GeneVentiv Therapeutics Secures NHLBI Funding for Hemophilia A Therapy

GeneVentiv Therapeutics Selected for NHLBI Catalyze Program

GeneVentiv Therapeutics is making significant headway in the field of biotechnology through its recent selection for the prestigious Catalyze Program by the National Heart, Lung, and Blood Institute (NHLBI). This competitive initiative supports groundbreaking medical innovations with transformative potential, and GeneVentiv's entry into this program is a key milestone for the company.

The funding provided by the NHLBI Catalyze Program will particularly focus on carrying out the essential toxicology studies needed for the Investigational New Drug (IND) application for GeneVentiv's innovative gene therapy, known as GENV-HEM. This targeted treatment is designed as a one-time therapy for patients suffering from hemophilia A, catering to the needs of individuals with or without inhibitors.

Damon Race, the CEO of GeneVentiv Therapeutics, expressed his enthusiasm about this development, stating, "Selection into the NHLBI Catalyze Program represents strong validation of our science and regulatory strategy. It alleviates a major financial barrier in gene therapy development and allows us to redirect investor funds toward promising activities that will hasten the progression of GENV-HEM towards clinical use. Furthermore, this funding enables us to allocate more resources to the continuing development of GENV-002, our gene editing therapeutic for Pompe disease."

The NHLBI Catalyze Program is aimed at supporting the most elite medical breakthroughs, thus significantly reducing the financial burden associated with the IND-enabling toxicology phase. This financial relief is crucial as it accelerates the timeline for GeneVentiv to initiate clinical trials, which are vital for bringing new therapies to patients who desperately need them.

In addition to this advancement, GeneVentiv is also benefiting from its ongoing Small Business Innovation Research (SBIR) award provided by NHLBI. This grant helps fund critical assays and safety studies on animal models, showcasing a broader national commitment to supporting innovative solutions in medicine. Together, these resources not only enhance efficacy and safety assessments but also play a pivotal role in preparing for regulatory compliance, effectively mitigating costs and risks associated with IND progression.

Dr. Paris Margaritis, the Chief Scientific Officer at GeneVentiv, commented on the significance of having NHLBI awards for both efficacy and toxicology. He remarked, "Having merit-based NHLBI awards for both efficacy and toxicology underscores the strength and broad applicability of our approach. GENV-HEM is engineered to be the first gene therapy targeting all hemophilia A patients, including those with inhibitors, through a singular product. With NHLBI backing for essential preclinical studies, we are uniquely positioned to deliver a one-time therapy capable of transforming the lives of patients within the hemophilia community."

GeneVentiv Therapeutics is not merely creating conventional treatments; the company is dedicated to revolutionizing therapy options for rare and complex diseases. Their flagship project, GENV-HEM, represents an AAV (Adeno-associated Virus)-based gene therapy that expresses activated Factor V, aiming to provide a long-term solution for hemophilia A patients. In parallel, the company is working on GENV-002, which utilizes CRISPR technology to address both Infantile and Late Onset Pompe disease, demonstrating their broad scope of innovation.

As GeneVentiv Therapeutics continues to evolve and expand its commitments to pioneering genetic therapies, the recent selection for the NHLBI Catalyze Program stands as a testament to its dedication to scientific excellence and patient care. The future looks promising for hemophilia A research as GeneVentiv accelerates toward its clinical objectives, bringing hope to patients worldwide who are yearning for new, effective, and reliable treatment options.