nov 2024 Updated efficacy data from the phase I/II CANaspire trial in Canavan disease released by BridgeBio Pharma
24 Oct 2024 Updated efficacy, adverse event and pharmacodynamics from the phase I/II CANaspire trial in Canavan disease released by BridgeBio Pharma
10 Sep 2024 BBP 812 receives Regenerative Medicine Advanced Therapy (RMAT) status for Canavan disease in USA

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