Professor Haiyan Zhou is a Professor of Genetic Medicine at University College London (UCL) and a UK Harrington Rare Disease Scholar. Her research specializes in the preclinical development and clinical translation of nucleic acid therapies (NAT) for rare genetic disorders. She serves as the Deputy Theme Lead for the Novel Therapy Theme at the NIHR Biomedical Research Centre at UCL Great Ormond Street Hospital (GOSH BRC), where she leads the Individualized RNA Therapy Program for paediatric rare diseases.

Prof. Zhou is the Director of the UK Platform of Nucleic Acid Therapy (UPNAT) for rare diseases, a specialized initiative supported by the UK Medical Research Council (MRC) and the NIHR. As the principal investigator on a range of research projects, she is at the forefront of developing RNA-based therapeutics for a wide spectrum of genetic disorders. In addition to her research, Prof. Zhou directs the MSc in Personalized Medicine and Novel Therapies at UCL, where she plays a key role in training the next generation of scientists in cutting-edge therapeutic development.

Rafael J. Yáñez-Muñoz is Professor of Advanced Therapy and Director of the Royal Holloway Centre of Gene and Cell Therapy at the Department of Biological Sciences, Royal Holloway University of London, UK. Rafael received his BSc and PhD in Biochemistry and Molecular Biology from the Autonomous University of Madrid, Spain. Following appointments with MRC/Imperial College, University College London, Queen Mary’s University of London and King’s College London, he now leads the Advanced Gene and Cell Therapy lab (AGCTlab.org) at Royal Holloway. He is also the outgoing President (2021-2025) of the British Society for Gene and Cell Therapy. Rafael was Editor-in-Chief of Gene Therapy (2017-2020), where he promoted the internationalization of the journal and EDI in senior editing roles and the editorial board. Rafael also believes it is very important to engage with the wider society, was a Trustee and then Chair of Trustees of the Genetic Alliance UK (2015-2020) and has organised a yearly outreach event on Rare Disease Day from 2011. Rafael’s laboratory currently works on novel therapies for ataxia telangiectasia and spinal muscular atrophy.

Florian Merkle is a NYCSF-Roberston Stem Cell Investigator and Associate Professor of Pharmacology at the University of Cambridge. His group studies the disease mechanisms of metabolic and neurodegenerative disease using human induced pluripotent stem cell (hiPSC)-derived and animal model systems with the ultimate aim of developing new therapies, covering three main themes. First, he and his group study genetic stability of hiPSCs and are identifying culture methods that minimise the appearance of culture-acquired mutations such a TP53 point mutations. They have nominated the hiPSC line KOLF2.1J as a ‘reference’ for the field that is now used by over 600 groups worldwide and forms the basis of several large-scale genetic studies. Second, Florian’s group differentiates hiPSCs into hypothalamic neurons that regulate appetite and has used this model system to reveal how anti-obesity drugs like semaglutide (Ozempic/Wegovy) signal and to identify new candidate appetite-regulatory targets and anti-obesity therapeutic strategies. Finally, the Merkle group studies the mechanisms linking metabolic and neurodegenerative disease including how some anti-obesity drugs are neuroprotective. These studies, bridging hiPSC and mouse models, have the potential to rapidly enter the clinic due to drug repurposing. Florian is also an affiliate member of the Institute of Metabolic Science and Cambridge Stem Cell institute, and serves as an industry consultant, board member, and entrepreneur.

Uta Griesenbach, Professor of Molecular Medicine, National Heart and Lung Institute Imperial College London

Uta is a Professor of Molecular Medicine and has over 25 years experience in developing advanced therapeutics. Her research interests are related to the development of gene therapy for cystic fibrosis (CF) and other lung diseases. She has overseen vector and biomarker development, toxicology studies, as well as vector manufacturing and has been a Co-I on several clinical trials. Uta is a member of the UK Respiratory Gene Therapy Consortium (GTC) strategy group, Co-director of the Imperial Centre for Excellence in Advanced Therapies, a Co-founder of AlveoGene (Imperial College Spinout) and a Non-executive director of the Cell and Gene Therapy Catapult. In addition to her research activities, she has an active role in education and workforce development related to advanced therapeutics in the UK.

Steven Howe, Resolution Therapeutics

Steven has more than 25 years of experience working in cell and gene therapy, including postdoctoral positions at the UCL Great Ormond Street Institute of Child Health, London, before joining GlaxoSmithKline in 2015 as Senior Director of Product Development leading a team in developing manufacturing processes for advanced therapies.

During his career Steven has led groups researching a wide range of advanced therapies and technologies to apply them, including viral vectorology, stem cell biology and cell processing. His recent positions have focused on Process Development for manufacturing platforms to translate cell and gene therapies into the clinic, for disease areas including rare diseases, metabolic disorders and cancer.

In his current role at Resolution Therapeutics, he leads the team responsible for developing manufacturing approaches to produce regenerative macrophage therapies to treat inflammatory and fibrotic disease.