Sanfilippo Syndrome Type B


The Transpher B Study: A Phase 1/2 Clinical Trial of ABO-101

An open-label, dose-escalation clinical trial assessing the safety of one-time gene therapy ABO-101 for patients with Sanfilippo syndrome type B (MPS IIIB).


Enrollment Closed

Can my child participate?

Who is eligible to participate in the study?

Key criteria for eligible patients include the following. A full list of inclusion and exclusion criteria are available here.

  • Diagnosis of MPS IIIB
  • Birth to 2 years of age or 2+ years with a Development Quotient ≥60
  • Evidence of neurologic dysfunction

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.
  • Additional visits may be required for eligible patients receiving long-term follow-up care.

About the Study

Sanfilippo syndrome type B, or MPS IIIB, is caused by a build-up of toxic sugars called glycosaminoglycans (GAGs) because of a mutation in the gene encoding the NAGLU enzyme. The study involves a one-time intravenous administration of ABO-101 using an adeno-associated virus vector (AAV) to deliver a normal copy of the NAGLU gene. The delivery of the normal NAGLU gene may allow cells to produce the NAGLU enzyme responsible for breaking down GAGs. After treatment, patients are followed for 24 months to assess safety and efficacy.


For more information about enrolling in our clinical trials, contact us at

Locations & Contacts

Nationwide Children's Hospital

Columbus, Ohio
United States
Kristen Brown:

Hospital Clínico Universitario de Santiago

Santiago De Compostela
Maria Luz Couce, MD:
Maria Jose de Castro, MD:

Armand-Trousseau Hospital

Bénédicte Héron, MD:
Claudia Revelli, MD:

The safety and efficacy of the investigational use of this product has not been determined. There is no guarantee that the investigational use listed will be filed with and/or approved for marketing by a regulatory agency.