Dialog Box


Our Projects


Investigating potential of stem cell therapy for Sanfilippo Type B


  • Project title: Neural and Mesenchymal stem cell mediated gene therapy for MPS IIIB
  • Chief investigator: Dr. Coy Heldermon
  • Amount: $90K (over 1 year)
  • Location: University of Florida, USA
  • Start date: March 2017

Read more about Dr. Heldermon's research here 


Improving delivery of gene therapy to brain for Sanfilippo Type C


  • Project title: Improving brain delivery of adeno-associated gene therapy vectors for the treatment of MPS IIIC and other neurodegenerative diseases 

  • Chief Investigator: Dr. Brian Bigger
  • Amount: $90K (1 year)
  • Location: University of Manchester, UK
  • Start date: March 2017

 Read more about Dr. Brian Bigger's research here

High throughput screening for new drugs to treat Sanfilippo

  • Project title: New therapy for six Mucopolysaccharidoses
  •  Chief Investigator: Associate Professor Andreas Schulze
  •  Amount: $448K (over 2 years)
  •  Location: Hospital for Sick Children, Toronto, Canada
  •  Start Date: March 2017


Read more about Assoc. Prof. Andreas Schulze's research here


Investigating the role of brain lipids (fats) in Sanfilippo

  • Project title: Brain lipid homeostasis: A critical event in Sanfilippo A neuropathology
  • Chief Investigator: Associate Professor Maria Fuller
  • Amount: $90,900 (1 year)
  • Location: SA Pathology, Adelaide
  • Grant Type: Incubator
  • Status: Active  


Read more about Assoc. Prof.Maria Fuller's research here 


Abeona Therapeutics Gene Therapy Program



  • Project title:  ABO-102 Gene Therapy for Patients with Sanfilippo Syndrome Type A (MPS IIIA) and  ABO-101 Gene Therapy for Patients with Sanfilippo Type B (MPS IIIB)
  • Chief Investigator: Abeona Therapeutics
  • Amount $USD 1 million
  • Status: Active


Read more about Abeona's clinical trial here


Read further about our Research Strategy our Research Funding Program and our Scientific Advisory Board