UCL Advanced Therapies Symposium 2026 Programme

Maximise your symposium experience with our guide. Explore the agenda, speaker bios, poster details, floor map, and sponsor highlights, plus essential on-site information for all attendees.

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Important updates - transportation

There is a planned tube strike from 21–22 April (12:00 Tuesday to 12:00 Wednesday). The event will remain in person. If you will arrive by train, the closest stations to come on foot are Euston, King’s Cross Station or St Pancras Station. The closest Elizabeth line stations to the venue are Tottenham Court Road or Farringdon, There are multiple bus routes that connect to the venue too.
Please be aware that your journey may be disrupted and allow extra time to get to your destination. You can visit the Transport for London or National Rail websites, or use apps such as the Citymapper app, which might be useful for planning alternative types of transport such as bicycle and scooter hire, walking, buses etc.
Please do keep following the news and the Transport for London website for the latest updates.

When and where

Date: Wednesday 22 April 2026
Time: Registration 09.00-9.20; Seminar 9.20-17.30; Networking Reception 17.30-19.00
Venue: Kennedy Lecture Theatre, UCL Great Ormond Street Institute of Child Health, 30 Guilford Street London WC1N 1EH (Google Map, Local area map and transportation)

Welcome

Welcome to the UCL Advanced Therapies Symposium, brought to you by the UCL Translational Research Office (TRO) and the Therapeutic Innovation Networks (TINs).

Over the past year, UCL has continued to strengthen one of the most extensive ATMP pipelines globally, with over 100 projects in development, two launched products, including one now available on the NHS. Our academic research portfolio continues to be bolstered by over £70M of publicly funded Phase I/II and preclinical projects.

Our clinical strength remains a national leader. Close to 44% of UK academically sponsored and non-profit ATMP trials and over 50% of commercial ATMP trials are delivered through UCL partner hospitals*, underscoring the depth and capability of our translational and clinical delivery infrastructure.

Today’s Programme

The 2026 symposium highlights UCL’s multidisciplinary expertise across (1) CAR T therapies for autoimmune conditions, (2) gene therapy for rare diseases, (3) cell therapy innovation and (4) innovation and commercialisation.

Panel discussions will explore commercialisation pathways with industry and investor leaders, as well as the expansion of CAR T therapies to additional disease areas, featuring a patient perspective. Talks and poster presentations by early career researchers will showcase emerging ideas shaping the future of therapeutic innovation.

Celebrating UCL200

As UCL marks its bicentennial year, UCL200, we celebrate 200 years of discovery, impact and partnership. This symposium reflects that legacy—demonstrating how scientific excellence, engineering innovation and clinical expertise combine to deliver tomorrow’s therapies. We invite you to join us in shaping the next chapter of therapeutic advancement for the benefit of patients and society.

Thank you to all our speakers, contributors and sponsors for making today’s event possible. Please visit the exhibition desks during breaks and take the opportunity to connect with colleagues from across the ecosystem.

We hope you enjoy the programme and look forward to engaging with you throughout the day.

Professor Simon Waddington
Professor of Gene Therapy, Maternal and Fetal Medicine, UCL
Chair of UCL Therapeutic Innovation Networks (TINs) – Cell and Gene Therapy

*Data from Cell and Gene Therapy Catapult, ATMP Clinical Trials Database 2025.

Agenda

View here.

