CLOSE

Dialog Box

Loading...

Our Projects

 

 Creating a zebrafish model for Sanfilippo Type A

 

  • Project title: Targeting Creating a MPSIII zebrafish model and brain specific in vivo lysosome reporter
  • Chief investigator: Dr. Jan Kaslin
  • for drug screening and in vivo imaging of disease pathology
  • Amount: $45K (over 1 year), with Cure Sanfilippo contributing a further $45K
  • Location: Victoria, Australia
  • Status: Active
  • Start date: February 2018  

                                                                                                                                                       

Read more about Dr. Kaslin's research here                                                                                                                                                                                                                                                                                                                                        


Designing "chaperone" drugs for Sanfilippo Type A

 

  • Project title: Pharmalogical chaperones for MPSIIIA
  • Chief investigator: Associate Professor Vito Ferro

  • Amount: $90K (over 1 year)
  • Location: Queensland, Australia
  • Status: Active
  • Start date: February 2018

     

Read more about Prof. Vito Ferro's research here 

 


 Targeting misfolded proteins for therapy for Sanfilippo Type C

 

  • Project title: Targeting Misfolded Proteins for Therapy of MPSIIIC
  • Chief investigator: Professor Alexey Pshezhetsky

  • Amount: $98K (over 1 year)
  • Location: Montreal, Canada
  • Status: Active 
  • Start date: February 2018
 

Read more about Prof.  Alexey Pshezhetsky'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

 


PhD Scholarship - SAHMRI

 

  • Project Title: How are the neurons damanged 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 in the Childhood Dementia Research Group, as part of the Hopwood Centre for Neurobiology 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. Laura is co-supervised by Dr Kim Hemsley (SAHMRI) and Prof Robert Richards (University of Adelaide)
  • Amount: $10,000 over 2 years
  • Status: Active

 

Read more about Laura Hewson's research here


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
  • Status: Completed 
  • Start date: March 2017 

 

Project completed. Read a Research Update here

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 or Scientific Advisory Board