Phoenix Nest Licenses Sanfilippo Syndrome Gene Therapy Prepares Clinical Trial

Rare Daily Staff

Phoenix Nest exclusively licensed a gene therapy for Sanfilippo syndrome (also known as Mucopolysaccharidosis IIIC, (MPS IIIC)) from the University of Manchester in the United Kingdom. MPS IIIC is a devastating neurodegenerative lysosomal storage disorder of childhood for which there is no cure or effective treatment available.

Research results on the rAAV gene therapy, which had recently been published in the journal Brain, demonstrate preclinical evidence, according to Brian Bigger, professor of Cell and Gene Therapy, University of Manchester. “The vector we are using has an established safety profile and is in widespread use in treating conditions of neurological origin. It is our expectation that this will translate into a successful treatment for this disease.”

“We are excited about the potential of this gene therapy,” said Jill Wood, co-founder and chief financial officer of Phoenix Nest. “Phoenix Nest has an orphan designation for this treatment from the European Medicines Agency and is currently submitting this designation to the US Food and Drug Administration along with the Pre-IND. We are also starting vector manufacturing in preparation for a clinical trial in the very near future.”

Phoenix Nest is focused on developing Sanfilippo Syndrome. It was co-founded by Jill Wood, who is a parent of a child with Sanfillipo Syndrome type C, and Sean Ekins, a scientist with more than 20 years of experience in pharmaceutical R&D. Phoenix Nest has been the recipients of five National Institutes of Health grants to date.

“Our focus on developing different treatment modalities for Sanfilippo syndrome leverages a wide array of academic partners. Our strategy can help us translate these outstanding preclinical efforts and bring them to the patients faster. This creates considerable value for all stakeholders,” said Ekins, CEO and co-founder.

“Phoenix Nest is in great admiration of the parents of children affected by Sanfilippo Syndrome. These families have taken a disease with no hope of survival to the brink of a clinical trial. These are the heroes that deserve all the credit for identifying and funding leading academics and inspiring the scientists to create a drug for this insidious disease.  If not for my child, then for the next generation, no parent should have to watch their child slowly die of a genetic syndrome that can be treated,” said Wood.

November 9, 2018

Photo: Jill Wood, chief financial officer and co-founder of Phoenix West