Lysogene and French Scientist Partner to Develop Gene Therapy for Fragile X Syndrome

Ana de Barros, PhD avatar

by Ana de Barros, PhD |

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Tetra Therapeutics BPN14770

Lysogene has signed a partnership agreement with Hervé Moine, a pioneering French researcher, to potentially develop a gene therapy for fragile X syndrome (FXS) based partly on work supported by the Fraxa Research Foundation.

Moine, a member of the Institute of Genetics, Molecular and Cell Biology (IGBMC) and SATT Conectus Alsace, is working to use gene therapy to compensate for reduced diacylglycerol kinase kappa (DgkK) gene expression — an innovative therapeutic target and potential a key player in neuronal gene regulation. Its synthesis is regulated by the fragile X mental retardation protein (FMRP), the missing protein responsible for this syndrome.

The DgkK gene is responsible for diacylglycerol kinase kappa (DGKK) production, an enzyme that converts diacylglycerol (DAG) to phosphatidic acid (PA), two lipids used by cells for key molecular processes. DGKK serves as a switch that terminates the signaling of one lipid (DAG) while simultaneously activating signaling by another (PA).

FMRP regulates DgkK, and the lack of this protein in fragile X patients is thought to affect DGKK ability to work, and consequently for disease pathology.

Earlier work by Moine, supported by the Fraxa Research Foundation, successfully tested this approach to boost DGKK in a mouse model of fragile X.

Under the agreement, Moine and a team of researchers will work to develop a gene therapy to treat FXS patients.

If successful, Lysogene will have the option to license the product and guaranteed priority access to the technology.

“We are pleased by this agreement with prestigious partners in the field of Fragile X. This association, which should lead to an exclusive licensing agreement, is a recognition of Lysogene’s know-how in the development of therapies for rare CNS [central nervous system] diseases,” Philippe Mendels-Flandre, chief operating officer at Lysogene, whose headquarters are near Paris, said in a press release.

The project is the result of a strategic partnership that is also facilitated by the Fondation Maladies Rares in Paris, France, through its proof-of-concept (POC) Club. The POC Club promotes research and knowledge management with academic and industrial leaders in the field. First results are anticipated in 2019.

“The involvement of the Fondation Maladies Rares, the expertise of Lysogene and the support of Conectus, will enable us to test a new therapeutic approach for Fragile X, based on the discovery of a fundamental disease mechanism. Our team is very proud of this partnership, which is a unique opportunity to see our academic work developed into a treatment, awaited by families” Moine said.