LYSOGENE launches the first gene therapy clinical trials in Sanfilippo disease

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LYSOGENE announces the authorization by AFSSAPS (the French Agency for the Safety of Health Products) and by the Ethical Review Board of SAF-301, its leading intra-cerebral gene therapy phase I/II clinical trial aiming at treating Sanfilippo Type A Syndrome.

LYSOGENE crosses an historical gap and launches the clinical trial of its first product, SAF-301, an experimental investigational medicinal product drug intended to treat MPSIII- A also known as the Sanfilippo Type A Syndrome - type A.

Sanfilippo Syndrome belongs to the group of lysosomal storage diseases (LSD) with extremely severe central nervous system (CNS) involvement. It is a heavy debilitating and life threatening disease with high unmet medical needs. Today, as for all LSDs with CNS involvement, there is no cure available.

SAF-301 is designed to bring the functional version of the defective gene. Preclinical studies have evidenced very promising safety and efficacy profiles.

LYSOGENE’s SAF-301 study is a non-controlled, non-randomized open label pilot trial. Primary objective is to evaluate the clinical, radiological, biological tolerance associated to the proposed treatment. Secondary objective is to collect data to define exploratory tests that will become evaluation criteria for further clinical phase III efficacy studies (Brain MRI; neurological and biological markers).

Four patients will be included; The inclusion period will be of eighteen months; The length of follow-up is one year for each patient. Therefore, the maximum duration of the trial should be 2 years and a half.

Pr. Marc TARDIEU, Principal Investigator at Bicêtre hospital, will follow patients and Dr. Michel ZERAH, Neurosurgeon at Necker hospital, will conduct neurosurgical operations. The Scientific leader of the program is Dr. Olivier DANOS.

SAF-301 is the first paediatric clinical program using intra-cerebral gene transfer ever performed in Europe.

"Launching a clinical trial for an advanced gene therapy program is a great victory, in such a short timing. The AFSSAPS validation is a big step towards the development of the treatment of the SAN FILIPPO disease. It is also an extraordinary opportunity to quickly expand our therapeutic business model to other genetic neurodegenerative diseases" said Karen Aiach, Founder and CEO of the company.

Clinical trials will start in September 2011.


Founded in 2009 by Karen Aiach, LYSOGENE is a biopharmaceutical company pioneering in gene therapy targeting severe diseases of the central nervous system with genetic origin.

LYSOGENE is committed to develop advanced therapies for CNS diseases with high unmet medical needs, to make a difference and improve patients’ lives. Leading patients’ organizations such as Alliance SANFILIPPO, AFM-Telethon and Foundation Sanfilippo Suisse has supported the program.

About the Sanfilippo syndrome and LSDs:

Sanfilippo syndrome type A (or MPSIIIA) is a rare and fatal lysosomal storage disease due to an enzyme deficiency of the protein N-sulfohydrolase sulfoglycosamine (SGSH). The main clinical symptom is a degenerative neurological condemning afflicted child with a life expectancy of between 10 and 20.

Lysosomal storage disease (LSDs) are a group of over 40 inherited disorders typically characterized by a deficiency in one or more enzymes that degrade glycans, the carbohydrate chains of glycoproteins, proteoglycans and glycolipids. Their total combined incidence grater than 1 per 8,000 births.


The opinions expressed here are the views of the writer and do not necessarily reflect the views and opinions of News Medical.
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