Progress Towards Clinical Trial for Treatment of MPS IIIC

U.S.-based biotechnology company Phoenix Nest, Inc. has signed a license agreement with the University of Manchester in the U.K. to use the product of research conducted by Professor Brian Bigger’s laboratory at the University of Manchester, working in collaboration with Dr. Els Henckaerts’ laboratory at King’s College London. Dr. Bigger is Professor of Cell and Gene Therapy at the University of Manchester. Dr. Henckaerts is Lecturer in the Department of Infectious Diseases at King’s College London. Phoenix Nest, Inc. plans to take the licensed research product to clinical trial for patients with Sanfilippo syndrome type C (MPS IIIC). This license agreement is an important early step on the long journey to an FDA-approved clinical trial of this gene therapy.

The research product which has been licensed by Phoenix Nest, Inc. is based on the discovery of a novel adeno-associated viral vector (AAV) with an altered protein coat, which appears to make the virus work better within the brain. This new vector, called AAV-TT (AAV-true type), has been altered to efficiently deliver the missing HGSNAT gene to the brain to treat MPS IIIC. Comprising an international group of scientists, the research teams concluded that they had demonstrated complete behavioral and brain correction of Sanfilippo syndrome type C in mice. Dr. Bigger said, “This gene therapy technology, recently published in the journal Brain, will be used by Phoenix Nest to treat Sanfilippo syndrome type C.”

The Sanfilippo Children’s Foundation (in Australia), an LDN-associated patient advocacy group, was one of eight foundations who helped co-fund this research project. The other co-funding foundations included: Jonah’s Just Begun, another LDN-associated patient advocacy group; King’s College London Commercialization InstituteSanfilippo BarcelonaSanfilipo PortugalSanfilippo BrasilLe Combat de Haitem-Contre SanfilippoJLK Sanfilippo Research Foundation; and VML Foundation. Additional funding sources are listed near the end of the article in Brain.