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Sarepta, Lysogene ink deal to develop gene therapy for rare disorder

Sarepta Therapeutics Inc. and Lysogene SA are jointly developing a gene therapy, known as LYS-SAF302, to treat a rare inherited disorder that affects the brain and spinal cord.

LYS-SAF302 is being developed to treat mucopolysaccharidosis type 3A, also called Sanfilippo syndrome type A — a lysosomal storage disorder that leads to the increasing disability or death of the central nervous system's cells. Patients with the disease show behavioral problems and loss of previously acquired skills, resulting in their early death.

The companies signed a license deal under which Sarepta will receive exclusive commercial rights to LYS-SAF302 in the U.S. and elsewhere, except Europe, where Lysogene will retain its rights to the drug.

French biotech Lysogene will complete a pivotal study, set to begin in the fourth quarter, that will evaluate the effectiveness of the drug. Sarepta will globally manufacture the drug and will supply Lysogene for its territory.

Sarepta, a Cambridge, Mass.-based biopharmaceutical company, will also have certain option rights to an additional gene therapy candidate targeting the central nervous system.

In 2018, Sarepta will pay Lysogene $26 million in cash and will subscribe to an equity investment in Lysogene of $2.5 million via the issuance of 950,606 ordinary shares. In 2019, Sarepta will pay Lysogene up to an additional $19 million.

Total payments, considering all milestones are achieved, would amount to $125 million plus royalties. Lysogene said it will use proceeds to develop its other assets.

Torreya served as exclusive financial adviser to Lysogene.