Therapeutic Programs
GeneVentiv is transforming genes into medicines for inherited diseases.
High Affinity Delivery
We utilize AAV vectors with high target tissue affinity to deliver optimized transgenes that hold the potential to serve as medicines to treat inherited diseases.
Scientific and Regulatory Strategies
Our Scientific Team has over 28 years of experience with AAV gene therapy, from vector design to transgene optimization. Our Regulatory Team has a track record of FDA approvals and has worked on over 100 programs ranging from IND to NDA and BLA. Together our Scientific and Regulatory Teams work collaboratively with our investigators conducting contract and sponsored research to assess the efficacy and safety of our gene therapy product candidates.
Liver Delivery Programs
Pre-Clinical
IND Approval
Phase I/II
Phase III
GENV-HEM (AAV8.FVa) – Hemophilia A with or without inhibitors
Pre-Clinical
IND Approval
Phase I/II
Phase III
GENV-002 (AAV.GAA and AAV.Cas9) - Infantile Onset (IOPD) and Late Onset (LOPD) Pompe Disease
GENV-002, a first-in-class gene editing therapy that stably integrates a functional GAA transgene offering lifelong enzyme production for both Infantile Onset (IOPD) and Late Onset (LOPD) Pompe disease, something that other gene therapies are unable to achieve. With newborn screening for IOPD and LOPD, GENV-002 can be administered to all patients as infants, a decade or more in advance of non-genome editing AAV competitors. GENV-002 will be able to treat 120,000 developed world patients with IOPD and LOPD.