Phoenix Nest Inc. Licenses a Sanfilippo Syndrome Gene Therapy From the University of Manchester and is Preparing for a Clinical Trial

Phoenix Nest Inc. exclusively licensed a gene therapy for Sanfilippo syndrome (also known as Mucopolysaccharidosis IIIC, (MPSIIIC)) from The University of Manchester through its IP commercialization arm UMI3 Ltd. MPS IIIC is a devastating neurodegenerative lysosomal storage disorder of childhood for which there is no cure or effective treatment available.

Dr. Brian Bigger (Professor of Cell and Gene Therapy, University of Manchester) states “our recent publication in Brain* on this rAAV gene therapy demonstrated preclinical evidence from behavioral and cognitive tests which provide an expectation of direct clinical benefit. 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. Phoenix Nest Inc. has an orphan designation for this treatment from the EMA and is currently submitting this designation to the FDA along with the Pre-IND. We are also starting vector manufacturing in preparation for a clinical trial in the very near future,” said Ms. Jill Wood, Co-Founder and Chief Financial Officer, Phoenix Nest.

“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 Dr. Sean 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,” states Jill Wood. #

* Brain,  2018 Jul 1;141(7):2014-2031


About Phoenix Nest, Inc. - Phoenix Nest, Inc. is a biotechnology company focused on Sanfilippo Syndrome Co-founded by Jill Wood (a parent of a child with Sanfillipo Syndrome type C) and Dr. Sean Ekins (a scientist with over 20 years of experience in pharma R&D). The company collaborates with various academics to develop treatments for Sanfilippo Syndrome. Phoenix Nest has been the recipients of 5 NIH grants to date.   

For further information, please contact Sean Ekins, Co-Founder and CEO, Phoenix Nest, 215-687-1320

About University of Manchester/UMI3 Ltd

The University of Manchester, a member of the prestigious Russell Group, is the UK’s largest single-site university with 38,600 students and is consistently ranked among the world’s elite for graduate employability.

The University is also one of the country’s major research institutions, rated fifth in the UK in terms of ‘research power’. World class research is carried out across a diverse range of fields including cancer, advanced materials, addressing global inequalities, energy and industrial biotechnology.

UMI3 Ltd is wholly owned by the University, which has over a 30-year history of IP commercialization. For more information, please visit

Source: Phoenix Nest Biotech