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BBP-818

Development stage: Preclinical
Lead developer: BridgeBio Pharma
Modality: Gene Therapies

Overview

BBP-818 is an investigational **AAV gene therapy** being developed by **BridgeBio Pharma** for **classic galactosemia** caused by severe deficiency of **galactose-1-phosphate uridylyltransferase**. The program is designed to deliver a functional **GALT** gene so that treated cells can produce GALT enzyme and restore the body's ability to metabolize galactose. Publicly available preclinical data indicate activity in a mouse model of classic galactosemia, including restoration of GALT enzyme activity in the brain after a single dose. No approved brand name has been identified.

Targets

GALT (Galactose-1-phosphate uridylyltransferase)

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