Dialog Box


Abeona Therapeutics Gene Therapy Program


Our first & immediate project: Abeona’s gene therapy trial


With the support of its expert advisors, the Sanfilippo Children's Foundation has undertaken careful review of the current research programs around the world.


We have elected the Abeona’s gene therapy trial as our first and immediate project to fund and support.  We aim to contribute USD1M to the Abeona trial over a period of 18 months.



Abeona Therapeutics:

  • Abeona Therapeutics is a US-based biotech founded in March 2013.
  • Developing gene therapy based cures for Sanfilippo Type A & B, working in close collaboration with specialist clinicians at Nationwide’s Hospital (Columbus, Ohio). 
  • Closed 750,000 USD seed round in 4Q2013 and actively seeking funding.
  • Has secured strong international support from multiple patient organisations (Spain, Canada, US, Switzerland, Mexico, Argentina).

Treatment overview:

  • Therapies developed for both Type A and B.
  • Strong clinical data demonstrates potential for efficacy in pre-clinical research. 
  • High safety profile of non-invasive drug delivery mechanism (intravenous).
  • Administration method using Adeno-Associated Viral (AAV) vectors, proven as effective and safe for the delivery of corrective genes.
  • Clinical trial phase I/II began in the USA in 2016.
  • Multi-territory trial (USA, Spain & Australia).


Program Information  

Press Releases for latest info including trial results 


For further information on this program, contact:

Michelle Berg
Vice President, Patient Advocacy
Abeona Therapeutics
Phone: 216.346.7405


For information for clinicians/ families on the Australian arm of this program, contact Primary Investigator: 

Dr Nicholas Smith
Department Head, Paediatric Neurology 

Women’s and Children’s Hospital, Adelaide  

Phone:  (08) 8161 7308