P037: Design of a multi-center randomized phase 3 clinical trial (HURCULES) evaluating OTL-203 in MPS-IH vs allogeneic hematopoietic stem cell transplantation

• Paul Harmatz,
• Robert Wynn,
• Ashish Gupta,
• Sandhya Kharbanda,
• Caroline Lindemans,
• Rebecca Ahrens-Nicklas,
• Peter van Hasselt,
• Troy Lund,
• Timothy Olson,
• Francesca Tucci,
• Leonie Martin,
• Nathalie Boeglin,
• Jean Brooks,
• Su Syonmez,
• Laura Campbell,
• Simon Jones,
• Paul Orchard,
• Maria Ester Bernardo

Affiliations

Paul Harmatz

UCSF Benioff Children’s Hospital, San Francisco, CA

Robert Wynn

Manchester University NHS Foundation Trust, Royal Manchester Children’s Hospital, Manchester, UK

Ashish Gupta

University of Minnesota Department of Pediatrics, Minneapolis, MN

Sandhya Kharbanda

UCSF Benioff Children’s Hospital, San Francisco, CA

Caroline Lindemans

Princess Máxima Center for Pediatric Oncology, Utrecht University, The Netherlands

Rebecca Ahrens-Nicklas

The Children’s Hospital of Philadelphia, Philadelphia, PA

Peter van Hasselt

Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht, The Netherlands

Troy Lund

University of Minnesota Department of Pediatrics, Minneapolis, MN

Timothy Olson

The Children’s Hospital of Philadelphia, Philadelphia, PA

Francesca Tucci

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, Italy

Leonie Martin

Orchard Therapeutics, London, UK

Nathalie Boeglin

Orchard Therapeutics, London, UK

Jean Brooks

Orchard Therapeutics, London, UK

Su Syonmez

Orchard Therapeutics, London, UK

Laura Campbell

Orchard Therapeutics, London, UK

Simon Jones

Manchester University NHS Foundation Trust, Royal Manchester Children’s Hospital, Manchester, UK

Paul Orchard

University of Minnesota Department of Pediatrics, Minneapolis, MN

Maria Ester Bernardo

San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, Italy