Date: 2014-04-15
Type of information: Series A financing round
Company: AAVLife (France)
Investors: Versant Ventures (USA) Inserm Transfert Initiative (France)
Amount: $ 12 million (€ 8.68 million)
Funding type: series A financing round
Planned used: This funding will be used to advance into clinical studies AAVLife\'s work on Friedreich’s ataxia recently reported in Nature Medicine (Morgane Perdomini, Brahim Belbellaa, Laurent Monassier, Laurence Reutenauer, Nadia Messaddeq, Nathalie Cartier, Ronald G Crystal, Patrick Aubourg, Hélène Puccio. Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich\'s ataxia. Nature Medicine, 2014; DOI: 10.1038/nm.3510).This research paper reported the successful use of a gene-therapy approach in a mouse model developed by deleting the frataxin gene. The authors report that these mice develop the same progressive degradation of cardiac function, and that delivery of a normal frataxin gene using an adeno-associated virus completely restored normal cardiac function and reversed pathological heart enlargement in mice that had already progressed to heart failure. The results show that a single intravenous injection of AAVrh10 expressing frataxin is not only capable of preventing the development of heart disease in animals before the appearance of symptoms, but also, more impressively, of fully and rapidly curing the hearts of animals at an advanced stage of heart disease. After three weeks of treatment, the heart become fully functional again; mitochondrial function and the appearance of heart tissue being very similar to those of healthy mice. AAVLife has already advanced research of this gene-therapy approach into further preclinical studies to inform decisions about dosing and route of administration to be used in a clinical trial. The company will be conferring with regulatory authorities about toxicity-study requirements and clinical-trial design. The goal is to commence a clinical trial in 2015 to evaluate gene therapy for the cardiac dysfunction associated with Friedreich’s ataxia.
Others: * On April 15, 2014, AAVLife, a gene-therapy company focusing on rare diseases, has announced that it has raised $12 million in Series A financing to advance into clinical studies the work on Friedreich’s ataxia recently reported in Nature Medicine. Versant Ventures, an international life-sciences venture-capital firm, led the financing round with participation from Inserm Transfert Initiative, a French seed-investment firm linked to the French National Institute of Health and Medical Research (Inserm). The founders of AAVLife have already played roles in bringing gene therapy into clinical use. Patrick Aubourg, M.D. Ph.D, Head of Neuropediatrics at Hôpital Bicêtre Paris Sud, led the first clinical trial to use gene therapy in the treatment of adrenoleukodystrophy (ALD). Pierre Bougnères, M.D., Ph.D., Head of Pediatric Endocrinology at Hôpital Bicêtre Paris Sud has developed and performed the ALD trial with Dr. Aubourg. Ronald Crystal, M.D., Chairman of Genetic Medicine and Professor of Internal Medicine at Weill Cornell Medical College, New York leads the research group that first used a recombinant virus as a vehicle for in vivo gene therapy. The group has carried out human trials of gene therapy for cystic fibrosis, cardiac ischemia, cancer, and central-nervous-system disorders. Hélène Puccio, Ph.D., Head of a research team at the Institute of Genetics and Molecular and Cellular Biology, University of Strasbourg, Francen has concentrated her research on the pathophysiological mechanisms involved in ataxias such as Friedreich’s ataxia. She is the lead author on the paper in Nature Medicine. Amber Salzman, Ph.D., Chief Executive Officer. Dr. Salzman, a mathematician by training, served as a member of the R&D executive team at GSK, where she planned and managed drug-development projects and clinical trials comprising over 30,000 patients worldwide. Subsequently, she led Cardiokine Inc. as CEO prior to its acquisition by Cornerstone Therapeutics. She is the president of the Stop ALD Foundation, a patient-advocacy group driving forward improved ALD therapy. She played a key role in bringing about the initial ALD gene-therapy trial.
Therapeutic area: Rare diseases - Neurodegenerative diseases