Speakers, section chairs and panellists

In order of apperance:
Session 1 Chair: Prof. Simon Waddington Professor of Gene Therapy, Maternal & Fetal Medicine, UCL; Chair of the UCL Therapeutic Innovation Networks (TINs) – Cell and Gene Therapy Simon Waddington is a Professor of Gene Therapy at UCL. He leads a team developing translational gene therapy for childhood inherited genetic diseases including neurodegenerative diseases, inherited epilepsy and metabolic diseases. I chair the Cell and Gene Therapy Therapeutic Innovation Network of the UCL Translational Research Office. With Professor Manju Kurian, Professor Paul Gissen and Ahad Rahim, he has co-founded a company, “Bloomsbury Genetic Therapies”. This has received £5 million of seed funding from Albion VC/UCL Tech Fund. The company will progress four gene therapy programmes to the clinic. They are gene therapy for Ornithine Transcarbamylase Deficiency, Infantile Neuronaxonal Dystrophy, Niemann Pick C and Dopamine Transporter Deficiency Syndrome. The company is now in the process of raising Series A funding. He and his team have been funded by the UK Medical Research Council Developmental Pathway Funding Scheme and LifeArc for translational projects to treat Dravet Syndrome and Dopamine Transporter Deficiency Syndrome. They have licensed one of their projects to a gene therapy company with whom we have a £1.5 million sponsored research agreement in place. Collaborating with Prof Tristan McKay (Manchester Metropolitan University) he obtained a European Research Council starter grant of €1.5 million from the European Research Council to develop the means to quantify signalling pathways in diseased organs and tumours continually and non-invasively. Working alongside the teams of Professors Andy Baker (University of Edinburgh) and John McVey (University of Surrey) they elucidated one of the fundamental mechanisms by which adenovirus vectors transduce cells. He has several long-standing international collaborations. These include Drs Jerry Chan, Duke-NUS Graduate Medical School Singapore and Prof Patrick Arbuthnot, at the University of Witwatersrand, South Africa, where he holds an honorary chair. Prof. Paul Dalby, Co-Director of the EPSRC Future Targeted Healthcare Manufacturing Hub; Deputy Head of the Department of Biochemical Engineering at UCL Prof Paul Dalby's research focuses on technologies to improve the design and formulation of therapeutic proteins and gene therapy vectors, to improve their manufacture, stability, delivery and function. He has pioneered smart directed evolution approaches that accelerate evolution in the lab. Recent work has used molecular dynamics simulations and computational protein design tools to guide stability and function modifying mutations into proteins and viral vectors.
Prof. Charles Knowles Consultant colorectal surgeon and Chief Academic Officer at Cleveland Clinic London; Professor of Surgery at Queen Mary University of London Professor Charles Knowles's work centres on the development and evaluation of advanced technologies for managing chronic functional and neuromuscular disorders of the bowel and pelvic floor, with particular expertise in neuromodulation and emerging cell‑based therapies. Recognised internationally for his leadership in constipation, incontinence, and gastrointestinal neuromuscular disease, he also holds honorary professorships at UCL and the University of Antwerp. In addition to his clinical and academic roles, he is an inventor, Chief Medical Officer of Amber Therapeutics, and an accomplished author and musician. In collaboration with: Prof. Richard Day Professor of Regenerative Medicine Technology at UCL and Director of the Centre for Precision Healthcare Professor Richard Day's research focuses on the discovery, development and clinical translation of novel biomedical engineering technologies. He is the inventor of TIPS microparticles, a versatile platform technology with applications in tissue engineering, drug delivery, and regenerative medical devices. Together, Richard and Charles have collaborated on several pioneering clinical trials, including UCL’s first regulated trial of a Class III biomaterial‑based medical device, as well as the EU‑funded AMELIE project, which is advancing a groundbreaking cell‑based therapy for faecal incontinence across 13 organisations in nine countries.
Panel Discussion 1 Chair: Dr Anne Lane CEO, UCL Business Anne started her career as a geneticist and completed a PhD at UCL before teaching and researching at Harvard University and Medical School in the US. Anne moved into university technology transfer at UCL in 2000, establishing UCL Business Ltd in 2006 as a wholly owned subsidiary of the university. She has been CEO of UCLB since 2019. Anne has worked on the establishment of two UCL Technology Funds raising in excess of £120m to support spinout businesses which in turn raised almost £3 billion in external investment, 5 having listed on NASDAQ. Their impacts include a ‘one-shot’ lifetime treatment for haemophilia A, AI-led climate change solutions and revolutionary diagnostic technologies in breast cancer.
Attilio Bondanza Vice President, Immunology Cell Therapy, AstraZeneca
Dr Sven Kili Partner, Saisei Ventures Dr. Sven Kili is a respected KOL in healthcare and biotechnology, boasting over 20 years of experience. He began as a surgeon in the NHS, where he gained valuable insights into patient care. Transitioning to Cell and Gene Therapies (CGT), Sven played key roles in leading the development, approval and commercialisation of both a cell therapy and the first ex-vivo gene therapy. With expertise across all stages of CGT development, he has built companies and teams in both large pharma/ biotech and startups. Sven is a partner at Saisei Ventures, a boutique therapeutic venture investment firm focused on creating and growing innovative companies, including with a focus on the Japanese CGT ecosystem. He also heads his consulting firm, Sven Kili Consulting Ltd., oQering strategic guidance to organizations in the biotech and healthcare sectors. Most recently he served as the Chief Development OQicer for CCRM (Centre for Commercialization of Regenerative Medicine) and OmniaBio. Sven actively contributes to industry boards and committees, including the BIA CGTAC, Innovation Hubs for Gene Therapy, ISCT, and ARM. Sven holds a visiting chair at UCL (University College London), where he heads the steering committee for a post-grad degree in Cell & Gene Therapy manufacturing and commercialisation. His dedication to advancing regenerative medicine and CGT is reflected in his multifaceted roles and ongoing eQorts to shape the future of healthcare through innovation and strategic collaboration.
Dr Mary Canning Partner, Epidarex Capital Mary Canning is a Partner with Epidarex Capital, a venture fund that specializes in building innovative life science companies in the UK and US. Mary serves on the Board of Directors of several Epidarex portfolio companies and is also an independent director of the University of Glasgow’s investment subsidiary, GU Holdings. She has 20 years of experience in the commercialization of early-stage health science technologies, having worked with technology transfer offices in leading UK academic institutions prior to joining Epidarex in 2013. She has a Ph.D. in Molecular Biology from the University of Edinburgh and undertook postdoctoral research at the University of Glasgow.
Session 2 Chair: Prof. Rajvinder Karda Professor in Gene Therapy, EGA Institute for Women's Health, UCL Prof. Karda and her research team are focusing on developing novel gene therapy and RNA editing treatments for early onset, incurable genetic neurological and neurometabolic diseases. Dr Karda is a member of the scientific advisory committee for Dravet Syndrome Foundation, Spain and Cure DHDDS Charity, UK. She is also on the executive committee of British Gene and Cell Therapy Society. She has received funding from, MRC DPFS, LifeArc and GOSHCC to develop novel genetic therapies for genetic childhood neurological diseases.
Early career researcher speaker: Dr Sara Aguti Senior Research Fellow, Neurodegenerative Diseases, UCL Dr Sara Aguti developed an interest in identifying effective treatments for neuromuscular and neurological diseases during her MSc at the University of Siena. She completed her PhD under the supervision of Francesco Muntoni and Haiyan Zhou, focusing on the development of RNA-based therapies for collagen VI–related myopathies. Following her postdoctoral work, she moved to MiNA Therapeutics to explore a different RNA-based therapeutic strategy based on the direct targeting of DNA to achieve gene upregulation. In 2023, Sara was appointed as a Senior Research Fellow at the Genetic Therapy Accelerator Centre (GTAC), where she focuses on developing RNA therapies using multiple strategies to correct disease-causing mutations in both neurological and neuromuscular disorders. In addition, her research aims to improve the targeted delivery of RNA therapeutics to specific tissues and cell types, one of the major bottlenecks for the clinical translation of RNA-based therapies.
Early career researcher speaker: Dr George Hall Research Fellow, Sergi Castellano Lab, UCL Great Ormond Street Institute of Child Health George is a postdoctoral research fellow in the lab of Sergi Castellano at the UCL Great Ormond Street Institute of Child Health. Following his PhD in Computer Science, he moved to biology to develop computational methods to address unmet needs in translational clinical research. His research uses single-cell and spatial transcriptomics to compare biological structures, recently assessing the biological recapitulation of bioengineered oesophagi and organoid models of the developing midbrain. He is currently developing methods to allow the more sensitive comparison of samples profiled with single-cell transcriptomics and to generate realistic synthetic spatial transcriptomic datasets in settings where few reference examples are available. He co-organises the Institute of Child Health's Computational Biology Club, a forum for discussion for recent computational methods and problems being faced by researchers.
Early career researcher speaker: Dr Maria Toms Research Fellow, Moosajee lab, The Francis Crick Institute and Institute of Ophthalmology, UCL Maria is a postdoc research fellow in Professor Mariya Moosajee’s group at the UCL Institute of Ophthalmology and the Francis Crick Institute. She completed her PhD in Molecular Genetics at UCL in 2018. Her research investigates the causes of genetic eye diseases and strategies to treat them, with a focus on non‑viral gene therapies.
