OUR PIPELINE
Liver-directed programs
A focused pipeline leveraging liver-directed genetic medicines to address significant unmet medical needs.
GENV-HEM (AAV8.FVa)
Hemophilia A, with or without FVIII inhibitors
Single-administration, liver-directed activated Factor V gene therapy.
Explore GENV-HEM ↗Preclinical
FDA INTERACT
Pre-INDCurrent stage
IND-Enabling
Phase 1/2
Phase 3
BLA
GENV-002
Infantile-onset and late-onset Pompe disease
In vivo gene-editing approach designed to establish sustained GAA production from the liver.
Explore GENV-002 ↗PreclinicalCurrent stage
FDA INTERACT
Pre-IND
IND-Enabling
Phase 1/2
Phase 3
BLA