Lysogene Announces Workshop at National Institutes of Health (NIH) on CNS Immunogenicity Considerations for AAV- mediated Gene Therapy

Lysogene Announces Workshop at National Institutes of Health (NIH) on CNS Immunogenicity Considerations for AAV- mediated Gene Therapy 150 150 Lysogene

Paris, France, – 6 June 2019, at 06:00 pm CEST — Lysogene (FR0013233475 – LYS), a leading, biopharmaceutical company pioneering gene therapy technologies to treat central nervous system diseases, today announced the organization of a workshop on CNS immunogenicity considerations for adeno-associated virus (AAV)-mediated Gene Therapy on June 11, 2019 from 8:30 a.m. to 5 p.m. ET at the National Institutes of Health (NIH).

Lysogene played a major role in the planning and organization of this workshop. The purpose of the meeting is to bring together the scientific and medical expert community for AAV-mediated therapies to focus on issues related to the central nervous system (CNS) administration and immunogenicity of these products.

AAV gene therapy holds the promise to transform CNS disorders by providing a durable therapeutic effect following a single intervention. However, transgene expression and overall efficacy of gene therapy strategies in humans can be impaired by undesirable humoral and cellular host immune response against the viral capsid protein and/or against the protein expressed from the administered gene. How to prevent immune reactions, and how to evaluate their consequences on efficacy and safety of AAV gene delivery, are crucial questions.

“By sharing immunogenicity findings from AAV clinical trials and bringing together the main experts in the field, this workshop holds the promise to provide guidance for the advancement of CNS gene therapy,” said Samantha Parker, Chief Patient Officer at Lysogene, member of the Organizing Committee for this event.

“It is crucially important to discuss and share our current understanding with experts in the field and to reach consensus on best treatment practices”, continued Sophie Olivier, Chief Medical Officer at Lysogene.

Individuals, who wish to view the workshop, can register to the live videocast on

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 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 development strategy for the treatment of Fragile X syndrome, a genetic disease related to autism.


Mathilde Bohin / Emmanuel Huynh NewCap

+ 33 1 44 71 94 95

Lysogene’s forward-looking statements

This press release may contain forward-looking statements, especially on the Company’s progress of its Phase 2/3 clinical trial. Although the Company believes that its expectations are based on reasonable assumptions, any statements other than statements of historical facts that may be contained in this press release relating to future events are subject to (i) change without notice, (ii) factors beyond the Company’s control and (iii) the Company’s financial capabilities. These statements may include, but are not limited to, any statement beginning with, followed by or including words or phrases such as “objective”, “believe”, “anticipate”, “foresee”, “aim”, “intend”, “may”, “anticipate”, “estimate”, “plan”, “project”, “will”, “may”, “probably”, “should”, “could” and other words and phrases of the same meaning or used in negative form. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that may, if any, cause actual results, performance or achievements to differ materially from those anticipated or expressed explicitly or implicitly by such forward- looking statements. A list and description of these risks, contingencies and uncertainties can be found in the documents filed by the Company with the Autorité des Marchés Financiers (AMF) pursuant to its regulatory obligations, including the Company’s 2018 registration document, registered with the AMF on 29 April 2019 under number R. 19-016, as well as in the documents and reports to be published subsequently by the Company. In addition, 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. Except as required by law, the Company does not undertake any obligation to publicly update these forward-looking statements or to update the reasons why actual results could differ materially from those anticipated by the forward-looking statements, including in the event that new information becomes available. The Company’s update of one or more forward- looking statements does not imply that the Company will make any further updates to such forward-looking statements or other forward-looking statements.

This press release is for information purposes only. The information contained herein does not constitute an offer to sell or a solicitation of an offer to buy or subscribe for the Company’s shares in any jurisdiction, in particular in France. Similarly, this press release does not constitute investment advice and should not be treated as such. It is not related to the investment objectives, financial situation or specific needs of any recipient. It should not deprive the recipients of the opportunity to exercise their own judgment. All opinions expressed in this document are subject to change without notice. The distribution of this press release may be subject to legal restrictions in certain jurisdictions. Persons who come to know about this press release are required to inquire about and comply with these restrictions.

Lysogene COVID-19 update

Get In Touch

Don’t hesitate to contact us if you need more help.