Clinical Trial


Phase 1 study
Testing the safety and tolerability of RO7248824, administered via IT injection.


RO7248824 is an antisense oligonucleotides (ASO) which aims to unsilence UBE3A production.


Participant Requirements

Below is a preliminary list of inclusion criteria.
See the full list of eligibility criteria on the clinical trials website.

  • Diagnosis of Angelman syndrome
  • Between 1 and 12 years old
  • Stable medical status for 4 weeks prior to screening visit
  • Ability to tolerate blood draws
  • Able to undergo LP and IT injection, under sedation or anesthesia 3 times during an 8-week period

Locations & Contacts

Los Angeles, CA
UCLA Neuropsychiatric Institute
(323) 361-2471

San Diego, CA
Rady Children’s Hospital – San Diego
Lynne Bird, MD

Chicago, IL
Rush University Medical Center
Kathryn Kudlacz

Carrboro, North Carolina
University of North Carolina
Hannah Riehl

Houston, TX
Baylor College of Medicine/ Texas Children’s Hospital
Beverly Feldman

New York, NY
Columbia University Irving Medical Center
Jennifer Bain, MD


Roma, Lazio, Italy
Ospedale Pediatrico Bambino Gesù; Dip. Neuroscienze e Neuroriabilitazione

Rotterdam, Netherlands
Erasmus MC / location Sophia Kinderziekenhuis

Barcelona, Spain
Hospital Sant Joan De Deu
Esplugues De Llobregas 

Sabadell, Barcelona, Spain
Corporacio Sanitaria Parc Tauli

Sevilla, Spain
Hospital Universitario Virgen del Rocío

More Information and Screening Enrollment

Find more information on the Roche website.

More details on

Questions about this study and enrollment interest can be directed to:
888-662-6728 (US and Canada) or

Reference Study ID Number: BP41674