Dialog Box


Abeona Phase 1B trial of Gene Therapy Program

The Sanfilippo Children’s Foundation has joined a collaborative agreement between nine global Sanfilippo Foundations to conduct a phase 1b gene therapy trial for Sanfilippo types A and B.

This experimental treatment uses a virus to deliver a healthy copy of the faulty gene to the cells of children with Sanfilippo types A and B. The ongoing phase 1A trial is producing promising preliminary results.

Collectively the foundations will grant US$13.875m to Abeona with Sanfilippo Children’s Foundation contributing US$1.5m. This will secure an Australian site for the phase 1b trial which is expected to start by mid next year, and give more Australian children the opportunity to access this emerging treatment.


Read press release in full here

For further information on this program, contact:
Michelle Berg
Vice President, Patient Advocacy 
Abeona Therapeutics
Phone 216.346.7405


  • Project Title: Phase 1b trial of gene therapy (ABO-101 & ABO-102)
  • Chief Investigator: Abeona Therapeutics
  • Amount: US $1.5 Million
  •  Status: Mid 2018