Unparalleled gene therapy and rare disease expertise
Launched in October 2022, we are a UK-based spin-out from University College London (UCL), emerging from the world-leading gene therapy and rare diseases expertise of our academic founders.
Our Academic Founders
Our academic founders are long-term collaborators and world leaders in their respective fields. Their expertise spans discovery, translation and clinical insights, and together with their research and clinical development groups, they form our unique and integrated R&D core.
Professor Paul Gissen
Prof. Paul Gissen
Paul Gissen is a Professor of Paediatric Metabolic Diseases at University College London (UCL) and an Honorary Consultant at Great Ormond Street Hospital (GOSH) and a National Institute of Health and Care Research (NIHR) Senior Investigator. He leads the Gene, Stem and Cellular Therapies Theme at GOSH and the GOS ICH Biomedical Research Centre. Paul has previously held research fellowships from GSK and the Wellcome Trust. He has published more than 170 peer reviewed papers and book chapters and is involved in several filed patents. Professor Gissen has been involved in industry funded clinical trials since 2005 and is currently a UK Chief Investigator for several clinical trials in Niemann Pick Type C and Batten Diseases and leads specialist clinics for patients with these disorders at GOSH. He is the clinical lead for NICE managed access agreement for cerliponase alfa and acted as a clinical expert for NICE assessments of several drugs for rare metabolic disorders.
Professor Manju Kurian
Prof. Manju Kurian
Manju Kurian is Professor of Neurogenetics at University College London (UCL) and National Institute of Health and Care Research (NIHR) Professor. She is the Head of Molecular Neurosciences at UCL Great Ormond Street Institute of Child Health (GOS ICH) and leads the Accelerating Novel Therapies theme at GOS ICH Biomedical Research Council. Professor Kurian holds the Sir Jules Thorn Award for Biomedical Research and is an Honorary Consultant in Paediatric Neurology at Great Ormond Street Hospital. She is a clinician with over 15 years’ experience in paediatric neurology, running a unique, internationally renowned UK specialist service for genetic movement disorders in children, including the development of clinical trials.
Professor Ahad Rahim
Prof. Ahad Rahim
Ahad Rahim is Professor of Translational Neuroscience at University College London (UCL), Wellcome Chair in Pharmacology and the Head of Pharmacology Department at the UCL School of Pharmacy. He has over 20 years of experience in the gene therapy field, has filed a number of patents and has published extensively on the use of both viral and non-viral delivery platforms. Professor Rahim is also a co-founder of the UCL GTxNeuro Viral Vector Core Facility that facilitates progression of academic gene therapy programs. He leads a research team that focuses on studying paediatric and adult neurometabolic disorders and developing novel therapies. His laboratory is involved in the development and preclinical testing of various biologics and advanced medicinal therapeutic products to treat these conditions including gene therapy, exosomes and neuroprotective peptide therapies.
Professor Simon Waddington
Prof. Simon Waddington
Simon Waddington is Professor of Gene Therapy at University College London (UCL) and is an expert in preclinical models of gene & stem cell transfer for perinatal gene delivery for early onset, debilitating and lethal diseases. He chairs the UCL Cell and Gene Therapy Therapeutic Innovation Network. Professor Waddington has a wealth of research experience in gene therapy and has acted as consultant for several companies in the industry.
