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
Preclinical gene therapy R&D
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.
Partnership management for clinical manufacturing
Coordination with CDMO partners to advance AAV process development, GLP/GMP production and transfer of clinical‑grade viral vectors for first-in-human studies.
Assay development and validation for gene therapy programs
Development and validation of analytical assays for transgene expression, immunogenicity, biodistribution and other key preclinical/clinical endpoints.
Preclinical gene therapy R&D
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.
Partnership management for clinical manufacturing
Coordination with CDMO partners to advance AAV process development, GLP/GMP production and transfer of clinical‑grade viral vectors for first-in-human studies.
Assay development and validation for gene therapy programs
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