Prof. Nikki Robertson Professor of Perinatal Neuroscience, UCL; Consultant Neonatologist at University College London Hospitals (UCLH) Nikki is professor of perinatal neuroscience at UCL, working also as consultant neonatologist at UCLH. For the last 23 years at UCL, Nikki has led the preclinical development of new therapies for neonatal brain injury, recently taking melatonin into a phase I study in babies with hypoxic ischaemic encephalopathy (HIE). HIE is a significant burden worldwide with devastating lifelong health, social, and educational impacts. The only current treatment is therapeutic hypothermia which is only partially effective. In the last 5 years, Nikki and her team have focused on Wharton Jelly Mesenchymal Stromal Cell (WJ-MSC) therapy, assessing the safety and efficacy of intranasal administration of WJ-MSC after hypoxia ischaemia in a large preclinical model. Using clinically relevant MR and EEG biomarkers, these data suggest that combination therapy may soon be ready for early phase clinical trials in babies with HIE.
Session 3 Chair: Prof. Emma Morris BA MA MB BChir PhD FRCP FRCPath FMedSci Dean, Faculty of Medical Sciences, UCL; Professor of Clinical Cell and Gene Therapy, UCL Institute of Immunity & Transplantation. Consultant Haematologist (Haematopoietic Stem Cell Transplantation and Gene Therapy) at UCLH & Royal Free NHS FTs Professor Morris is a clinician scientist, clinically trained in haematology and allogeneic HSCT. Her current clinical practice is in HSCT, cellular and gene therapies for haematological malignancies and inherited immune deficiencies. She has developed and led the national allogeneic HSCT centre for adults with primary immune deficiencies (PID) at the UCL Centre for Immunodeficiency and co-ordinates an international MDT. Her basic science research focus is on T cell immunology, inherited immunodeficiencies, gene therapy and gene editing. She leads a group interested in altering both the specificity and the function of gene-modified immune cells and stem cells. More recent research is focused on developing gene editing techniques as treatments for patients with PID. This research spans the entire translational pathway from animal models to in vitro experimental immunology to Phase I ‘first time in man’ clinical studies. Alongside her role as Dean, Faculty Medical Sciences, Emma is the Director of the NIHR UCLH/UCL Biomedical Research Centre Inflammation, Immunity and Immunotherapeutics (III) Theme. She is patron of the charity Haematology Cancer Care (HCC) which raises money for UCLH. Professor Morris is a scientific founder of UCL Spin-out, Quell Therapeutics Ltd, which is developing gene-engineered regulatory T cells for transplant tolerance and autoimmune indications. She was elected to the Academy of Medical Sciences in 2020.
Prof. Despina Eleftheriou Professor of Paediatric Rheumatology, UCL; Consultant, UCL Great Ormond Street Institute of Child Health Professor Despina Eleftheriou leads a translational research programme focused on rare inflammatory diseases in children. Her work integrates genomics, biomarker discovery, and early-phase clinical trials to develop targeted therapies and improve patient outcomes. Her research includes the development of advanced therapies, including CAR T-cell approaches for autoimmune diseases and gene-based strategies such as lentiviral stem cell gene therapy and gene silencing for autoinflammatory conditions. Professor Eleftheriou has led international studies, and works closely with multidisciplinary and industry partners to translate innovative therapies into clinical application.
Dr Frederick Vonberg Clinical Fellow in CAR-T Neurology, UCL Queen Square Institute of Neurology and University College London Hospitals (UCLH); Neurologist at the National Hospital for Neurology and Neurosurgery Freddie is a Neurologist at the National Hospital for Neurology and Neurosurgery and Imperial College Healthcare Trust and a Clinical Fellow in CAR-T Neurology at the Queen Square Institute of Neurology and UCLH. His post-doctoral research, with Dr. Claire Roddie and Dr. Aisling Carr, explores the neurotoxic effects of Chimeric Antigen Receptor (CAR) T-cell therapy and the application of CAR-T to autoimmune neurological diseases. He is a founding member of the New and Emerging Oncotherapeutic Neurotoxicity (NEON) network. He undertook his doctoral work at Boston Children's Hospital, Harvard Medical School, and the Massachusetts Institute of Technology.
Katie Tinkler Patient Representative Mrs Katie Tinkler is one of the first three patients in the UK to receive a groundbreaking CAR T‑cell therapy for severe lupus through an NHS clinical trial led by University College London (UCL) and University College London Hospitals (UCLH) in 2024. A mother of three from Guildford, Katie has lived with lupus for more than 30 years, experiencing debilitating symptoms including extreme joint pain, fatigue, kidney disease and complications such as antiphospholipid syndrome. After decades relying on steroids, immunosuppressants and multiple additional medications as her health and immune function declined, Katie described joining the trial as “beyond exciting”, marking the first real possibility of long‑term remission. As a patient representative, Mrs Tinkler offers invaluable first‑hand insight into the lived experience of severe autoimmune disease and the profound impact that emerging therapies can have. Her perspective underscores the essential role of patients in shaping the future of advanced and potentially curative treatments.
Prof. Dimitri Michael Kullmann Professor of Neurology Clinical & Experimental Epilepsy, UCL Queen Square Institute of Neurology Prof. Kullmann’s interests span the fundamental mechanisms of synaptic transmission, the computational properties of small neuronal circuits, and alterations in neuronal and circuit excitability in epilepsy and other neurological disorders. Together with colleagues at the Institute of Neurology he has devised several gene therapies for pharmacoresistant epilepsy, and founded EpilepsyGTx, a UCL spinout company, to bring some of these to the clinic Prof. Claire Booth Mahboubian Professor in Gene Therapy and Paediatric Immunology, Head of Infection, Immunity and Inflammation Department, UCL; Deputy Theme Lead Gene, Stem and Cell Therapy, GOSH NIHR BRC; Cell & Gene Therapy Service Clinical Academic Lead; Consultant in Paediatric Immunology, Great Ormond Street Hospital Claire is a Professor of Paediatric Immunology and Gene Therapy at UCL Great Ormond Street Institute of Child Health, leading the stem cell gene therapy programme. She oversees gene therapy trials for immune deficiencies, haematological, and metabolic disorders, focusing on developing novel treatments for immune disorders. Claire has extensive experience in leading first-in-human clinical trials and translating research into clinical practice. As an attending physician, she manages patients with immunodeficiencies, including stem cell transplants, with a particular interest in HLH disorders. Claire is an elected board member of the European and American Societies of Gene and Cell Therapy and the European Society for Immunodeficiencies, holds an honorary position at Boston Children’s Hospital and Harvard Medical School, and co-founded the AGORA Foundation to improve access to gene therapies for ultra-rare diseases.
Prof. Paul Gissen Clinical Professor in Paediatric Metabolic Medicine and an Honorary Consultant at UCL Great Ormond Street Hospital for Children; NIHR Senior Investigator Paul Gissen is the Director Designate of the National Institute for Health and Care Research GOSH Biomedical Research Centre (NIHR GOSH BRC) and leads the Gene, Stem and Cellular Therapies. He is also the UK Chief Investigator for a number of industry and academic sponsored clinical trials of novel therapies. Professor Gissen obtained his medical degree from the University of Glasgow and trained in paediatrics at Manchester, Sheffield and Birmingham Children’s Hospitals specialising in inherited metabolic disorders. He undertook his PhD at Birmingham University where he identified genetic causes of several rare paediatric diseases. His research interests are in developing novel gene based therapies for Rare Paediatric Metabolic Disorders.
Professor Andrew Dick Director UCL-Institute of Ophthalmology; Duke Elder Professor of Ophthalmology, UCL Institute of Ophthalmology; Co-Director of National Health Institute for Research Biomedical Research Centre, Moorfields Eye Hospital and UCL-Institute of Ophthalmology, London Andrew qualified in medicine with a degree in Biochemistry (BSc (Hons)) from the University of London, and during his medical education he also undertook an MRC secondment in Biochemistry with Professor Coleman in Yale. Following completing MRCP, he entered ophthalmology residency and obtained his postgraduate research degree in Immunology in 1993 at the University of Aberdeen. He underwent an MRC Post-Doctoral Fellowship to work with Jon Sedgwick at the Centenary Institute of Cancer Medicine and Cell Biology in Sydney Australia. His clinical expertise is in medical and surgical management of retinal and inflammatory disease. His research spans basic and translational science including early and phase III trials in inflammation as related to autoinflammatory, autoimmune and degenerative retinal disease. Professor Dick is a Fellow of the Academy of Medical Sciences in the UK for his significant contribution to research and scholarship, in particular his development of molecular targets and biologic therapy for inflammatory eye disease. He was awarded the Alcon Research Institute Research award in 2011. His work has led to discoveries in ocular inflammatory disease and degenerative disease and co-founder of Cirrus Therapeutics. Prior to becoming Director of institute of Ophthalmology, the UCL-Institute of Ophthalmology, he was Director of Research for the Faculty of Medicine and Dentistry at University of Bristol. He has previously been Editor of British Journal of Ophthalmology, President of European Vision and Eye Research (EVER), Master of Oxford Ophthalmological Congress and currently Vice-President of ARVO.

