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Our Projects

Optimising gene therapy for Sanfilippo Syndrome Type B

 

  • Project title: Gene therapy with intracranial delivery of capsid enhanced AAV NAGLU (MPSIII B)
  • Chief investigator: Dr. Coy Helermon 
  • Amount: $90K (over 1 year)
  • Location: University of Florida, USA
  • Status: Active  
  • Start date: March 2017

 

Read more about Dr. Heldermon's research here

 

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 Dr Andreas Schulze
  •  Amount: $448K (over 2 years)
  •  Location: Hospital for Sick Children, Toronto, Canada
  •  Start Date: March 2017

 

Read more about Assoc. Prof. Dr. 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