ReciBioPharma to advance haemophilia treatment with GeneVentiv Therapeutics

By Isabel Cameron

- Last updated on GMT

© Getty Images
© Getty Images

Related tags Haemophilia Gene Gene therapy AAV AAV platform

ReciBioPharm has partnered with pre-clinical gene therapy company 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.

According to the company, approximately 30% of people with haemophilia A and 5% with haemophilia B will develop an inhibitor (an antibody) to the treatment they receive to manage a bleeding episode.

This partnership aims to address the currently unmet need for AAV-based gene therapies for haemophilia patients with inhibitors.

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.”

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.

AAV therapy development is complex and the manufacturing process requires cost efficiencies, flexibility and speed to ensure key milestones are met.

ReciBioPharm will utilise its AAV manufacturing platform at its Watertown facility in Massachusetts, to advance GeneVentiv’s therapy from early stage pre-clinical to phase 1/2 clinical studies.

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 minimise 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.”

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