In order of apperance:

Session 1 Chair:
Prof. Simon Waddington
Professor of Gene Therapy, Maternal & Fetal Medicine, UCL; Chair of the UCL Therapeutic Innovation Networks (TINs) – Cell and Gene Therapy

Simon Waddington is a Professor of Gene Therapy at UCL. He leads a team developing translational gene therapy for childhood inherited genetic diseases including neurodegenerative diseases, inherited epilepsy and metabolic diseases. I chair the Cell and Gene Therapy Therapeutic Innovation Network of the UCL Translational Research Office.

With Professor Manju Kurian, Professor Paul Gissen and Ahad Rahim, he has co-founded a company, “Bloomsbury Genetic Therapies”. This has received £5 million of seed funding from Albion VC/UCL Tech Fund. The company will progress four gene therapy programmes to the clinic. They are gene therapy for Ornithine Transcarbamylase Deficiency, Infantile Neuronaxonal Dystrophy, Niemann Pick C and Dopamine Transporter Deficiency Syndrome. The company is now in the process of raising Series A funding. He and his team have been funded by the UK Medical Research Council Developmental Pathway Funding Scheme and LifeArc for translational projects to treat Dravet Syndrome and Dopamine Transporter Deficiency Syndrome. They have licensed one of their projects to a gene therapy company with whom we have a £1.5 million sponsored research agreement in place. Collaborating with Prof Tristan McKay (Manchester Metropolitan University) he obtained a European Research Council starter grant of €1.5 million from the European Research Council to develop the means to quantify signalling pathways in diseased organs and tumours continually and non-invasively. Working alongside the teams of Professors Andy Baker (University of Edinburgh) and John McVey (University of Surrey) they elucidated one of the fundamental mechanisms by which adenovirus vectors transduce cells. He has several long-standing international collaborations. These include Drs Jerry Chan, Duke-NUS Graduate Medical School Singapore and Prof Patrick Arbuthnot, at the University of Witwatersrand, South Africa, where he holds an honorary chair.

Prof. Paul Dalby,
Co-Director of the EPSRC Future Targeted Healthcare Manufacturing Hub; Deputy Head of the Department of Biochemical Engineering at UCL

Prof Paul Dalby's research focuses on technologies to improve the design and formulation of therapeutic proteins and gene therapy vectors, to improve their manufacture, stability, delivery and function. He has pioneered smart directed evolution approaches that accelerate evolution in the lab. Recent work has used molecular dynamics simulations and computational protein design tools to guide stability and function modifying mutations into proteins and viral vectors.

