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Abeona gene therapy trial news – first Australian treated and new higher dose

October 12, 2017

On October 11, 2017 it was announced that two children with Sanfilippo Type A had been treated in the Abeona gene therapy trial at a new higher dose including in Australia. This is the third dose tested in the trial with children previously being treated at lower doses. Encouraging results have been seen in all the treated children with a more pronounced effect in the higher the dose, and no safety concerns.


It is still very early, but 30 days after the first child received the high dose of gene therapy, the trend is continuing with large reductions in heparan sulfate in the cerebral spinal fluid and urine and the liver notably reducing in size. Children with Sanfilippo have enlarged livers and heparan sulfate is the damaging sugar molecule that accumulates in the cells, which is particularly damaging in the brain.

Experience from gene therapy trials for other similar conditions has suggested that treatment outcomes will be improved by earlier intervention, at the highest possible tolerated doses. One of the children who received the new higher dose was treated at Adelaide Women’s & Children’s Hospital and the other was at Nationwide Children’s Hospital in the US. Patient screening is also underway at a site in Spain.


"We are pleased to initiate our enrolment in the ABO-102 trial,” stated Nick Smith, M.D., Ph.D, Principal Investigator and Department Head of Neurology at the Adelaide Women’s & Children’s Hospital in Australia.


Executive Director of the Sanfilippo Children's Foundation, Megan Donnell: "I am thrilled by this progress which sees Australian children have the same opportunity to access emerging therapies as their counterparts in the US and Europe, and thankful that we have such a strong clinical team here running this program."

About the trial
The Abeona Therapeutics gene therapy program is a whole-body treatment approach using an intravenously delivered adeno-associated virus (AAV) gene therapy to treat paediatric patients with Sanfilippo Syndrome Type A (MPS IIIA). The virus delivers a correct copy of the gene (SGSH) that is altered in children with Sanfilippo.

Read the full press release

Category: News