Gene Therapy Webinar

On Wednesday 16th September 2020, our gene therapy webinar discussed the new gene therapies being developed for NKH and ASA, and future developments which may also be suitable for those living with other conditions including MSUD, PFIC2, MMA, PA and OTC. Please see the webinar, including details of the talks given below.

Gene Therapy Webinar Topics

Elin Haf Davies, Chair, Metabolic Support UK
Introduction to Metabolic Support UK, and the purpose of the webinar

Section 1 | Concepts of Gene Therapy
Simon Waddington, Professor of Gene Therapy, University College London

  • What is gene therapy
  • Overview of core concepts

Simon has been working in the field of gene therapy for more than twenty years, mainly on the development of treatments for Inherited Genetic Diseases. In the mid-2000s he was developing treatments for haemophilia. As the field advanced, he started to witness how gene therapy could be applied to severe childhood genetic diseases.

Section 2 | Gene Therapy Technologies
John Counsell, Senior Research Fellow, University College London

  • Different therapies under development
  • Pros and cons of each approach in context of Inherited Metabolic Disorders (specifically liver applied)
  • Possible use of lentivirus

John is a Senior Research Fellow based at UCL Great Ormond Street Institute of Child Health. He specialises in engineering viral vector technologies for treating Inherited Metabolic Diseases by gene therapy.

Section 3 | Urea Cycle Disorders
Julien Baruteau, MRC Clinician Scientist, University College London

  • Gene therapies under development for ASA (model for other UCDs)
  • Status of lentivirus work

Julien is a MRC Clinician Scientist Fellow based at UCL Great Ormond Street Institute of Child Health and a consultant at Great Ormond Street Hospital. His work focusses on designing novel therapies for Inherited Metabolic Diseases.

Section 4 | Non-Ketotic Hyperglycinemia
Nick Greene, 
Professor of Developmental Neurobiology, University College London

  • NKH gene therapy programme
  • How liver rescue could help

Nick is Professor of Developmental Neurobiology at UCL Great Ormond Street Institute of Child Health, where he has led a research group since 2001. Nick’s group focus on development and disease of the nervous system using a range of experimental approaches. Over recent years he has particularly worked on disorders of the glycine cleavage system which he will talk about today.

Section 5 | Clinical Translation
Paul Gissen, Clinical Professor and Honorary Consultant in Paediatric Metabolic Diseases, University College London

  • How do gene therapies get to clinic
  • Which gene therapies are close
  • What should we expect from the projects that we heard about, and the timeframes

Paul is a Clinical Professor of Paediatric Metabolic Medicine at UCL Great Ormond Street Institute of Child Health and Consultant at GOSH.  He has 25 years clinical experience and leads both laboratory projects and clinical trials .


This webinar was in collaboration with Joseph’s Goal.