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.
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