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Genetic Therapy Accelerator Centre

The Centre is based at Queen Square and directed by Professor Francesco Muntoni, a Paediatric Neurologist with dual appointments in both institutions (IoN QS and GOSH ICH).

In 2022, Professor Muntoni was made director of the Genetic Therapy Accelerator Centre, a new gene therapy translational research partnership, based at Queen Square Institute of Neurology and in close collaboration with the UCL Great Ormond Street Institute of Child Health. This cross-faculty collaboration within UCL will drive forward an exciting evolving area with direct therapeutic benefits for patients with disabling neurological conditions. In the last few years, he obtained several international awards, including the 2022 Ottorino Rossi Award, the World Duchenne Organisation 2022 Leadership Awards, the 2023 European Paediatric Neurology Society Jean Aicardi Award and the 2024 Richard Attenborough Award for Outstanding Achievement for Muscular Dystrophy.

Contacts

Professor Francesco Muntoni, Director of UCL Genetic Therapy Accelerator Centre, Institute of Neurology, Professor of Paediatric Neurologist, Director of the Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children, London, UK. Email: f.muntoni@ucl.ac.uk

Sophie Ostler, Centre Officer, Genetic Therapy Accelerator Centre. Email: s.ostler@ucl.ac.uk

Professor Haiyan Zhou, Professor of Genetic Medicine, Dubowitz Neuromuscular Centre and Genetic Therapy Accelerator Centre. Email: haiyan.zhou@ucl.ac.uk

Professor Jenny Morgan, Emeritus Professor of Cell Biology, Dubowitz Neuromuscular Centre and Genetic Therapy Accelerator Centre. Email: jennifer.morgan@ucl.ac.uk

Professor Jo Ng, Professorial Research Fellow, Genetic Therapy Accelerator Centre. Email: j.ng@ucl.ac.uk

Dr Silvia Torelli, Senior Cell Biologist, Dubowitz Neuromuscular Centre and Genetic Therapy Accelerator Centre. Email: s.torelli@ucl.ac.uk

Dr Sara Aguti, Senior Research Fellow, (currently on maternity leave) Genetic Therapy Accelerator Centre. Email: sara.aguti@ucl.ac.uk

Dr Riccardo Privolizzi, Senior Research Fellow, (maternity cover), Genetic Therapy Accelerator Centre. Email: riccardo.privolizzi.13@ucl.ac.uk

TBC, Senior Research Fellow, Cellular Myologist, Genetic Therapy Accelerator Centre and Dubowitz Neuromuscular Centre. Email:

Dr Laia Torres Masjoan, Research Fellow, Genetic Therapy Accelerator Centre. Email: l.masjoan@ucl.ac.uk

Dr Rebeca Gil, Research Fellow, Genetic Therapy Accelerator Centre. Email: rebeca.gil@ucl.ac.uk

Hester Chu, Research Assistant and PhD Candidate, Genetic Therapy Accelerator Centre. Email hester.chu.16@ucl.ac.uk

Arta Aghaeipour, Research Assistant, Genetic Therapy Accelerator Centre. Email: a.dehkaei@ucl.ac.uk

Camila Vallve Maine, Research Assistant, Genetic Therapy Accelerator Centre. Email: c.maine@ucl.ac.uk

UCL is ranked number two in London for Life Sciences and number nine globally (QS World University Rankings 2023). UCL has also been ranked number two in the UK for research power in the latest REF. The faculty is home to a vibrant community of outstanding students, researchers and academics, supported by a committed professional services team who together research, teach and study the science and biology of life.

Centre Director, Professor Francesco Muntoni

Professor Francesco Muntoni is the Centre Director, Professor of Paediatric Neurologist and the Director of the Dubowitz Neuromuscular Centre, at the UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children, London, UK. In the Institute and hospital, he led the Novel Therapies Programme of the Biomedical Research Centre between 2008 and 2022 and also led the Developmental Neuroscience Programme between 2008 and 2018. Since 2008 he is the co-director of the MRC Centre for Neuromuscular Diseases at UCL.

