More Gene Therapy Trials from New Players

ReciBioPharm and GeneVentiv Therapeutics are developing GENV-HEM, a gene therapy for hemophilia patients, including those with inhibitors. ReciBioPharm will help GeneVentiv take GENV-HEM from preclinical development to Phase 1/2 clinical testing. A one-time infusion, GENV-HEM is designed to deliver a gene coding for activated clotting factor V, through an adeno-associated virus 8 (AAV8), which has been modified to not cause disease in humans. In preclinical studies, GENV-HEM safely and effectively prevented bleeding in hemophilia animal models, regardless of the presence of inhibitors. In November 2021, GENV-HEM was granted orphan drug designation by the US FDA for hemophilia A and B, with or without inhibitors. Read more here.

Source: LA Kelley Communications, Inc., May 2024

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