Amsterdam Molecular Therapeutics (AMT) has entered into an agreement with Institut Pasteur, and a group of French research institutes (the “Consortium”) to support clinical development of a gene therapy to treat Sanfilippo B., a rare genetic disease affecting newborn children that leads to progressive neuronal degeneration and death.

Institut Pasteur will lead the development program and sponsor the initial Phase I/II study of a gene therapy to replace an enzyme (alpha-N-acetylglucosaminidase) that is missing in brain cells of SanfilippoB patients. This enzyme is specifically required for the degradation of heparan sulfate glycosaminoglycans (GAGs), essential carbohydrate molecules used to build tissue. The accumulation of incompletely degraded GAG molecules triggers a cascade of pathological events leading to neuronal dysfunction and death.

AMT will manufacture and supply the adeno-associated viral 5 (AAV5) gene therapy product to the Consortium. The overall manufacturing contract is valued at $2.3 million. If the Phase I/II study is successful, AMT will have an option to acquire full commercial rights for the program. The study is scheduled to begin before 2013. AMT has a cGMP-licensed, 375-sq.-m. manufacturing facility to produce its AAV vectors for gene therapy products.

“This partnership leverage’s AMT’s proven expertise in cGMP manufacturing of gene therapy products and our experience in progressing these products through clinical development and the regulatory processes needed for successful commercialization,” said Jorn Aldag, chief executive officer of AMT. “In addition, we will have an option to acquire full commercial rights for the program on completion of the Phase I/II study, which supports our strategy to build a pipeline of orphan and ultra-orphan indications.”

Muriel Eliaszewicz, medical director of Institut Pasteur, said, “AMT is one of the only companies in the world that has a proven ability in manufacturing cGMP quality gene therapy products, not only in batches sufficient for clinical development but also in support of a potential regulatory approval. We are delighted that we now have the ability to commence the difficult process of developing a cure for patients with this very challenging disease.”