Abeona Therapeutics (now ULTRAGENYX) Gene Therapy Program
The Sanfilippo Children’s 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 is also a clinical trial site in Spain.
In 2022, this gene therapy programme was taken over by another biotech company, Ultragenyx. Ultragenyx will complete the clinical trial of this AAV9-based gene therapy treatment for Sanfilippo type A and, dependent on the trial outcomes, will seek regulatory approval for the treatment.
For up-to-date information on this trial and other Sanfilippo clinical trials, please go to our webpage
Abeona Therapeutics is a US-based biotech company developing gene therapy-based treatments for epidermolysis bullosa and retinal eye diseases.
Abeona initiated the Sanfilippo Type A & B programs in close collaboration with specialist clinicians at Nationwide’s Hospital (Columbus, Ohio) with strong international support from multiple patient organisations (Spain, Canada, US, Switzerland, Mexico, Argentina, Australia)
In 2022, Abeona handed the Sanfilippo type A gene therapy programme over to Ultragenyx and terminated its type B program
Ultragenyx is a US-based biotech company with a strong track record in developing and marketing treatments for rare and ultra-rare conditions
Ultragenyx’s portfolio includes medicines for X-linked hypophosphatemia; long-chain fatty acid oxidation disorders; and Mucopolysaccharidosis VII (Sly syndrome).
They have a pipeline of seven other therapies in pre-clinical and clinical development including UX111, an AAV9-based gene therapy for Sanfilippo type A (formerly known as ABO-102)
For further information on this program, contact:
Patient Advocacy, Ultragenyx
Phone: +1 415.483.8800
For information for clinicians/ families on the Australian arm of this program, contact:
SA Health Australia
Phone: +61 08 81617295