PARIS – Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform Company targeting central nervous system (CNS) diseases, today announces that it has entered into an exclusive, worldwide license agreement with SATT Conectus for the development and commercialization of a gene therapy candidate for the treatment of Fragile X syndrome.
Under the terms of the agreement, Lysogene will be responsible for the preclinical and clinical development, manufacturing, regulatory activities, and commercialization of the drug candidate, globally. SATT Conectus will receive an initial lump sum payment and may be eligible for development milestones and royalties on future product sales.
The gene therapy drug candidate aims at compensating for reduced DGKk, a novel therapeutic target whose synthesis is regulated by Fragile X Mental Retardation Protein (FMRP), the missing protein responsible for Fragile X syndrome. Lysogene has been collaborating since 2018 with Dr. Hervé Moine of the Institut de Génétique et de Biologie Moléculaire et Cellulaire (IGBMC) in Strasbourg, France, and SATT Conectus, the Technology Acceleration and Transfer Organization of the Alsace territory.
Fragile X syndrome affects approximately 1 birth in 4,000 to 5,000 boys and 1 birth in 8,000 girls, or about 110,000 patients in Europe and about 70,000 patients in the US. It is the most common inherited cause of intellectual disability as well as the most commonly known cause of autism spectrum disorder. There is currently no specific treatment available to cure the disease.
Karen Aiach, Founder, Chairman and CEO of Lysogene commented: “The acquisition of this global license for Fragile X syndrome is a major step forward. This incurable disease affects tens of thousands of patients across the globe. Our mission is to find a transforming therapeutic solution for patients. This transaction also testifies to the growing attractiveness of Lysogene: indeed, our powerful translational gene therapy platform for CNS applications attracts an increasing number of top-tier academic institutions. Our level of complementarity and synergy is complete, which will create significant value for Lysogene, the academic partners, and patients”.
Caroline Dreyer, CEO of Conectus commented: “I am delighted with this technology transfer between a renowned gene therapy company and one of the most important biomedical research institutes in Europe. It is the touchstone of a close collaboration forged over three years between the IGBMC, Lysogene and Conectus. A genuine co-conception process has guided this therapeutic adventure. SATT Conectus invested a significant amount and benefited from Lysogene’s expertise to conduct preclinical testing of this new therapeutic strategy. In return, Lysogene obtained an exclusive licensing option that was rapidly finalized at the end of the project. This co-development model is a guarantee for all parties to move forward in the best direction and in the shortest possible time”.
Carrel acted as counsel to the Company for this licensing agreement.
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 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.
About SATT Conectus*
SATT* Conectus Alsace is the main entry point for business partners and companies to access all the innovations and capabilities from public research in Alsace, one of the top ranked territories for the excellence of research in France. It borders Germany and Switzerland at the intersection of the most innovative areas of Europe. Thanks to its investment fund (up to 500k€ per innovative project), SATT Conectus offers advanced, applicative technologies whose proof of concept has been established, and ready to industrialise. Therefore investors will have access groundbreaking startups built around those breakthrough yet matured technologies. Another type of collaboration allows companies to co-develop high potential innovative projects with public research, in order to tailor them to their specific needs.
Since 2012: 1 375 collaborative research contracts signed – 120 innovative projects financed – 120 technologies transferred – 23 startups created, over 183 M€ raised from investors I Shareholders: CNRS, INSERM, ENGEES, INSA, UNIVERSITE DE STRASBOURG, UNIVERSITE DE HAUTE ALSACE, BPIFRANCE
www.conectus.fr/en ○ @ConectusAlsace
* Technology transfer acceleration organization
Stéphane Durant des Aulnois
Chief Financial Officer
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