Gene Therapy Biotech Lysogene Reels In $22 Million

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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”.

About Lysogene

LYSOGENE is a clinical stage biotechnology company committed to the development of innovative therapies for patients affected with rare disorders and high unmet medical needs. LYSOGENE’s team translated its rAAVrh10 lead product for Sanfilippo from bench to bedside in an unprecedented fashion over the last years. Its lead product is for Sanfilippo syndrome, a neurodegenerative lysosomal storage disorder considered to be a perfect model for gene therapy. LYSOGENE is currently expanding its pipeline to additional diseases with high unmet medical needs.

For more information about LYSOGENE, please visit

About Sanfilippo syndrome Type A

Sanfilippo syndrome Type A (Mucopolysaccharidosis IIIA or MPS IIIA) is a fatal, rare, autosomal recessive condition characterized by the accumulation of heparan sulphate in cells, in particular brain cells, resulting in rapid neurodegeneration, severe and invasive behavioural disorders and mild peripheral symptoms. The disease affects approximately 1 in 100,000 births (still believed to be largely underdiagnosed) and patients only survive until their late teens or early twenties. It is caused by mutations in a gene that encodes the N-sulfoglucosamine sulfohydrolase (sulfamidase) enzyme. Lysosomal storage diseases are a group of approximately 50 rare inherited metabolic disorders characterised by the deficiency in one or more enzymes responsible for degradation of components that the cell doesn’t need anymore (such as mucopolysaccharides) within the lysosome. Lack or deficiency of such enzyme leads to the accumulation of un-degraded components in the lysosomes resulting in the dysfunction of the implicated organ.

About Sofinnova Partners

Sofinnova Partners is an independent venture capital firm based in Paris, France. For more than 40 years, the firm has backed nearly 500 companies at different stages of their development – pure creations, spin-offs, as well as turnaround situations – and worked alongside key entrepreneurs in the Life Sciences industry around the globe. With over €1.3 billion of funds under management, Sofinnova Partners has created market leaders with its experienced team and hands-on approach in building portfolio companies through to exit.

For more information, please visit:

About Bpifrance

Bpifrance’s equity investments are operated by Bpifrance Investissement. Bpifrance invests directly or indirectly has a minority party through partner’s equity, in French companies, PME, ETI and larger companies. InnoBio is a 139 million euro FCPR managed by Bpifrance and subscribed by the FSI (37%) and the key global pharmaceutical companies (Sanofi Aventis, GSK, Roche, Novartis, Pfizer, Lilly, Ipsen, Takeda, Boehringer- Ingelheim) operating in France. The main objective of the fund is to directly invest equity or partial equity in the capital of firms who provide innovative and technological products and services in the Health sector. InnoBio, is made up of a team of investors specialised in the industry and has to this day made 13 investments of a total of 65 million euros.

About Novo A/S

Novo A/S, the holding company in the Novo Group, is responsible for the management of the assets of the Novo Nordisk Foundation, which are currently valued at more than USD 30 billion. Novo A/S is a private limited liability company fully owned by the Novo Nordisk Foundation. Besides being the major shareholder in Novo Nordisk A/S and Novozymes A/S, Novo A/S provides seed and venture capital to development stage companies and takes significant ownership positions in well-established companies, within life science and biotechnology, as well as manages a broad portfolio of financial assets. For further information, please visit

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