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.
Abeona Therapeutics:
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Abeona Therapeutics is a US-based biotech company developing gene therapy-based treatments for epidermolysis bullosa and retinal eye diseases.
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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)
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In 2022, Abeona handed the Sanfilippo type A gene therapy programme over to Ultragenyx and terminated its type B program
Ultragenyx:
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Ultragenyx is a US-based biotech company with a strong track record in developing and marketing treatments for rare and ultra-rare conditions
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Ultragenyx’s portfolio includes medicines for X-linked hypophosphatemia; long-chain fatty acid oxidation disorders; and Mucopolysaccharidosis VII (Sly syndrome).
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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
Email: patientadvocacy@ultragenyx.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