Prof. Charles Knowles
Consultant colorectal surgeon and Chief Academic Officer at Cleveland Clinic London; Professor of Surgery at Queen Mary University of London

Professor Charles Knowles's work centres on the development and evaluation of advanced technologies for managing chronic functional and neuromuscular disorders of the bowel and pelvic floor, with particular expertise in neuromodulation and emerging cell‑based therapies. Recognised internationally for his leadership in constipation, incontinence, and gastrointestinal neuromuscular disease, he also holds honorary professorships at UCL and the University of Antwerp. In addition to his clinical and academic roles, he is an inventor, Chief Medical Officer of Amber Therapeutics, and an accomplished author and musician.

In collaboration with:
Prof. Richard Day
Professor of Regenerative Medicine Technology at UCL and Director of the Centre for Precision Healthcare

Professor Richard Day's research focuses on the discovery, development and clinical translation of novel biomedical engineering technologies. He is the inventor of TIPS microparticles, a versatile platform technology with applications in tissue engineering, drug delivery, and regenerative medical devices.

Together, Richard and Charles have collaborated on several pioneering clinical trials, including UCL’s first regulated trial of a Class III biomaterial‑based medical device, as well as the EU‑funded AMELIE project, which is advancing a groundbreaking cell‑based therapy for faecal incontinence across 13 organisations in nine countries.

Panel Discussion 1 Chair:
Dr Anne Lane
CEO, UCL Business

Anne started her career as a geneticist and completed a PhD at UCL before teaching and researching at Harvard University and Medical School in the US.

Anne moved into university technology transfer at UCL in 2000, establishing UCL Business Ltd in 2006 as a wholly owned subsidiary of the university. She has been CEO of UCLB since 2019. Anne has worked on the establishment of two UCL Technology Funds raising in excess of £120m to support spinout businesses which in turn raised almost £3 billion in external investment, 5 having listed on NASDAQ. Their impacts include a ‘one-shot’ lifetime treatment for haemophilia A, AI-led climate change solutions and revolutionary diagnostic technologies in breast cancer.

Attilio Bondanza
Vice President, Immunology Cell Therapy, AstraZeneca

Dr Sven Kili
Partner, Saisei Ventures

Dr. Sven Kili is a respected KOL in healthcare and biotechnology, boasting over 20 years of experience. He began as a surgeon in the NHS, where he gained valuable insights into patient care. Transitioning to Cell and Gene Therapies (CGT), Sven played key roles in leading the development, approval and commercialisation of both a cell therapy and the first ex-vivo gene therapy. With expertise across all stages of CGT development, he has built companies and teams in both large pharma/ biotech and startups.

Sven is a partner at Saisei Ventures, a boutique therapeutic venture investment firm focused on creating and growing innovative companies, including with a focus on the Japanese CGT ecosystem. He also heads his consulting firm, Sven Kili Consulting Ltd., oQering strategic guidance to organizations in the biotech and healthcare sectors. Most recently he served as the Chief Development OQicer for CCRM (Centre for Commercialization of Regenerative Medicine) and OmniaBio.

Sven actively contributes to industry boards and committees, including the BIA CGTAC, Innovation Hubs for Gene Therapy, ISCT, and ARM. Sven holds a visiting chair at UCL (University College London), where he heads the steering committee for a post-grad degree in Cell & Gene Therapy manufacturing and commercialisation. His dedication to advancing regenerative medicine and CGT is reflected in his multifaceted roles and ongoing eQorts to shape the future of healthcare through innovation and strategic collaboration.

Dr Mary Canning
Partner, Epidarex Capital

Mary Canning is a Partner with Epidarex Capital, a venture fund that specializes in building innovative life science companies in the UK and US. Mary serves on the Board of Directors of several Epidarex portfolio companies and is also an independent director of the University of Glasgow’s investment subsidiary, GU Holdings. She has 20 years of experience in the commercialization of early-stage health science technologies, having worked with technology transfer offices in leading UK academic institutions prior to joining Epidarex in 2013. She has a Ph.D. in Molecular Biology from the University of Edinburgh and undertook postdoctoral research at the University of Glasgow.

Session 2 Chair:
Prof. Rajvinder Karda
Professor in Gene Therapy, EGA Institute for Women's Health, UCL

Prof. Karda and her research team are focusing on developing novel gene therapy and RNA editing treatments for early onset, incurable genetic neurological and neurometabolic diseases. Dr Karda is a member of the scientific advisory committee for Dravet Syndrome Foundation, Spain and Cure DHDDS Charity, UK. She is also on the executive committee of British Gene and Cell Therapy Society. She has received funding from, MRC DPFS, LifeArc and GOSHCC to develop novel genetic therapies for genetic childhood neurological diseases.

Early career researcher speaker:
Dr Sara Aguti
Senior Research Fellow, Neurodegenerative Diseases, UCL

Dr Sara Aguti developed an interest in identifying effective treatments for neuromuscular and neurological diseases during her MSc at the University of Siena. She completed her PhD under the supervision of Francesco Muntoni and Haiyan Zhou, focusing on the development of RNA-based therapies for collagen VI–related myopathies. Following her postdoctoral work, she moved to MiNA Therapeutics to explore a different RNA-based therapeutic strategy based on the direct targeting of DNA to achieve gene upregulation. In 2023, Sara was appointed as a Senior Research Fellow at the Genetic Therapy Accelerator Centre (GTAC), where she focuses on developing RNA therapies using multiple strategies to correct disease-causing mutations in both neurological and neuromuscular disorders. In addition, her research aims to improve the targeted delivery of RNA therapeutics to specific tissues and cell types, one of the major bottlenecks for the clinical translation of RNA-based therapies.

Early career researcher speaker:
Dr George Hall
Research Fellow, Sergi Castellano Lab, UCL Great Ormond Street Institute of Child Health

George is a postdoctoral research fellow in the lab of Sergi Castellano at the UCL Great Ormond Street Institute of Child Health. Following his PhD in Computer Science, he moved to biology to develop computational methods to address unmet needs in translational clinical research. His research uses single-cell and spatial transcriptomics to compare biological structures, recently assessing the biological recapitulation of bioengineered oesophagi and organoid models of the developing midbrain. He is currently developing methods to allow the more sensitive comparison of samples profiled with single-cell transcriptomics and to generate realistic synthetic spatial transcriptomic datasets in settings where few reference examples are available. He co-organises the Institute of Child Health's Computational Biology Club, a forum for discussion for recent computational methods and problems being faced by researchers.

