Presentations to Highlight Results from Natural History and Treatment Studies for MPS IIIA and IND-enabling studies in GM1 gangliosidosis
Paris, France and Cambridge, MA, US —March 22, 2019 at 07:30am CET— Lysogene (FR0013233475 – LYS), a leading, biopharmaceutical company pioneering gene therapy technologies to treat central nervous system diseases, today announced that data will be presented at several scientific and family events in the upcoming weeks.
47th European Society for Pediatric Neurology (SENP), Paris, France
- Presentation: Design, baseline characteristics, and 2-year follow-up from the MPS IIIA natural history study used as control group in gene therapy trial
- Presenter: Dr Bénédicte Heron, Hôpital Trousseau, Paris
- Date: March 22, 2019
- Time: 12:15-12:30pm CET
3rd Annual Gene Therapy for Rare Disorders, Boston MA, USA
- Presentation: Navigating the Gene Therapy Path for CNS Diseases to the Clinic – Rationale, Pharmacology, and Delivery
- Presenter: Dr Ralph Laufer, Lysogene
- Date: March 27, 2019
- Time: 11:45am EST
National Tay Sachs and Associated Diseases (NTSAD) 41st Annual Family Conference, Raleigh, NC
- Research session: GM1 breakout
- Presenter: Samantha Parker, Lysogene
- Date : April 12, 2019
- Time: 9:30-10:45am EST
Lysogene is a gene therapy company focused on the treatment of orphan diseases of the central nervous system (CNS). The company has built a unique capability to enable a safe and effective delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. A pivotal clinical trial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. is ongoing and a phase 1-2 clinical trial in GM1 Gangliosidosis is in preparation. In accordance with the agreements signed between Lysogene and Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc. will hold exclusive commercial rights to LYS-SAF302 in the United States and markets outside Europe; and Lysogene will maintain commercial exclusivity of LYS-SAF302 in Europe. Lysogene is also collaborating with an academic partner to define the strategy of development for the treatment of Fragile X syndrome, a genetic disease related to autism. www.lysogene.com.
Julie Coulot / Emmanuel Huynh NewCap
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This press release may contain certain forward-looking statements, especially on the Company’s progress of its phase 2-3 clinical trial. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice,
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