Sarepta, Lysogene Announce Exclusive License Agreement for LYS - SAF302, a Late- stage Gene Therapy for the Treatment of MPS IIIA; Grant of Option Rights to an Additional CNS Gene Therapy Candidate

Sarepta Therapeutics, Inc.

announced today it has signed a license agreement with Lysogene (FR0013233475 – LYS), a pioneering biopharmaceutical company specializing in gene therapy targeting central nervous system (CNS) diseases, for the development of a gene therapy, LYS-SAF302, to treat Mucopolysaccharidosis type IIIA (MPS IIIA). Under the terms of today's agreement, Sarepta will also have certain option rights to an additional CNS-targeted gene therapy candidate. Under the terms of the license, Lysogene shall be responsible for completion of the pivotal trial, which is set to commence in the fourth quarter of 2018. Sarepta shall have exclusive commercial rights to LYS-SAF302 in the United States and all territories outside of Europe, and Lysogene will retain exclusive commercial rights to LYS-SAF302 in Europe. Sarepta will be responsible for global manufacturing of LYS-SAF302 and will supply Lysogene for its territory. In 2018, Sarepta will make committed cash payments to Lysogene totaling $26 million (€22 million) plus the purchase of Lysogene equity for $2.5 million (€2.2 million). In 2019, Sarepta will pay Lysogene up to an additional $19 million (€16 million). Payments from Sarepta to Lysogene for all items, if all milestones are met, would total approximately $125 million (€108 million) plus royalties. The financial terms of the agreement significantly extend Lysogene's cash runway, enabling the company to continue the development of its other assets. "We stand together today with the MPS community and Lysogene in service of a common goal of developing what could be a transformative therapy for this cruel disease," stated Doug Ingram, Sarepta's president and chief executive officer. "As with our other therapies targeted to serious, lifealtering genetic diseases, we share with Lysogene a sense of urgency and a deep commitment to see this program through to fruition. Toward that goal, Sarepta will leverage its expertise in rare disease therapies and gene therapy to bring LYS-SAF302 to the MPS community." "This partnership with Sarepta, an innovative global leader in genetic diseases, is a major step forward for Lysogene in our commitment to bring a therapy to market to treat MPS IIIA patients," said Karen Aiach, Lysogene's chief executive officer and founder. "We believe that Sarepta is the ideal partner for LYS-SAF302 in the U.S. and other countries outside of Europe. We are proud of this important validation of our efforts to date and look forward to working together closely with Sarepta." As part of this partnership, Sarepta is subscribing to an equity investment of $2.5 million (€2.2 million) at a 30% premium to the 5-day volume-weighted average share price, through the issuance of 950,606 ordinary shares, with the same rights as existing shares, under a capital increase with cancellation of preemptive rights under article L.225-138 of the French Commercial Code and the 22d resolution of Lysogene's Extraordinary General Meeting in June 2018. The issuance of these shares will result in dilution of 7.1% for existing Lysogene shareholders. The issuance of these shares does not require the publication of a prospectus submitted to the visa of the Autorité des Marchés Financiers in accordance with article 211-3 of the AMF General Regulations. Lysogene intends to apply the funds raised for general corporate purposes including the further development of LYS-GM101 for GM1 gangliosidosis. Torreya acted as exclusive financial advisor to Lysogene.