Dialog Box


New Abeona trial for older children

September 18, 2019


The original Abeona Sanfilippo Type A gene therapy trial (ABT-001) changed its criteria at the beginning of 2019 to enrol only patients under the age of two or with a minimum Cognitive Development Quotient (DQ) of 60 or above. DQ is a measure of a child’s cognitive ability. This new trial aims to enrol up to 12 patients with a DQ below 60 who are still able to walk. 

Key criteria for eligible patients include the following, but a full list of inclusion and exclusion criteria are available here
•      Diagnosis of MPS IIIA (Sanfilippo type A)
•      Cognitive Development Quotient (DQ) lower than 60
•      Must be ambulatory, with/without assistance

What will be required of patients and their families?

•      Up to two screening visits.
•      A treatment visit, once eligibility is confirmed. Patients will be admitted to the hospital for gene transfer and will stay for approximately 48 hours for observation.
•      Patients and their caregiver(s) will be required to stay near the clinical site for 30 days after gene therapy is administered.
•      After gene transfer, a total of nine follow up visits will take place at the clinical site, with the last visit occurring 24 months after gene transfer.
•      Home visits for collection of blood in between the follow-up visits to the clinical site.
•      After 24 months patients may be invited to participate in a long-term follow up study.

Abeona is planning a series of three webinars for families and advocates, to present more information about this trial.

1) Dr Nicholas Smith from Adelaide's Women's and Children's Hospital will present on Monday 30th of September at 9pm

Adelaide time (9.30pm for the East Coast, and 7.30pm for WA). To join the webinar, register here:  

2) Dr. Maria Luz Couce from Spain will present to European audiences, registration and details:

3) Dr Maria Escolar from Pittsburgh will present for US time zones details and registration:

For the full study details please see

Category: News