Lysogene is a leading biotechnology company specialized in the development of AAV-based gene therapies with a portfolio of candidates to treat rare central nervous system diseases.

Lead candidate LYS-SAF302 is indicated for the treatment of MPSIIIA (Sanfilippo Syndrome), a rare lysosomal storage disease for which there is no treatment. This disease affects children who suffer from impaired cognitive and motor functions and have short life expectancy.

Lysogene has already obtained efficacy signals in a Phase I/II study and is finalizing the recruitment of the pivotal Phase II/III trial for LYS-SAF302, which has the potential to become first- and best-in-class.

Proceeds will primarily be used to finance the Phase I/III trial of LYS-GM101 in GM1 gangliosidosis and the preparation of LYS-SAF302’s commercial launch in Europe for MPSIIIA.


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 phase 2/3 clinical trial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. is ongoing and a phase 1/3 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 strategy of development for the treatment of Fragile X syndrome, a genetic disease related to autism.

Discover Lysogene

Hervé Ronin

Investment Banking

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Pierre Kiecolt-Wahl

Equity Capital Markets

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Vincent Meunier

Investment Banking

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Cosme Rosellini

Equity Capital Markets

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Mickael Dubourd

Investment Banking

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Pierre Sertour

Equity Capital Markets

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