Abeona Therapeutics Gene Therapy Program

The Sanfilippo Childrens Foundation's first major investment in research was AUD $450,000 into Abeona Therapeutics in 2015. This led to the opening of a gene therapy clinical trial site in Adelaide and has given Australian children the opportunity to gain early access to a potential treatment.

The gene therapy trial for Sanfilippo type A started in the USA in May 2016 and the first Australian patients were treated at Adelaide’s Women’s and Children’s Hospital in 2017. There are also clinical trial sites in Spain, Germany and France.

Abeona Therapeutics:

  • Abeona Therapeutics is a US-based biotech founded in March 2013.
  • It is 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).


For further information on this program, contact:

Jodie Gillon
Vice President, Patient Advocacy
Abeona Therapeutics
Phone: 646.813.4714
Email: jgillon@abeonatherapeutics.com


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

Louise Jaensch

Research Nurse
SA Health Australia 
Phone: +61 08 81617295
Email: Louise.Jaensch@sa.gov.au