ReciBioPharm & GeneVentiv Therapeutics Partner to Advance First AAV-Based Gene Therapy for Haemophilia Patients With Inhibitors

ReciBioPharm has recently announced a collaboration agreement with GeneVentiv Therapeutics to advance development of an Adeno-Associated Virus (AAV)-based universal gene therapy for haemophilia, and the first to treat haemophilia patients with inhibitors.

On average 30% of people with haemophilia A and about 5% of people with haemophilia B will develop an inhibitor (an antibody) to the treatment they receive to manage a bleeding episode. The ReciBioPharm and GeneVentiv partnership will help address the currently unmet need for AAV-based gene therapies for haemophilia patients with inhibitors.

GeneVentiv’s GENV-HEM (AAV8.FVa) is the first, single infusion, universal AAV-based gene therapy for all types of haemophilia and has demonstrated therapeutic efficacy and safety in preclinical studies. This collaboration will see ReciBioPharm accelerate the development of this technology using its cutting-edge AAV manufacturing platform.

AAV therapy development is complex: the manufacturing process requires cost efficiencies, flexibility, and speed to be built into every stage of the process to ensure crucial milestones are met. ReciBioPharm will utilize its AAV platform at its Watertown facility in Massachusetts to advance GeneVentiv’s therapy from early stage preclinical to Phase 1/2 clinical studies.

Xiaojun Liu, Director of AAV Process Development at ReciBioPharm, said “We are delighted to be working with GeneVentiv, an ambitious and innovative biotech who wanted to leverage not just our equipment and space, but our extensive knowledge and expertise too.”

Damon Race, CEO of GeneVentiv Therapeutics, added “Gene therapies pose unique development and manufacturing challenges, so it was essential we chose the right partner to collaborate with, to minimize manufacturing risks and ensure we meet our key development milestones. ReciBioPharm quickly demonstrated that their team is the perfect development and manufacturing partner for our asset, enabling us to access their extensive experience and impressive capabilities. Our collaboration with them provides us with GLP and GMP product to meet both our IND and Phase 1/2 milestones.”

Recipharm is a leading Contract Development and Manufacturing Organization (CDMO) in the pharmaceutical industry employing over 7,000 employees. Recipharm offers manufacturing services of pharmaceuticals and biologics in various dosage forms, production of clinical trial material and APIs, pharmaceutical product development and development and manufacturing of medical devices. Recipharm manufactures several hundred different products for customers ranging from big pharma to smaller research and development companies. The company operates development and manufacturing facilities in France, Germany, India, Israel, Italy, Portugal, Spain, Sweden, the UK and the US and is headquartered in Stockholm, Sweden. For more information, visit www.recipharm.com

GeneVentiv Therapeutics is a preclinical gene therapy company focused on blood disorders. Our lead program, GENV-HEM (AAV8.FVa), is the only single infusion, universal, AAV-based gene therapy able to treat all types of haemophilia. Unlike other AAV-based haemophilia gene therapies, GENV-HEM is the only gene therapy able to treat the 33% of haemophilia patients with neutralizing antibodies (inhibitors) to their missing clotting factor. There are 50,000 inhibitor patients in the developed world. GENV-HEM has received Orphan Drug Designation from the FDA for Haemophilia A and B with or without inhibitors and a Letter of Support from the National Bleeding Disorders Foundation.