Early career researcher speaker:
Dr Maria Toms
Research Fellow, Moosajee lab, The Francis Crick Institute and Institute of Ophthalmology, UCL

Maria is a postdoc research fellow in Professor Mariya Moosajee’s group at the UCL Institute of Ophthalmology and the Francis Crick Institute. She completed her PhD in Molecular Genetics at UCL in 2018. Her research investigates the causes of genetic eye diseases and strategies to treat them, with a focus on non‑viral gene therapies.

Prof. Nikki Robertson
Professor of Perinatal Neuroscience, UCL; Consultant Neonatologist at University College London Hospitals (UCLH)

Nikki is professor of perinatal neuroscience at UCL, working also as consultant neonatologist at UCLH. For the last 23 years at UCL, Nikki has led the preclinical development of new therapies for neonatal brain injury, recently taking melatonin into a phase I study in babies with hypoxic ischaemic encephalopathy (HIE). HIE is a significant burden worldwide with devastating lifelong health, social, and educational impacts. The only current treatment is therapeutic hypothermia which is only partially effective. In the last 5 years, Nikki and her team have focused on Wharton Jelly Mesenchymal Stromal Cell (WJ-MSC) therapy, assessing the safety and efficacy of intranasal administration of WJ-MSC after hypoxia ischaemia in a large preclinical model. Using clinically relevant MR and EEG biomarkers, these data suggest that combination therapy may soon be ready for early phase clinical trials in babies with HIE.

Session 3 Chair:
Prof. Emma Morris BA MA MB BChir PhD FRCP FRCPath FMedSci
Dean, Faculty of Medical Sciences, UCL; Professor of Clinical Cell and Gene Therapy, UCL Institute of Immunity & Transplantation. Consultant Haematologist (Haematopoietic Stem Cell Transplantation and Gene Therapy) at UCLH & Royal Free NHS FTs

Professor Morris is a clinician scientist, clinically trained in haematology and allogeneic HSCT. Her current clinical practice is in HSCT, cellular and gene therapies for haematological malignancies and inherited immune deficiencies. She has developed and led the national allogeneic HSCT centre for adults with primary immune deficiencies (PID) at the UCL Centre for Immunodeficiency and co-ordinates an international MDT.

Her basic science research focus is on T cell immunology, inherited immunodeficiencies, gene therapy and gene editing. She leads a group interested in altering both the specificity and the function of gene-modified immune cells and stem cells. More recent research is focused on developing gene editing techniques as treatments for patients with PID. This research spans the entire translational pathway from animal models to in vitro experimental immunology to Phase I ‘first time in man’ clinical studies.

Alongside her role as Dean, Faculty Medical Sciences, Emma is the Director of the NIHR UCLH/UCL Biomedical Research Centre Inflammation, Immunity and Immunotherapeutics (III) Theme.

She is patron of the charity Haematology Cancer Care (HCC) which raises money for UCLH.

Professor Morris is a scientific founder of UCL Spin-out, Quell Therapeutics Ltd, which is developing gene-engineered regulatory T cells for transplant tolerance and autoimmune indications.

She was elected to the Academy of Medical Sciences in 2020.

Prof. Despina Eleftheriou
Professor of Paediatric Rheumatology, UCL; Consultant, UCL Great Ormond Street Institute of Child Health

Portrait of Professor DespinaEleftherious

Professor Despina Eleftheriou leads a translational research programme focused on rare inflammatory diseases in children. Her work integrates genomics, biomarker discovery, and early-phase clinical trials to develop targeted therapies and improve patient outcomes.

Her research includes the development of advanced therapies, including CAR T-cell approaches for autoimmune diseases and gene-based strategies such as lentiviral stem cell gene therapy and gene silencing for autoinflammatory conditions. Professor Eleftheriou has led international studies, and works closely with multidisciplinary and industry partners to translate innovative therapies into clinical application.

Dr Frederick Vonberg
Clinical Fellow in CAR-T Neurology, UCL Queen Square Institute of Neurology and University College London Hospitals (UCLH); Neurologist at the National Hospital for Neurology and Neurosurgery

Freddie is a Neurologist at the National Hospital for Neurology and Neurosurgery and Imperial College Healthcare Trust and a Clinical Fellow in CAR-T Neurology at the Queen Square Institute of Neurology and UCLH. His post-doctoral research, with Dr. Claire Roddie and Dr. Aisling Carr, explores the neurotoxic effects of Chimeric Antigen Receptor (CAR) T-cell therapy and the application of CAR-T to autoimmune neurological diseases. He is a founding member of the New and Emerging Oncotherapeutic Neurotoxicity (NEON) network. He undertook his doctoral work at Boston Children's Hospital, Harvard Medical School, and the Massachusetts Institute of Technology.

Katie Tinkler
Patient Representative

Mrs Katie Tinkler is one of the first three patients in the UK to receive a groundbreaking CAR T‑cell therapy for severe lupus through an NHS clinical trial led by University College London (UCL) and University College London Hospitals (UCLH) in 2024. A mother of three from Guildford, Katie has lived with lupus for more than 30 years, experiencing debilitating symptoms including extreme joint pain, fatigue, kidney disease and complications such as antiphospholipid syndrome.

After decades relying on steroids, immunosuppressants and multiple additional medications as her health and immune function declined, Katie described joining the trial as “beyond exciting”, marking the first real possibility of long‑term remission.

As a patient representative, Mrs Tinkler offers invaluable first‑hand insight into the lived experience of severe autoimmune disease and the profound impact that emerging therapies can have. Her perspective underscores the essential role of patients in shaping the future of advanced and potentially curative treatments.

