US-based biopharmaceutical company Abeona Therapeutics has secured regulatory approval from the Australian Health Therapeutic Goods Administration to commence a Phase I/II clinical trial of its ABO-102 for the treatment of Sanfilippo syndrome type A (MPS IIIA).

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ABO-102 (AAV-SGSH) is a gene therapy programme based on the adeno-associated virus (AAV).

To be conducted at the Adelaide Women’s and Children’s Hospital, the Phase I/II trial is designed to assess the safety and efficacy of single, intravenous ABO-102.

The injection of the drug is to systematically deliver the AAV viral vector throughout the body for generating a corrective copy of the gene associated with the disease.

Abeona president and chief executive officer Timothy Miller said: "Abeona has enrolled more MPS IIIA patients in a gene therapy trial than any other group in the world, and the addition of a third clinical site will accelerate our ability to evaluate ABO-102 as a potential treatment for patients with Sanfilippo syndrome type A, or MPS IIIA.

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"We remain encouraged by the recently reported clinical data and look forward to enrolling patients in Australia and Spain shortly."

“We remain encouraged by the recently reported clinical data and look forward to enrolling patients in Australia and Spain shortly.”

During the initial six months after the injection, the evaluation for safety and initial signals of biopotency is carried out at various time points.

The Phase I/II trial is being supported by the MPS III natural history study that examined the potential efficacy, neurocognitive evaluations, biochemical assays and MRI data for one year in 25 subjects.

The trial has also obtained fast-track designation, orphan product designation and rare paediatric disease designation from the US Food and Drug Administration (FDA).

Image: Surface of the AAV-2 serotype of the adeno-associated virus. Photo: courtesy of Jazzlw.