Sarepta, Lysogene end partnership for rare disease treatment

Jan. 14, 2022

Sarepta Therapeutics ended its partnership to develop a gene therapy for rare disease treatment with Lysogene Therapeutics months before data from the study is expected to arrive. 

The drug, called LYS-SAF302, is intended to be used for the treatment of mucopolysaccharidosis Type IIIA (MPS IIIA), a fatal neurological disease for which there is currently no treatment. Sarepta stayed with Lysogene through the death of a child during phase 2/3 of clinical trials and a clinical hold imposed on the trial due to localized findings on MRI images at injection sites in the brain. LYS-SAF302 was granted Orphan Drug Designation in the U.S. and European Union. In the U.S. it has also received Fast Track and Rare Pediatric Disease designations.

Sarepta had inked the $15 million partnership with France-based Lysogene for the development of LYS-SAF302 back in 2018. The deal gave Sarepta full commercial rights to LYS-SAF302 in the U.S. and other markets outside of Europe, while Lysogene retained rights in Europe. As part of the deal, Sarepta assumed responsibility for all global manufacturing of the gene therapy. According to Lysogene, the deal fell apart over "unsuccessful discussions" over transferring this production responsibility back to Lysogene.

Now, post-separation, Lysogene will regain development and commercialization rights in the U.S. and other non-EU territories.  The breakup will become effective as of July 11.

Even without its partner, Lysogene  is not giving up on the treatment. “With the data readout expected in a few months time, we believe more than ever that LYS-SAF302 is an important treatment option for patients suffering from MPSIIIA, and we remain fully committed to the MPSIIIA community,” said Karen Aiach, CEO of Lysogene.