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. Teaching the body to make functional proteins can address a range of inherited diseases, including liver directed diseases such as hemophilia, lysosomal storage disorders and inherited ocular diseases. Our first product candidate incorporates three technologies consisting of an optimized codon, a strong liver promoter and a novel cleavage sequence. Learn more about our gene delivery platform below.

Gene Design

High Affinity Delivery

Proven Scientific and Clinical Development 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 cure for all types of hemophilia with or without inhibitors.

High Affinity Delivery

We have expertise in increasing target tissue affinity, increasing transduction and reducing required dosing through innovative design of promotors, linkers and vector selection. Vectors loaded with our optimized genes hold the potential to serve as medicines to treat inherited diseases.

Proven Scientific and Clinical Development Strategies

Our scientific team has over 20 years of experience with AAV gene therapy, from vector design to codon optimization. Our clinical development team has a track record of FDA approvals and works collaboratively with investigators and regulators to design trials with demanding endpoints to assess the potential of our gene therapy product candidates.

Liver Delivery / Hemophilia Program

DISCOVERY

Pre-IND

Phase 1/2

Phase 3

GENV-HEM - Hemophilia A and B with or without inhibitors

GENV-HEM is our investigational gene therapy for Hemophilia A, Hemophilia B or Factor VIII deficiency, with or without inhibitors. Hemophilia A is a serious and rare inherited hematologic disorder, characterized by mutations in the F8 gene, which lead to deficient blood coagulation and an increased risk of bleeding or hemorrhaging. GeneVentiv has discovered that instructing liver cells to produce additional Factor Va, can restore the clotting cascade. GENV-HEM is being developed as a novel, one-time infusion for hemophilia utilizing the AAV8 vector containing an optimized gene encoding for FVa.