Encouraging results from ongoing gene therapy clinical trial for Sanfilippo type A

15 Feb 2021

Abeona Therapeutics presented encouraging results from their ongoing gene therapy clinical trial for Sanfilippo type A at WORLD Symposium over the weekend. Nineteen children have now been treated in this trial and includes Australian children at a trial site at the Women’s and Children’s Hospital in Adelaide. The presentation focused on the three youngest children given the gene therapy, at ages 27 months, 19 months, and 12 months. The results showed that the cognitive development of these children has been preserved within the normal range.  They are now at ages ranging from 3.5 years to 5+ years and their development would be expected to have slowed by now. Although the cognitive results for the older children in the trial were not positive, the trial continues to collect data on other symptoms of the disease that may have improved the quality of life of these participants. We eagerly await the full results of the trial. 

Results from the Abeona trial for Sanfilippo type B patients were also presented and the results appear to be similar, however it is still early days for this trial and more data needs to be gathered.

The research team at Sanfilippo Children’s Foundation is preparing a full report of the conference and will share as soon as possible.

Read the full press release here