Lysogene Launches World Class Clinical Advisory Board

Lysogene Launches World Class Clinical Advisory Board 150 150 Lysogene

September 12th 2017


PARIS, France, and CAMBRIDGE, MA, US – September 12th 2017, 06:00 pm CET – Lysogene (FR0013233475 – LYS), a leading biopharmaceutical company pioneering gene therapy technologies to treat central nervous system diseases, today announced the appointments of leading experts to its newly formed Clinical Advisory Board (CAB). The CAB will provide strategic advice to Lysogene as it continues to advance its clinical development programs and devise commercialization paths for its orphan gene therapy candidates to treat rare CNS diseases, beginning with Mucopolysaccharidosis Type IIIA (MPS IIIA) and GM1 Gangliosidosis (GM1) patients.

“The launch of the CAB further position Lysogene to successfully reach our goal of bringing transformational medicines to patients with severe neurological rare diseases”, said Karen Aiach, Founder and Chief Executive Officer of Lysogene. “Our CAB members will bring invaluable experience and insight to Lysogene as we move our clinical-stage gene therapy product candidates forward.”

Lysogene’s inaugural CAB members include:

  • Anupam Chakrapani, MD, Great Ormond Street Hospital for Children NHS (GOSH), London, UK
  • Ronald G. Crystal, MD, Weill Cornell Medicine, New York, USA
  • Roberto Giugliani, PhD, MSc, MD, University of Rio Grande do Sul, Porto Alegre, Brazil
  • Bénédicte Héron, MD, Armand-Trousseau Hospital, APHP, Paris, France
  • Nicole M. Muschol, MD, University Medical Center Hamburg-Eppendorf, Germany
  • Raymond Y. Wang, MD, Children’s Hospital of Orange County, CA, USA
  • Chester B. Whitley, PhD, MD, University of Minnesota, MN, USA
  • Michel Zerah, MD, Necker Enfants Malades, Paris, France

The CAB complements Lysogene’s existing Scientific Advisory Board comprised of the most eminent international gene therapy and Lysosomal Storage Disease experts.

About Lysogene

Lysogene ( is a global biopharma leader in orphan CNS disease research and development. Lysogene has generated five non-cumulative years of clinical safety data to show the efficiency of a direct delivery route to the CNS with its initial gene therapy trial for MPS IIIA. Lysogene has

recently completed the enrollment for the first multi-national observational study in MPS IIIA which will function as the non-concurrent control for the first pivotal trial for MPS IIIA in H1 2018. Lysogene also plans a clinical trial for GM1 Gangliosidosis for 2019. Lysogene has obtained orphan drug designation from the EMA and FDA and rare pediatric designation by the FDA for both programs.

Lysogene is listed on the Euronext regulated market in Paris (ISIN code: FR0013233475). For more information:

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