LYSOGENE, a leading gene therapy company, is developing a treatment for Sanfilippo syndrome Type A, a fatal disease affecting children for which there is currently no cure.
Paris, May 21. 2014 – LYSOGENE, a leading gene therapy biotechnology company focused on the development of breakthrough treatments for severe genetic pathologies with central nervous system involvement, announced today that it has closed a €16.5 million Series A financing. Further to its initial seed to the series A investment, Paris-based Sofinnova Partners led this financing round, becoming the firm's main institutional investor. New and co-lead investors include Bpifrance, through its Innobio fund, and Novo Seeds, part of Novo A/S. Rafaèle Tordjman, Chahra Louafi and Henrijette Richter, respectively as representatives of Sofinnova Partners, Innobio and Novo A/S, will sit on the board.
LYSOGENE was founded in 2009 by Karen Aiach, CEO, and Professor Olivier Danos, Scientific Advisor, an internationally renowned gene therapy scientist who among key positions served as Chief Scientific Officer at Genethon and as Director of the Gene Therapy Consortium at University College London. The firm is specialised in intracerebral gene therapy aimed at treating neuro-degenerative diseases, such as Sanfilippo syndrome Type A, an extremely severe and debilitating disorder which is fatal in childhood and affects approximately 1 in 100,000 births. Sanfilippo syndrome is a lysosomal storage disease stemming from a deficiency in a particular enzyme, for which there is currently no treatment. In 2013, LYSOGENE successfully completed a phase I-II study (Eudract 2010-019962-10 / NCT01474343) using its lead product in Sanfilippo patients.
LYSOGENE's unique and highly innovative therapeutic approach consists in injecting a functional version of the defective gene directly into a young patient's brain. In a single dose treatment, it enables to target the central nervous system providing constant levels of therapeutic protein. Unlike existing potential developing approaches focusing on managing symptoms or preventing complications, LYSOGENE's technology addresses the root cause and, therefore, ultimately aims at curing the disease. Importantly, LYSOGENE's single-dose approach is also expected to represent a considerable quality of life benefit for patients and their families. The funds raised will be used to expand the clinical studies for its main product developed for patients with Sanfilippo syndrome Type A, towards its marketing approval in Europe and the US. Funds will also allow the expansion of the pipeline with a second product targeting another genetic disease affecting the central nervous system.
Karen Aiach, CEO, adds: "Sofinnova Partners' ongoing support since their seed to the series A financing has been a real booster. It has allowed us to successfully reach new decisive milestones. In addition, LYSOGENE has been able to attract renowned investors like Innobio and Novo Seeds. Today, we are in a strong position to successfully face our challenges: the company is preparing the pivotal clinical study for its lead product and quickly plans on developing a second product addressing another genetic pathology involving the central nervous system, a field where patients' and families' needs still remain highly unfulfilled".
Rafaèle Tordjman, MD, PhD, Managing Partner at Sofinnova Partners said: "We are thrilled to back such a dynamic and experienced team. The firm has achieved extraordinary progress in less than five years. We are convinced that the technology has the potential to become a realistic option for such particularly vulnerable patients who currently have no treatment options".
Henrijette Richter, Investment Director at Novo Seeds, comments: "We are excited to support LYSOGENE. It has the most advanced gene therapy approach on Sanfilippo syndrome type A, and its initial results are extremely encouraging".
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