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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 Heldermon 
  • 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


PhD Scholarship - SAHMRI

  • Project Title: How are the neurons damaged in Sanfilippo and how can it be prevented? 
  • Chief Investigator: Andrew Schoubridge University of Adelaide PhD project will be carried out under the guidance of Dr Kim Hemsley at the Lysosomal Diseases Research Unit at the South Australian Health and Medical Research Institute (SAHMRI).
  • Amount: $15,000 over 3 years
  • Status: Active 

 

Read more about Andrew Schoubridge's research here


PhD Scholarship - University of Adelaide

  • Project title: Investigating the molecular mechanisms that mediate neuropathology in Sanfilippo Syndrome
  • Chief investigator: Laura Hewson’s PhD project will be carried out under the guidance of Dr Lousie O’Keefe at the University of Adelaide 
  • Amount: $10,000 over 2 years
  • Status: Active

 

Read more about Laura Hewson's research here


Abeona Phase 1B trial of Gene Therapy program

 

  •  Project Title: Phase 1B trial of gene therapy (ABO-101 & ABO-102)
  • Chief Investigator: Abeona Therapeutics
  • Amount: US 1.5 Million
  • Status:  Commencement TBA

 

 Read more about Abeona Therapuetics Phase 1B clinical trial here

 

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