Lysogene delays Sanfilippo gene therapy phase 3

Paris, France, where Lysogene is based. (iStock/Getty Images Plus/PocholoCalapre)

A manufacturing delay has pushed back the start of Lysogene’s pivotal Sanfilippo syndrome trial. The gene therapy study is now due to get underway in the second half of the year. 

Paris, France-based Lysogene wrapped up preclinical toxicology work on LYS-SAF302 last year, giving it the dose for the phase 2/3 trial. And the European Medicines Agency signed off on the pediatric investigation plan for the candidate in February. Those events teed Lysogene up to get the gene therapy into the pivotal trial but events elsewhere have slowed its progress.

Lysogene has been working on CMC matters for some time but is yet to get its manufacturing setup to the point where it can support the phase 2/3. That has pushed the start date for the trial to the back half of 2018.


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With manufacturing acting as a bottleneck, Lysogene is working on other preparations for the study to militate against further delays. Plans to activate the eight trial sites involved in the study are up and running. And enrollment in an observational trial that will serve as a comparator to the phase 2/3 study is complete.  

Lysogene is looking to the pivotal trial for evidence its gene therapy provides a one-shot improvement to the health of people with Sanfilippo syndrome type A, also known as mucopolysaccharidosis type IIIA. The gene therapy delivers a copy of the SGSH gene, which encodes for an enzyme that is absent in children with the condition.

While the manufacturing delay means Lysogene will have to wait a little longer to learn if clinical data validate its hypothesis, the French biotech is nonetheless entering a busy and important period.

“This year, we expect to initiate our pivotal clinical trial in mucopolysaccharidosis type IIIA in the U.S. and Europe and pursue IND enabling studies for our GM1 Gangliosidosis product,” Lysogene CEO Karen Aiach said in a statement. “We are also delighted to have secured a collaboration agreement for the development of an AAV-based gene therapy for Fragile X syndrome, the most common genetic cause of autism, a condition of high unmet medical need.”

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