Dialog Box


Sanfilippo Type C: One step closer to clinical trial

June 29, 2018

In exciting news, US-based biotech Phoenix Nest Inc has signed a licence deal with the University of Manchester to take its research by Professor Brian Bigger’s laboratory to clinical trial for patients with Sanfilippo Type C. This is a highly anticipated and much welcomed move for the families of children with Sanfilippo Type C – and will be the first clinical trial for this particular Sanfilippo subtype.


The Sanfilippo Children’s Foundation was one of eight Foundations who helped co-fund this research project.  Special mention to the Morrice family in Brisbane Australia who have helped to fund this research via their Hope for Alec campaign. You can read about our Foundation's contribution here


The technology developed by Professor Bigger involves the use of a specially modified virus called adeno-associated viral vector (AAV), which has been specifically altered to efficiently deliver the missing HGSNAT gene to the brain to treat the disease.

Working with an international group of scientists, the team was able to demonstrate complete behavioural and brain correction of Sanfilippo Syndrome Type C in mice.


Prof Brian Bigger, Professor of Cell and Gene Therapy at The University of Manchester, said: “This gene therapy technology recently published in the journal Brain, will be used by Phoenix Nest to treat Sanfilippo syndrome Type C.


“We were really impressed that we were able to completely correct working memory and hyperactivity in the mouse model – traits shared by children with the disease.


“Working together with Phoenix Nest Inc we hope this therapy will be successful in treating children with MPSIIIC in the next few years.”


The study was funded by MRC, King’s College Commercialisation Institute, Jonah’s Just Begun,  Sanfilippo Barcelona, Sanfilipo Portugal, Sanfilippo Brasil, Le Combat de Haitem-Contre Sanfilippo, JLK- Sanfilippo Research Foundation, Sanfilippo Children’s Foundation, and VML charities.


Michelle Morrice, mother to Alec who has Sanfilippo Syndrome Type C,  said: “When I heard the news of this project it felt like I was no longer looking down a tunnel just seeing darkness, now I can see the slightest glimmer of light.  


Fundraising is not easy... you put yourself into a domain you're not necessarily comfortable with becuase you have the passion to change something that touches your soul. You work towards a goal with the hope of success but your mind keeps you a realist. This news,  for the first time, allows me to let my mind open to the idea of a future with my son." 


Read press release in full here




Category: Research News