Denali MPS IIIA Clinical Study

Dec 13, 2023 | Clinical Trials, News, Research

December 12, 2023

Dear Sanfilippo syndrome Type A Community,

We are pleased to share that a Phase 1/2 clinical study of DNL126, an investigational enzyme replacement therapy designed to address the behavioral, cognitive, and physical symptoms in Sanfilippo syndrome Type A (MPS IIIA), is screening participants.  This study is currently active at one study site, UNC Children’s Research Institute, with a second site, UCSF, to begin shortly. Contact information for the sites include:

UNC Children’s Research Institute in the U.S (active and screening participants)

To learn more, you may contact: Lindsay Torrice, 919-966-1135 or

University of California-San Francisco (UCSF) in the U.S. 

To learn more, you may contact: Ella Rushing (study coordinator) 510-428-3885, ext. 4785, or

We are in the early stage of investigating DNL126, and this Phase 1/2 study will be the first time this treatment is assessed in individuals with MPS IIIA. Therefore, we are first focused on enrolling a small number of participants to understand the appropriate dose of DNL126 and to evaluate its safety.

We are striving to identify the most effective and timely path to advance DNL126 as a potential treatment for the MPS IIIA community. In pursuit of this, we continually assess our clinical development plans, and may implement modifications in the future to optimize our approach. We are committed to keeping this community apprised of program developments. 

Once details of this study are available at we will share this information.  You may also contact our patient advocacy team at  We are grateful for our collaboration with the MPS IIIA community, and for the participation and commitment of the families who participate in these important studies.

Thank you,

The Denali Team

 DNL126 is an investigational drug and is not approved by any Health Authority.