Sarepta licenses Lysogene’s Sanfilippo gene therapy in deal worth up to $142M

Illustration of three DNA helices
Lysogene expects to start a pivotal trial for the gene therapy, LYS-SAF302, by the end of the year. (Darwin Laganzon)

Lysogene’s gene therapy for Sanfilippo syndrome hit a snag earlier this year, when a manufacturing delay put off its pivotal trial. Now, the company is licensing the program to Sarepta Therapeutics for $15 million upfront, a $2.5 million equity investment and up to $125 million in potential milestone payments. 

Lysogene will bear responsibility for the pivotal trial and hold onto the European rights to the program, LYS-SAF302, while Sarepta will pick up the full commercial rights in the U.S. as well as in other markets outside of Europe. The deal brings Sarepta’s gene therapy portfolio to “as many as 14 programs,” the company said in a statement. It has gene therapy programs in Duchenne muscular dystrophy and Limb-Girdle muscular dystrophy, as well as in Pompe disease and a couple of undisclosed CNS indications. 

In addition to the Sanfilippo treatment, which is slated to start a pivotal phase 2/3 trial by the end of the year, Sarepta will have “certain option rights to an additional CNS-targeted gene therapy candidate.” 

Sanfilippo syndrome, also called Mucopolysaccharidosis type III, is a progressive disorder that affects the brain and spinal cord. It is divided into subtypes IIIA, IIIB, IIIC and IIID based on their genetic cause.

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LYS-SAF301 is designed to treat children with Sanfilippo syndrome type A by delivering a copy of the SGSH gene, which codes for an enzyme that is absent in children with the disorder. It has no specific treatment, so care focuses on managing symptoms, which include behavioral problems, joint stiffness, vision problems and hearing loss, among others. 

RELATED: Lysogene delays Sanfilippo gene therapy phase 3 

“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,” said Sarepta CEO Doug Ingram, in the statement.

“As with our other therapies targeted to serious, life-altering 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,”  he added.

RELATED: Sarepta leaps 50% on early data for Duchenne gene therapy 

Sarepta has been building its gene therapy unit for the past couple of years, inking deals in 2017 with Nationwide Children’s Hospital and Genethon around microdystrophin gene therapy programs. And this year, the biotech added another R&D pact, this one with Myonexus Therapeutics.

It forked over $60 million up front to license a clutch of gene therapies for Duchenne and Limb-Girdle muscular dystrophies. A few months later, the company hired Myonexus cofounder and chief scientific officer Louise Rodino-Klapac as vice president of gene therapy.

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