PARIS--(BUSINESS WIRE)-- Regulatory News:

Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform Company targeting central nervous system (CNS) diseases, announces today that it will give an oral presentation at the ESGCT 2021 Annual Virtual Conference on its gene therapy drug candidate LYS-GM101 in GM1 gangliosidosis.

Dr. Michaël Hocquemiller, Lysogene’s Head of Non-Clinical Development, will present the results of the non-clinical pharmacology and toxicology studies and the current status of the ongoing global adaptive design clinical trial (NCT04273269) during Session “4a CNS & Sensory II” on Thursday, October 21 at 10:00 am.

“These preclinical results demonstrate that LYS-GM101 was able to correct the pathology in the mouse and feline model of the disease and to achieve strong and extensive transgene expression and distribution throughout the central nervous system (CNS) of non-human primates following intracisternal administration. Extrapolating these preclinical results to humans, it is reasonable to expect that LYS-GM101 would restore sufficient enzymatic activity in the CNS of patients to have a significant positive impact on disease progression,” said Dr. Michaël Hocquemiller.

About Lysogene
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 delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. A phase 2/3 clinical trial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. is ongoing. An adaptive clinical trial in GM1 gangliosidosis is ongoing. 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 has also entered into an exclusive worldwide license agreement with SATT Conectus for a gene therapy candidate for the treatment of the Fragile X syndrome, a genetic disease related to autism. www.lysogene.com.

Forward Looking Statement
This press release may contain certain forward-looking statements, especially on the Company’s progress of its clinical trials and cash runway. 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, (ii) factors beyond the Company’s control, (iii) clinical trial results, (iv) increased manufacturing costs, (v) potential claims on its products, and (vi) a modification of the terms of its agreements with Sarepta Therapeutics. These statements may include, without limitation, any statements preceded by, followed by or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “objective”, “project,” “will,” “can have,” “likely,” “should,” “would,” “could” and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance or achievements to be materially different from the expected results, performance or achievements expressed or implied by such forward-looking statements. A further list and description of these risks, uncertainties and other risks can be found in the Company’s regulatory filings with the French Autorité des Marchés Financiers, including in the 2020 universal registration document, registered with the French Markets Authorities on April 12, 2021, under number D.21-0296, and future filings and reports by the Company. Furthermore, these forward-looking statements are only as of the date of this press release. Readers are cautioned not to place undue reliance on these forward-looking statements. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. If the Company updates one or more forward-looking statements, no inference should be drawn that it will or will not make additional updates with respect to those or other forward-looking statements.

This press release has been prepared in both French and English. In the event of any differences between the two texts, the French language version shall supersede.

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