GeneVentiv Therapeutics, Inc.
GeneVentiv Therapeutics is a pre-clinical gene therapy company focused on developing transformative gene therapies for blood disorders, especially hemophilia. Their mission is to discover, develop, and deliver innovative treatments that can potentially cure all types of hemophilia, including those with inhibitors, through universal gene therapy solutions. They aim to improve lives by providing long-lasting, effective treatments and are actively advancing their programs towards clinical trials and regulatory approval.
Industries
Nr. of Employees
small (1-50)
GeneVentiv Therapeutics, Inc.
Products
GENV-HEM
A preclinical, single-infusion, AAV-based liver-directed gene therapy that delivers a transgene encoding an activated Factor V (FVa) variant to restore hemostasis in hemophilia A or B patients, designed to be effective with or without pre-existing neutralizing antibodies to missing clotting factors.
GENV-001 (hemophilia arthropathy program)
A preclinical gene therapy program developed for direct joint injection to prevent or treat hemophilia-associated joint disease (hemarthropathy) by local delivery to affected joints.
GENV-002 (gene editing program for Pompe disease)
A preclinical gene editing approach intended to stably integrate a functional GAA transgene to provide lifelong enzyme production for infantile and late-onset Pompe disease.
GENV-HEM
A preclinical, single-infusion, AAV-based liver-directed gene therapy that delivers a transgene encoding an activated Factor V (FVa) variant to restore hemostasis in hemophilia A or B patients, designed to be effective with or without pre-existing neutralizing antibodies to missing clotting factors.
GENV-001 (hemophilia arthropathy program)
A preclinical gene therapy program developed for direct joint injection to prevent or treat hemophilia-associated joint disease (hemarthropathy) by local delivery to affected joints.
GENV-002 (gene editing program for Pompe disease)
A preclinical gene editing approach intended to stably integrate a functional GAA transgene to provide lifelong enzyme production for infantile and late-onset Pompe disease.
Services
End-to-end preclinical development of gene therapy programs including proof-of-concept efficacy studies, pharmacology, biodistribution and safety testing to support IND-enabling packages.
Coordination with CDMO partners to advance AAV process development, GLP/GMP production and transfer of clinical‑grade viral vectors for first-in-human studies.
Development and validation of analytical assays for transgene expression, immunogenicity, biodistribution and other key preclinical/clinical endpoints.
End-to-end preclinical development of gene therapy programs including proof-of-concept efficacy studies, pharmacology, biodistribution and safety testing to support IND-enabling packages.
Coordination with CDMO partners to advance AAV process development, GLP/GMP production and transfer of clinical‑grade viral vectors for first-in-human studies.
Development and validation of analytical assays for transgene expression, immunogenicity, biodistribution and other key preclinical/clinical endpoints.
Expertise Areas
- Gene therapy development
- AAV vector process development and manufacturing partnerships
- Preclinical IND‑enabling studies (pharmacology, biodistribution, toxicology)
- Liver-directed gene delivery
Key Technologies
- Adeno-associated virus (AAV) vectors
- AAV8 serotype liver-directed delivery
- Transgene design for coagulation pathway modulation (activated FVa)
- Gene editing with stable genomic integration
News & Updates
GeneVentiv Therapeutics was announced as the winner of the Startup Spotlight pitch competition at BioPharm USA 2023, highlighting its innovative universal gene therapy for hemophilia.
GeneVentiv announced its participation in the BIO CEO & Investor Conference in New York, where it will present its lead gene therapy GENV-HEM for hemophilia.
GeneVentiv announced its attendance at BIO Europe Fall 2023, where it will meet with prospective investors and present its gene therapy programs.
GeneVentiv appointed Dr. Valder Arruda to its Scientific Advisory Board to guide the development of its hemophilia gene therapy programs.
GeneVentiv expanded its Scientific Advisory Board with experts in hemophilia and gene therapy, including David Lillicrap, Steven Pipe, Benjamin Samelson-Jones, and Linda Couto.
GeneVentiv announced the addition of Robert Baffi and Deborah Wild as advisors to its team, bringing extensive experience in gene therapy manufacturing and biotech leadership.
GeneVentiv Therapeutics was announced as the winner of the Startup Spotlight pitch competition at BioPharm USA 2023, highlighting its innovative universal gene therapy for hemophilia.
GeneVentiv announced its participation in the BIO CEO & Investor Conference in New York, where it will present its lead gene therapy GENV-HEM for hemophilia.
GeneVentiv announced its attendance at BIO Europe Fall 2023, where it will meet with prospective investors and present its gene therapy programs.
GeneVentiv appointed Dr. Valder Arruda to its Scientific Advisory Board to guide the development of its hemophilia gene therapy programs.
GeneVentiv expanded its Scientific Advisory Board with experts in hemophilia and gene therapy, including David Lillicrap, Steven Pipe, Benjamin Samelson-Jones, and Linda Couto.
GeneVentiv announced the addition of Robert Baffi and Deborah Wild as advisors to its team, bringing extensive experience in gene therapy manufacturing and biotech leadership.