Lysogene, a phase 3 biopharmaceutical company relying on a gene therapy technological platform targeting central nervous system (CNS) diseases, today announces that the US regulatory agency (FDA ) approved its Investigational New Drug (IND) application for LYS-GM101, its gene therapy drug candidate for the treatment of GM1 gangliosidosis, a serious and life-threatening pediatric disease. LYS-GM101 builds on Lysogene's extensive experience in the clinical development of gene therapies based on adeno-associated viral vectors (AAVs) directly targeting the CNS.

This regulatory approval from the US FDA follows that recently granted by the MHRA in the United Kingdom. The Company intends to launch a comprehensive, multicenter, open-label, adaptive design, two-phase clinical trial of LYS-GM101 in patients with infantile forms of GM1 gangliosidosis. The clinical trial will include a phase focusing on safety analysis and a confirmatory phase of efficacy. The Company plans to treat 16 patients in total, with the first patient expected to be treated in the first half of 2021.

“We are very pleased to receive this IND authorization for LYS-GM101 which complements the MHRA approval received a few weeks ago. It is a major milestone that allows our second CNS gene therapy program to enter a global clinical trial, ” said Karen Aiach, Founding President and CEO of Lysogene . “This authorization once again demonstrates our quality and speed of execution, as well as our strong determination to provide new therapeutic solutions for diseases that currently have no treatment. "

Christine Wagoner, President and Co-Founder of the Cure GM1 Foundation added, “Children with GM1 gangliosidosis have a genuine unmet medical need, and we are delighted to see a new therapeutic option enter the clinical program as it brings great hope to families and to the entire GM1 gangliosidosis community ”.

LYS-GM101 ("adeno-associated viral vector serotype rh.10 expressing beta-galactosidase") received orphan drug designation for the treatment of GM1 gangliosidosis in the European Union and the United States in 2017, as well as the rare pediatric disease designation in the United States in 2016.

Leading international centers for gene therapy and lysosomal overload diseases are planning to participate in the clinical trial (NCT04273269).

Lysogene is also funding a natural history study on GM1 gangliosidosis conducted by Casimir Trials to collect prospective and / or retrospective videos of children with infant and juvenile GM1 gangliosidosis performing certain daily tasks and behaviors (NCT04310163).

About Lysogene
Lysogene is a biopharmaceutical company specializing in gene therapy targeting diseases of the central nervous system (CNS). The Company has acquired a unique expertise enabling it to deliver CNS gene therapies to treat lysosomal overload diseases and other genetic diseases of the CNS. Lysogene is currently conducting a Phase 2/3 clinical study in MPS IIIA, in partnership with Sarepta Therapeutics, Inc. An adaptive clinical study in GM1 gangliosidosis is under preparation. In accordance with the agreement between Lysogene and Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc. will hold the exclusive commercial rights for LYS-SAF302 in the United States and in markets outside Europe, while Lysogene will maintain the exclusive commercial rights of LYS -SAF302 in Europe.www.lysogene.com

Lysogene forward-looking statements
This press release may contain forward-looking statements, in particular concerning the progress of clinical trials and the Company's cash flow forecasts. Even if the Company considers that its forecasts are based on reasonable assumptions, all statements other than statements of historical fact that this press release may contain relating to future events are subject (i) to change without notice, (ii) factors beyond the Company's control, (iii) results of clinical studies, (iv) increases in production costs, and (v) potential claims on its products. These statements may include, without this list being exhaustive, all statements beginning with, followed by or including words or expressions such as "objective", "believe", "Foresee", "aim", "intend to", "be able", "anticipate", "estimate", "plan", "plan", "shall", "may have", "probably", " should ”,“ could ”and other words and expressions with the same meaning or used in the negative form. Forward-looking statements are subject to intrinsic risks and uncertainties beyond the control of the Company which may, from time to time, cause significant differences between the actual results, performance or achievements of the Company and those anticipated or expressed explicitly or implicitly by said forward-looking statements. A list and description of these risks, uncertainties and uncertainties appear in the documents filed by the Company with the Autorité des Marchés Financiers (AMF) in accordance with its regulatory obligations, including in the 2019 universal registration document of the Company, registered with the AMF on April 30, 2020 under number D.20-0427, as well as in the documents and reports that will be published subsequently by the Company. Furthermore, these forward-looking statements speak only as of the date of this press release. Readers are cautioned not to place undue reliance on these forward-looking statements. Unless required by law, the Company does not recognize any obligation to publicly update these forward-looking statements, nor to update the reasons why the actual results could vary significantly from the results anticipated by the forward-looking statements, including in the event that new information would become available.

Contacts
Stéphane Durant des Aulnois
Administrative and Financial Director
stephane.durant-des-aulnois@lysogene.com
+ 33 1 41 43 03 99

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