Professor Muntoni has an interest in pathogenesis, deep phenotyping, gene identification for rare neuromuscular conditions and translational research in Duchenne muscular dystrophy, spinal muscular atrophy and congenital myopathies. He is involved in several natural history studies and clinical trials. His research funded by the Department of Health, MRC and the European Commission led to the development and early clinical trials of 2 morpholino antisense oligonucleotides, now approved by the FDA, that induces partial correction of the processing defect of the DMD gene in boys with Duchenne muscular dystrophy. He is the UK chief investigator for multiple clinical trials on DMD and SMA, including genetic therapies involving antisense oligonucleotides and AAV-delivered transgenes. In the last few years he obtained several international awards, including the 2022 Ottorino Rossi Award, the World Duchenne Organisation 2022 Leadership Awards, the 2023 European Paediatric Neurology Society Jean Aicardi Award and the 2024 Richard Attenborough Award for Outstanding Achievement for Muscular Dystrophy.

Email: f.muntoni@ucl.ac.uk

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Centre Officer, Sophie Ostler: Sophie works as part of the team led by Professor Francesco Muntoni, she is responsible for the day-to-day management of the centre as well as ensuring effective communication practices across the research team, collaborators and funders. In addition to working closely with the team and strategic partners, her responsibility focuses on delivering the communications plan, monitoring the project's expenditure, budgets and liaising with relevant partners and advisors on a range of research activities. Sophie studied at the University of Cambridge and has a keen interest in actively promoting and supporting awareness for individuals living and working with rare diseases. She also provides greater understanding for mental health support, (equality, diversity, Inclusion and well-being) to staff and students through outreach programmes and networking. Email: s.ostler@ucl.ac.uk

Professor of Genetic Medicine, Professor Haiyan Zhou: Professor Haiyan Zhou is Professor of Genetic Medicine at University College London. Her research focuses on preclinical development of RNA-targeted novel therapies and translational biomedical research in rare genetic disorders. Professor Zhou is the deputy lead of the Novel Therapy Theme in the NIHR Biomedical Research Centre at UCL Great Ormond Street Hospital and Institute of Child Health. She is also the director of the MSc Personalized Medicine and Novel Therapies Programme at UCL. Haiyan is member of World Muscle Society, Oligonucleotide Therapeutic Society and American Society of Gene and Cell Therapy. She is also the UK representative and management committee member of the European Cooperation in Science and Technology (e-COST) on ‘Delivery ofAntisense RNA Therapeutics’. Professor Zhou is the 2021 Harrington UK Rare Disease Scholar and holds a medical degree in clinical medicine and PhD award from Peking Union Medical College. Twitter: @lab_zhou
Emeritus Professor of Cell Biology, Professor Jenny Morgan: Professor Jenny Morgan, research focuses on the biology of skeletal muscle and its capacity to regenerate, using in vitro models of stem cell activation, proliferation and differentiation and in vivo transplantation models. The major areas of research are the identification of stem cells that contribute to skeletal muscle regeneration, and the genetic and functional manipulation of these cell populations to enhance muscle repair in skeletal muscle regenerative medicine and in the treatment of muscular dystrophies. Other research areas include the restoration of dystrophin in Duchenne muscular dystrophy. Dubowitz Neuromuscular Centre, Developmental Neurosciences Research and Teaching Department, UCL Great Ormond Street Institute of Child Health. Email: jennifer.morgan@ucl.ac.uk Twitter: @JenniferEMorgan
Professorial Research Fellow, Professor Jo Ng: Professor Jo Ng is a Professorial Research Fellow developing gene therapies for untreatable neurological disorders that have rapid potential for translation. By understanding the clinical disease and disease pathophysiological mechanisms Dr Ng and her team use Adeno-associated virus (AAV) technology to develop gene therapy approaches for neurological disorders. We evaluate viral capsids, synthetic promoters, and response elements to custom-design gene therapies in order to improve delivery to the CNS and develop cell-specific targeting. To facilitate translation, we use techniques that include lentiviral gene therapy in vitro on iPSC-derived neurons from patients with AAV gene therapy in transgenic neurological mouse models to establish in vivo gene therapy efficacy and model clinical application. Dr Ng collaborates with academic research groups and industry partners to accelerate translation of new CNS genetic therapy approaches. Email: j.ng@ucl.ac.uk Twitter: @DrJo_Ng Dr Silvia Torelli worked as a senior cell biologist at the University of Cagliari, Italy and as a researcher for 16 years, before joining the Neuromuscular Unit (Department of Paediatrics) at Hammersmith Hospital Campus, Imperial College London in 2007. In 2007 Silvia moved to UCL, Great Ormond Street Institute of Child Health, and has been working at the Dubowitz Neuromuscular Centre. Her research focuses on understanding the cause of congenital muscular dystrophies and in particular a group of diseases known as “secondary dystroglycanopathies' (characterised by the altered glycosylation of alpha-dystroglycan). She also collaborates in studies on Duchenne and Becker Muscular Dystrophies and is specifically involved in the optimisation of techniques for dystrophin quantification. Email: s.torelli@ucl.ac.uk