Prof. Dimitri Michael Kullmann
Professor of Neurology Clinical & Experimental Epilepsy, UCL Queen Square Institute of Neurology

Prof. Kullmann’s interests span the fundamental mechanisms of synaptic transmission, the computational properties of small neuronal circuits, and alterations in neuronal and circuit excitability in epilepsy and other neurological disorders. Together with colleagues at the Institute of Neurology he has devised several gene therapies for pharmacoresistant epilepsy, and founded EpilepsyGTx, a UCL spinout company, to bring some of these to the clinic

Prof. Claire Booth
Mahboubian Professor in Gene Therapy and Paediatric Immunology, Head of Infection, Immunity and Inflammation Department, UCL; Deputy Theme Lead Gene, Stem and Cell Therapy, GOSH NIHR BRC; Cell & Gene Therapy Service Clinical Academic Lead; Consultant in Paediatric Immunology, Great Ormond Street Hospital

Claire is a Professor of Paediatric Immunology and Gene Therapy at UCL Great Ormond Street Institute of Child Health, leading the stem cell gene therapy programme. She oversees gene therapy trials for immune deficiencies, haematological, and metabolic disorders, focusing on developing novel treatments for immune disorders. Claire has extensive experience in leading first-in-human clinical trials and translating research into clinical practice. As an attending physician, she manages patients with immunodeficiencies, including stem cell transplants, with a particular interest in HLH disorders. Claire is an elected board member of the European and American Societies of Gene and Cell Therapy and the European Society for Immunodeficiencies, holds an honorary position at Boston Children’s Hospital and Harvard Medical School, and co-founded the AGORA Foundation to improve access to gene therapies for ultra-rare diseases.

Prof. Paul Gissen
Clinical Professor in Paediatric Metabolic Medicine and an Honorary Consultant at UCL Great Ormond Street Hospital for Children; NIHR Senior Investigator

Paul Gissen is the Director Designate of the National Institute for Health and Care Research GOSH Biomedical Research Centre (NIHR GOSH BRC) and leads the Gene, Stem and Cellular Therapies. He is also the UK Chief Investigator for a number of industry and academic sponsored clinical trials of novel therapies.

Professor Gissen obtained his medical degree from the University of Glasgow and trained in paediatrics at Manchester, Sheffield and Birmingham Children’s Hospitals specialising in inherited metabolic disorders. He undertook his PhD at Birmingham University where he identified genetic causes of several rare paediatric diseases.

His research interests are in developing novel gene based therapies for Rare Paediatric Metabolic Disorders.

Professor Andrew Dick
Director UCL-Institute of Ophthalmology; Duke Elder Professor of Ophthalmology, UCL Institute of Ophthalmology; Co-Director of National Health Institute for Research Biomedical Research Centre, Moorfields Eye Hospital and UCL-Institute of Ophthalmology, London

Andrew qualified in medicine with a degree in Biochemistry (BSc (Hons)) from the University of London, and during his medical education he also undertook an MRC secondment in Biochemistry with Professor Coleman in Yale. Following completing MRCP, he entered ophthalmology residency and obtained his postgraduate research degree in Immunology in 1993 at the University of Aberdeen. He underwent an MRC Post-Doctoral Fellowship to work with Jon Sedgwick at the Centenary Institute of Cancer Medicine and Cell Biology in Sydney Australia. His clinical expertise is in medical and surgical management of retinal and inflammatory disease.

His research spans basic and translational science including early and phase III trials in inflammation as related to autoinflammatory, autoimmune and degenerative retinal disease.

Professor Dick is a Fellow of the Academy of Medical Sciences in the UK for his significant contribution to research and scholarship, in particular his development of molecular targets and biologic therapy for inflammatory eye disease. He was awarded the Alcon Research Institute Research award in 2011. His work has led to discoveries in ocular inflammatory disease and degenerative disease and co-founder of Cirrus Therapeutics.

Prior to becoming Director of institute of Ophthalmology, the UCL-Institute of Ophthalmology, he was Director of Research for the Faculty of Medicine and Dentistry at University of Bristol. He has previously been Editor of British Journal of Ophthalmology, President of European Vision and Eye Research (EVER), Master of Oxford Ophthalmological Congress and currently Vice-President of ARVO.

In Memoriam - Honoring Professor Pete Coffey:
UCL colleagues have paid tribute to world-leading eye researcher Professor Pete Coffey, who pioneered a treatment enabling people whose vision had been destroyed by disease to see again. At the symposium, a tribute session will be presented by Professor Andrew Dick in honour of Professor Coffey. Read the full tribute.

In Memoriam - Honoring Professor Pete Coffey:

UCL colleagues have paid tribute to world-leading eye researcher Professor Pete Coffey, who pioneered a treatment enabling people whose vision had been destroyed by disease to see again. At the symposium, a tribute session will be presented by Professor Andrew Dick in honour of Professor Coffey.

Read the full tribute.

Research Posters

In order of display position:

