AAVLife, a Paris, France-based gene-therapy company focusing on rare diseases, raised $12m in Series A financing.
The round was led by Versant Ventures, with participation from Inserm Transfert Initiative, a French seed-investment firm linked to the French National Institute of Health and Medical Research (Inserm).
The company intends to use the funds to advance into clinical studies.
Led by Amber Salzman, Ph.D., co-founder and Chief Executive Officer, AAVLife has already advanced research of a 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 start a clinical trial in 2015 to evaluate gene therapy for the cardiac dysfunction associated with Friedreich’s ataxia, which affects 10,000 to 20,000 people in the United States and Europe.
Other co-founders of the company include:
– Patrick Aubourg, M.D. Ph.D, Head of Neuropediatrics at Hôpital Bicêtre Paris Sud.
– Pierre Bougnères, M.D., Ph.D., Head of Pediatric Endocrinology at Hôpital Bicêtre Paris Sud. With Dr. Aubourg,
– Ronald Crystal, M.D., Chairman of Genetic Medicine and Professor of Internal Medicine at Weill Cornell Medical College, New York.
– Hélène Puccio, Ph.D., Head of a research team at the Institute of Genetics and Molecular and Cellular Biology, University of Strasbourg, France.