GeneVentiv Therapeutics Advances GENV-HEM with NHLBI Support
GeneVentiv Therapeutics Secures NHLBI Catalyze Program Funding
GeneVentiv Therapeutics, a pioneering company in next-generation gene therapies, has recently taken a significant step forward with the selection into the National Heart, Lung, and Blood Institute (NHLBI) Catalyze Program. This ultra-competitive initiative is designed to support medical innovations that have the potential to transform patient care.
Boosting Research in Gene Therapy
The NHLBI Catalyze Program focuses on funding vital preclinical studies, specifically toxicology assessments necessary for the Investigational New Drug (IND) submission of GENV-HEM. This investigational gene therapy aims to offer new hope for patients suffering from hemophilia A, including those who have developed inhibitors.
Reducing Barriers to Development
Funding from the NHLBI will help alleviate the financial burden associated with crucial toxicology work, allowing GeneVentiv to redirect investor capital towards higher-return projects. The Chief Executive Officer, Damon Race, highlighted how this selection gives strong validation to their science and regulatory strategy. By significantly lowering the cost barriers in gene therapy development, they are in a position to propel GENV-HEM into the clinical phase more rapidly.
Complementing Ongoing Initiatives
The NHLBI Catalyze support will complement GeneVentiv's current NHLBI Small Business Innovation Research (SBIR) award. This ongoing initiative allows the company to develop essential IND-enabling assays and conduct a large-animal efficacy and safety study. The combination of both programs ensures that GeneVentiv receives comprehensive support to handle aspects of efficacy, safety, and regulatory readiness, significantly mitigating costs and risks involved with advancing their therapies.
Transformative Potential of GENV-HEM
Dr. Paris Margaritis, Chief Scientific Officer, expressed confidence in their strategy, emphasizing the importance of having award recognition from NHLBI for both efficacy and toxicology. GENV-HEM aims to be the inaugural gene therapy that offers a solution for all hemophilia A patients, regardless of their inhibitor status, representing a significant breakthrough in treatment accessibility.
About GeneVentiv Therapeutics
GeneVentiv Therapeutics is dedicated to creating groundbreaking gene therapies for rare and serious diseases. Its flagship product, GENV-HEM, utilizes an AAV-based approach to create a one-time treatment for hemophilia A. The company is also developing GENV-002, a CRISPR gene editing therapy targeting both Infantile and Late Onset Pompe disease. The company's mission is clear: to develop therapies that can benefit every patient dealing with chronic illnesses, rather than limiting treatment options to a select few.
Contact Information
Damon Race
919-529-3352
For further inquiries, feel free to reach out via the contact information provided.
Frequently Asked Questions
What is the NHLBI Catalyze Program?
The NHLBI Catalyze Program is a prestigious initiative that funds innovative medical projects with significant potential to affect patient care positively.
How does this funding affect GeneVentiv's projects?
This funding will substantially reduce the costs associated with toxicology studies needed for the IND submission of GENV-HEM, enabling the company to focus on advancing its clinical timetable.
What is GENV-HEM designed to treat?
GENV-HEM is a gene therapy intended for the treatment of hemophilia A, including patients who may have developed inhibitors against traditional therapies.
What are the other notable projects in development by GeneVentiv?
In addition to GENV-HEM, GeneVentiv is also working on GENV-002, a dual-vector CRISPR gene editing therapy for both Infantile and Late Onset Pompe disease.
What are the implications of NHLBI support for GeneVentiv's future?
The NHLBI support reinforces the company's scientific and regulatory strategies, enhancing their credibility and potentially improving their market position in gene therapy innovation.
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