Consortium to Develop Sanfilippo B Gene Therapy

(National Institute on Aging, NIH)

Amsterdam Molecular Therapeutics (AMT), a biopharmaceuticalcompany in the Netherlands, and a group of French research institutes, including the Institut Pasteur in Paris, has formed a consortium to develop a gene therapy to treat Sanfilippo B. AMT will manufacture compounds to be tested in a clinical trial of the therapy.

Sanfilippo B is a rare genetic disease affecting new-born children that leads to progressive neuronal degeneration and death. Clinical symptoms usually first appear between the ages of 2 and 6 years in children who previously appeared normal. Typical symptoms begin with aggressive behavior, sleep disorders, hyperactivity and delayed development. Severe neurological deterioration begins by age 6 in most patients. There is no approved therapy currently available.

Institut Pasteur will lead the development program and will also sponsor the initial phase 1-2 clinical study of a gene therapy to replace an enzyme missing in brain cells of SanfilippoB patients. This enzyme is required to degrade heparan sulfate glycosaminoglycans (GAGs), key carbohydrate molecules used to build tissue. The accumulation of incompletely degraded GAG molecules triggers pathological events leading to neuronal dysfunction.

AMT will manufacture and supply the gene therapy product to the consortium. If the clinical trial shows the therapy’s effectiveness, AMT will have an option to acquire full commercial rights for the program. The clinical trial is funded by the the French Muscular Dystrophy Association, a member of the consortium.

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