NIHR GOSH BRC Gene Therapy in Paediatrics 2026

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 Bobby Gaspar - Clinical Professor of Paediatrics and Immunology at the UCL Great Ormond Street Institute of Child Health & Co-Founder of Orchard Therapeutics
• Professor Adrian Thrasher - Professor of Paediatric Immunology at the UCL Great Ormond Street Institute of Child Health, NIHR Senior Investigator Emeritus & Co-Founder of Orchard Therapeutics
• Professor Manju Kurian - Professor of Neurogenetics at UCL Great Ormond Street Institute of Child Health & Academic Paediatric Neurologist at Great Ormond Street Hospital
• Professor Robert Wynn - Consultant Paediatric Haematologist & Director of Paediatric Bone Marrow Transplant Programme at the Manchester University NHS Foundation Trust
• Professor Haiyan Zhou - Professor of Genetic Medicine at the UCL Great Ormond Street Institute of Child Health & Director of MRC/NIHR Rare Disease Node on Nucleic Acid Therapy
• Dr Stephanie Grünewald - Consultant Metabolic Paediatrician and Clinical Lead at Great Ormond Street Hospital & Senior Lecturer at the UCL Great Ormond Street Institute of Child Health
• Dr Robert Henderson - Consultant Paediatric Ophthalmologist, Adult & Paediatric Vitreoretinal Surgeon at Moorfields Eye Hospital and Great Ormond Street Hospital
• Dr Kristian Aquilina - Consultant Paediatric Neurosurgeon & Surgical Lead for Neuro-oncology at Great Ormond Street Hospital
• 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 Sara Benedetti - Non-Clinical Lecturer in Gene Therapy 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: Historical Perspective on Gene Therapy at GOSH

• History of gene therapy
• Translational pathway for developing gene therapy from bench to bedside

Session 2: Approved Gene Therapies for Brain Disorders

• Approved in-vivo and ex-vivo gene therapies for paediatric neurometabolic disorders
• Safety and effectiveness of In-vivo and ex-vivo gene Therapies

Session 3: RNA Therapies in Paediatrics

• Innovative and emerging RNA Therapies for paediatric Care
• Antisense Oligonucleotides (ASOs) and small interfering RNAs (siRNAs) for paediatric care

Session 4: Adverse Events and Horizon Scanning in Gene Therapy for Paediatrics

• Gene therapy safety and adverse events
• Emerging gene therapy research

To apply, please complete a short (Expression of interest (EOI) Form and submit to brc@gosh.nhs.uk by Monday December 08 2025, 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 seventy (70) 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 2026 for submissions after 8 December 2025) to confirm whether they have a place.

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