Laura Vede Ussingkær
'Next-generation glutamate-sensing gene therapy for focal refractory epilepsy'
Sijiang Liu
'Phenotypic Characterisation and Gene Therapy Development in a mouse model for SORD neuropathy'
Eriomina Shahaj
'Restoration of cilia motility via mRNA therapy in Primary Ciliary Dyskinesia'
Arki Tian
'Developing chimeric glutamate-gated potassium channels (GKv) as novel tools for neural circuit manipulation'
Sara Parvar
'Formulations for convection-enhanced delivery of therapeutics in diffuse midline glioma'
Arianna Fozzato
'Optimising codon sequence and delivery of an AAV9 gene therapy for deoxyguanosine kinase deficiency in brain and liver'
Sanjana Kurkimat
'Preparation of circular cell-free plasmids for viral vector therapies'
Benjamin Fiedler
'Poly(Acrylamido) lipids replace PEG to enhance mRNA-LNP efficacy in immune cells and evade anti-PEG antibodies'
Han Zhang
'Nanogel platform Enables Efficient and Reproducible mRNA Delivery Across Multiple Human iPSC Cell Lines'
Francesca Picco
'Developing a cell therapy for dopamine transporter deficiency syndrome'
Barbora Cerna
'Patient‑derived forebrain assembloids to investigate the GABAergic developmental switch in SLC12A5‑related epilepsy'
Li-An Brown
'Contaminating plasmid sequences and disrupted vector genomes in the liver following AAV gene therapy'
Poster withdrawn
Ella Whittle
'A scalable and equitable framework for target and patient prioritisation in rare disease antisense therapeutics'
Sean Briggs
'Using Phage Display to Identify Peptides for Targeted Antisense Oligonucleotide Delivery to Muscle Interstitial Fibroblasts'
Hassan Rashidi
'Histotripsy-Enabled Liver Organoid cell transplantation to Treat Congenital Liver Disorders'
Widyan Alamoudi, Lewis Hall, Hei Ming Kenneth Ho, Helena Ros, Han Zhang, Zoe Whiteley, Stephanie Schorge, Duncan Craig, Micheal T. Cook, David Shorthouse, Qasim Rafiq, Ahad A. Rahim
'Optimization of rAAV Production Using a Microfluidics-Fabricated Nanogel Platform via Machine learning'
Corinna Schlosser
'Biodistribution evaluation of a fibroblast-targeting peptide for cell type specific ASO delivery'
Uzoamaka Okoli
'SCREEN: Effects of a panel of pre-clinical targeted therapeutic combinations on patient derived prostate explants as revealed by Spatial Transcriptomics.'
Yiwen Li
'Reprogramming AAV performance through a rational platform targeting non-receptor-binding capsid regions'
Christina Karapouliou, Enzo Giardina, Ruhina Maeshima, Stephen Hart, Andrew Stoker
Great Ormond Street Institute of Child Health, University College London
'Repurposing Retinoic Acid for Toxin Gene Therapy of Neuroblastoma'
Tina (Qiantong) Chen
'Resurrecting and relocating ancestral MAAPs to boost AAV5 secretion'
Asma Naseem
'In vivo prenatal base editing for the treatment of Krabbe disease'
Lara Carter
'Robust Extraction of Translational Pharmacokinetic Evidence from Biomedical Literature to Support Animal-to-Human Dose Translation'
Kidus Estifanos Biru
'Polyphosphoester Steric Lipids (PSLs) as PEG-Lipid alternatives for Lipid Nanoparticles (LNPs)'
Francisco M. C. Moreira
'Multiplex CRISPR-activation Gene Therapy to Treat Intractable Epilepsy'

Symposium Organisers

UCL Translational Research Office (TRO)

The TRO is an integral part of UCL’s biomedical research powerhouse. As an expert team of applied scientists and business developers, we support our researchers in realising their translational ambition by offering strategic advice and funding support and bridging the gap with industry to help establish collaborations that drive the pipeline of therapeutic, device and diagnostic innovations. Learn more

Contact the TRO for partnerships:
If you are a UCL researcher looking for translational research consultation or funding application support, please contact: Pamela Tranter, p.tranter@ucl.ac.uk
If you are an industry partner looking for collaboration opportunities with UCL, please contact: Carmel Reilly, carmel.reilly@ucl.ac.uk

Social Media:
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UCL Therapeutic Innovation Networks (TINs)

The TINs are modality-focused community of UCL research expertise, centered around six of UCL’s core strengths: i) Small Molecules; ii) Cell & Gene Therapies; iii) Biologics; iv) Re-purposing; v) Devices & Diagnostics; and vi) Regenerative Medicine. The TINs were established by the UCL Translational Research Office (TRO), which brings together professionals from across academia and industry. Register with us to become a TINs member, get involved in our activities, and be the first to receive our news and event invitations. Learn more

Join TINs and become part of the collaborative network:
1. Complete the form to become a TINs members and register your interest area.
2. Be ready to receive our newsletter, TINs updates and event invitations.

Our sponsors

	Networking sponsor: UCLB is UCL's expert commercialisation partner. We work side by side with academics, clinicians and innovators to turn exceptional research into successful products, spinouts and technologies that change the way the world works, lives and thrives. As a powerhouse of commercialisation at the heart of London's innovation ecosystem, we bring brilliant ideas to life - and to market - delivering outstanding returns, substantial impact, and fuelling the next generation of discovery. Learn more about UCLB. Find out more about UCLB's impact report.
	The Cell and Gene Therapy Catapult is an independent innovation and technology organisation committed to the advancement of the CGT industry with a vision of a thriving industry delivering life changing advanced therapies to the world. Its aim is to create powerful collaborations which overcome challenges to the advancement of the sector. With around 300 experts covering all aspects of advanced therapies, it applies its unique capabilities and assets, collaborates with academia, industry and healthcare providers to develop new technology and innovation. Learn more
	Chemometic is a global leader in high-precision cell counting and analysis solutions, serving biotech, pharmaceutical and academic customers worldwide. Designed and manufactured in Denmark, our instruments deliver reliable, consistent data. Driven by innovation and strong growth, we are committed to advancing life sciences and becoming a leading provider of analytical instrumentation. Learn more
	Cytiva is a global life sciences leader dedicated to helping customers discover and commercialize the next generation of therapeutics. Together, we bring dedicated technical expertise and a broad portfolio of tools and technologies that enable the development, manufacture, and delivery of transformative medicines to patients. Protein Research \| Cytiva
	Entegris leverages 50+ years of materials science expertise to deliver clean, reliable, scalable life sciences solutions that reduce validation time, costs, and time to market. Our portfolio includes fluid management and single‑use systems, particle characterization, liquid packaging, filtration and purification, and specialty chemicals.Learn more
	NHSBT Cellular and Molecular Therapies provides GMP manufacturing and translational support for academic and commercial advanced therapy programmes. Benefiting from integrated NHS infrastructure, we deliver plasmid, viral vector, and cell therapy manufacturing—accelerating the transition from research innovation to clinical application through a collaborative, flexible, cost recovery model to maximise patient access to life-changing therapies. Learn more

The event will take place in the Kennedy Lecture Theatre, UCL Institute of Child Health (ICH), 30 Guilford Street, London, WC1N 1EH. Please see the accessibility information here.
Talks and presentations will take place in the ICH Kennedy Lecture Theatre
Posters will be displayed in the ICH Winter Garden
Lunch and refreshments will be served in the ICH Balcony and Winter Garden - Drinks reception will take place in the ICH Winter Garden