Therapeutic Programs

GeneVentiv is transforming genes into medicines for inherited diseases.

Our Gene Therapy Platform and Product Candidates

GeneVentiv is developing gene therapy product candidates to deliver healthy genes to a variety of cell targets within the body.  Our first product candidate uses a highly liver-tropic AAV vector containing an optimized transgene with regulatory elements for enhanced protein expression. Learn more about our gene delivery platform below.

Gene Design

High Affinity Delivery

Scientific and Regulatory Strategies

Gene Design

We have expertise in engineering genes to treat specific inherited diseases. Our first, patent pending, engineered gene is designed to be a universal treatment for all types of hemophilia with or without inhibitors.

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


IND Approval

Phase I/II

Phase III

GENV-HEM (AAV8.FVa) - Hemophilia A and B with or without inhibitors

GENV-HEM (AAV8.FVa) directs expression of human FVa in its native environment.  GENV-HEM is the first, single infusion, universal AAV-based gene therapy for all types of hemophilia and is the first to treat inhibitor patients.  There are 50,000 inhibitor patients in the developed world. GENV-HEM has demonstrated therapeutic efficacy and safety in pre-clinical studies.