NIHR GOSH BRC Gene Therapy in Paediatrics 2025

There are four sessions on the programme featuring talks and panel discussions. There will also be plenty of networking opportunities during the coffee break, lunch, and networking session. Confirmed speakers and panel include:

• Professor Paul Gissen, Theme Lead of the GOSH BRC Gene, Stem and Cellular Therapies Theme, Clinical Professor of Paediatric Metabolic Medicine at UCL Great Ormond Street Institute of Child Health
• Professor Giovanni Baranello – Professor of Paediatric Neuromuscular Disorders
• Professor David Long – Deputy Theme Lead of the GOSH BRC Gene, Stem and Cellular Therapies Theme, Professor of Paediatric Nephrology at UCL Great Ormond Street Institute of Child Health
• Professor Claire Booth – Deputy Theme Lead of the GOSH BRC Gene, Stem and Cellular Therapies Theme, Professor of Gene Therapy and Paediatric Immunology at UCL Great Ormond Street Institute of Child Health
• Dr Tom Whittaker – Research Fellow at UCL Great Ormond Street Institute of Child Health
• Dr Amy Geard – GOSH BRC Gene, Stem and Cellular Therapies Research Associate Lead Scientist at UCL Great Ormond Street Institute of Child Health
• Dr Julien Baruteau – MRC Clinician Scientist Fellow and Group Leader and Senior Research Fellow at UCL Great Ormond Street Institute of Child Health, Consultant in Metabolic Medicine at Great Ormond Street Hospital for Children
• Dr Christos Georgiadis – Senior Research Fellow at UCL Great Ormond Street Institute of Child Health

Each session is structured to provide comprehensive information on gene therapy for clinicians, with each session consisting of a speaker and an expert panel to answer and discuss questions you might have around the topic.

Session 1: Demystifying gene therapy technology

• The Science of Gene Therapy Technology.
• Gene Therapy application in genetic disorders.

Session 2: Immunodeficiencies and Gene Therapy

• Gene Therapy in treating inherited immune system disorders.
• Mechanisms behind immunodeficiencies, treatment strategies, and applications of gene therapy to improve immune function in children.

Session 3: Cancer and Gene Therapy

• Gene Therapy in treating children with cancer.
• Techniques modifying genes to fight tumour cells such as CAR-T cell therapy.

Session 4: Application of in vivo AAV based gene therapy.

• Adeno-associated viruses (AAV) to deliver therapeutic genes in conditions.
• Safety and effectiveness of AAV-based treatments in paediatrics.

To apply, please complete a short (Expression of interest (EOI) Form and submit to brc@gosh.nhs.uk by Monday 9 December 2024, 23:59. Submission after this date are welcome.

For anyone who is interested in attending but circumstances make it challenging to access, please contact us to discuss.

We are very much looking forward to bringing together paediatricians, paediatricians-in-training, GPs with specialist paediatric interests, and allied health professionals from across the UK. All applications are welcome. We have space for around sixty (60) attendees, priority for places will be given to those who are.

• Paediatricians
• GPs with an interest in paediatrics
• Paediatricians-in-training
• Specialist, Associate Specialist, and Specialty (SAS) doctors
• Locally Employed Doctors (LEDs)
• Allied Health Professionals in paediatrics

The event steering committee will review the EOI forms with the selection criteria:

• Likelihood the attendee will benefit from the event programme.
• Balanced representation of delegates regarding profession and career stage.

We will contact all applicants who have expressed an interest in attending the event by Mid December (or Early January 2025 for submissions after 9th December 2024) to confirm whether they have a place.

Please contact brc@gosh.nhs.uk if you have any questions.