Clinical Working Party

May 2023: Where is gene therapy for PID?

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ESID Grand Rounds Webinar May Website 949x315

Date: Tuesday, 30 May 2023, 17:00 CEST

Please, click here to view the webinar video. 


Where is gene therapy for PID?


The webinar will cover various topics related to gene therapy for PID.

After participating in the webinar, participants should be able to:
- Understand the diagnostic evaluation for X-SCID after positive TREC newborn screening.
- Recognize the curative treatment options for X-SCID.
- Understand the impact of pre-gene therapy conditioning on the immune outcome.
- Understand the underlying genetic basis of X-linked severe combined immunodeficiency.
- Gain knowledge on how gene editing technologies work and how they can be applied to PID.
- Understand the advantages and disadvantages of gene addition compared to gene editing in a clinical context.
- Have an overview of how other advances such as non-genotoxic conditioning and in vivo correction may make gene therapy an even more attractive therapeutic approach.



Siobhan v2

  • Moderator: Siobhán Burns
    Professor · University College London

Dr Burns is a clinical academic with an interest in primary immunodeficiency disorders (PID). Originally trained in paediatric immunology, she has a specialist interest in young adults with immunodeficiency. Her research group focuses on understanding the underlying causes for PID.

Sung v2

  • Sung-Yun Pai
    MD · Center for Cancer Research, National Cancer Institute, National Institutes of Health

Sung-Yun Pai MD is a physician-scientist dedicated to the study of inborn errors of immunity and their treatment with allogeneic hematopoietic stem cell transplantation and autologous gene therapy using viral vectors. She received her AB and MD degrees from Harvard, completed training in pediatrics and pediatric hematology-oncology at Boston Children’s Hospital/Dana-Farber Cancer Institute, and was faculty there for 19 years. She is now a tenured Senior Investigator and Chief of the Immune Deficiency Cellular Therapy Program at the National Cancer Institute, National Institutes of Health.


  • Carsten Speckmann
    Assistant Professor · University Freiburg Medical Center

Carsten Speckmann is a senior lecturer at the Faculty of Medicine of the Albert Ludwig University Freiburg, Germany. He works as a consultant for Pediatric Immunology and Hematopoietic Stem Cell Transplantation (HSCT) at the Freiburg University Medical Center’s Department for Pediatric Hematology/Oncology and the Center for Chronic Immunodeficiency (CCI). His research group has a focus on natural history studies for inborn errors of immunity (IEI) treatable by HSCT and/or targeted therapies. He is the speaker of the German Pediatric Working Party (API) Screening Committee (AG Screening) and has accompanied the political process of SCID screening implementation in Germany over the past 10 years.


  • Thomas Fox
    Postdoctoral Researcher and Academic Clinical Fellow · University College London

Dr. Thomas Fox is a clinical academic hematologist based at University College London. His clinical interest is in bone marrow transplantation for inherited primary immunodeficiencies. He completed his Ph.D. at UCL in March 2022 under the supervision of Professors Emma Morris, Claire Booth, Siobhan Burns, and David Sansom. During his Ph.D., he developed a gene editing approach for CTLA-4 insufficiency. His work was recently published in Science Translational Medicine, and he was awarded the UCL Dean’s Research Prize for Medicine in recognition of this work. He is currently working as a postdoctoral researcher in the Morris/Burns lab exploring in vivo delivery of gene editing machinery.

Claire v2

  • Claire Booth (Panel member)
    Professor · UCL Great Ormond Street Institute of Child Health/Great Ormond Street Hospital, London

Prof Claire Booth, MBBS Ph.D. is a Gene Therapist and Paediatric Immunologist at UCL Great Ormond Street Hospital Institute of Child Health in London and leads the clinical stem cell gene therapy program. Claire works as a clinical academic leading an expanding number of gene therapy clinical trials at Great Ormond Street Hospital which treats patients with immune deficiencies, and hematological and metabolic disorders. Her lab group is focused on developing novel therapies for immune system disorders using both gene therapy/gene editing and targeted small molecules. As an attending physician, she oversees the clinical management of patients with immune deficiencies, including hematopoietic stem cell transplantation, and maintains a strong interest in HLH disorders. Claire is currently a board member of ESGCT and Chair of the International Committee of ASGCT. She holds an honorary position at Boston Children’s Hospital/Dana Farber Cancer Institute and Harvard Medical School.

Arjan v2

  • Arjan Lankester (Panel member)
    Professor·Leiden University

Professor Arjan Lankester is professor in Pediatrics and Pediatric Stem Cell Transplantation. He is also head of the JACIE-accrediated pediatric stem cell transplantation program, chair of the Working Party Inborn Errors of the European Society for Blood and Marrow Transplantation (EBMT) and chair of the Working Party Inborn Errors of the European Society for Immune Deficiencies (ESID).

Krzysztof v2

  • Krzysztof Kalwak (Panel member)
    Professor · Wroclaw Medical University

Prof. Krzysztof Kalwak was born on 10 October 1969. In high school (VII LO in Wrocław) he was a scholarship holder of the National Children's Fund. From 1988 to 1994, he studied at the Faculty of Medicine of the Piasts of Silesia Medical University in Wrocław and graduated with honours. The next stage in his scientific career was his doctoral studies, followed by the defence of his doctoral thesis (titled "Early and late reanimation of the disease. Early and late immunological reconstitution in children after hematopoietic stem cell transplantation with consideration of immunomodulation and immunotherapy") written under the supervision of Prof. Janina Bogusławska-Jaworska . In 2000, he became a doctor of medical sciences. In 2006, he was awarded a postdoctoral degree in medical sciences. The dissertation topic was: "Selected issues in the immunology of the post-transplant period in children undergoing haematopoietic cell transplantation". He became professor on 19.02.2014. After two outstanding predecessors (Prof. Janina Boguslawska-Jaworska and Prof. Alicja Chybicka), he became the new head of the Department of Paediatric Bone Marrow Transplantation, Oncology and Haematology on 1.10.2021.

May 30, 2023 17:00 CEST in Amsterdam, Berlin, Rome, Stockholm, Vienna