Senior Cell Bilogist, Dr Silvia Torelli: Dr Silvia Torelli worked as a senior cell biologist at the University of Cagliari, Italy and as a researcher for 16 years, before joining the Neuromuscular Unit (Department of Paediatrics) at Hammersmith Hospital Campus, Imperial College London in 2007. In 2007 Silvia moved to UCL, Great Ormond Street Institute of Child Health, and has been working at the Dubowitz Neuromuscular Centre. Her research focuses on understanding the cause of congenital muscular dystrophies and in particular a group of diseases known as “secondary dystroglycanopathies' (characterised by the altered glycosylation of alpha-dystroglycan). She also collaborates in studies on Duchenne and Becker Muscular Dystrophies and is specifically involved in the optimisation of techniques for dystrophin quantification. Email: s.torelli@ucl.ac.uk
Senior Research Fellow, Dr Sara Aguti (currently on maternity leave): Dr Sara Aguti developed an interest in finding successful treatments for neuromuscular and neurological diseases during her MSc at the University of Siena, Italy, She completed her PhD under the supervision of Prof. Francesco Muntoni and Prof. Haiyan Zhou and focused on developing RNA therapies for collagen VI-related myopathy. Sara discovered antisense oligonucleotide (ASO) sequences able to correct common mutations in dermal fibroblasts established from collagen VI patients. After her postdoc, she moved to MiNA Therapeutics to explore another aspect of RNA therapy: RNA activation by directly targeting DNA and was recently appointed as a senior research fellow at the Genetic Therapy Accelerator Centre, focusing on developing RNA therapies using different strategies, such as ASOs for splicing modulation or silencing approaches, siRNA, and saRNA, to correct mutations in both neurological and neuromuscular disorders. Additionally, Sara is interested in enhancing the delivery of RNA therapies to specific tissue or cell lines, for one of the main bottlenecks for the development of RNA therapies. Email: sara.aguti@ucl.ac.uk

Senior Research Fellow, Dr Riccardo Privolizzi (maternity cover): Dr Riccardo Privolizzi is a Senior Research Fellow in the Genetic Therapy Accelerator Centre at the UCL Queen Square Institute of Neurology. He's also an Honorary Research Fellow and Visiting Lecturer in Gene Therapy at the UCL EGA Institute for Women's Health and GOS Institute of Child Health, and an ECR Board member of the British Society for Gene and Cell Therapy. Riccardo specialised in the characterisation of novel synthetic promoters and translational AAV gene therapy of a mouse model of infantile parkinsonism-dystonia. Riccardo has a keen interest in the identification and development of novel gene regulatory elements for selective targeting of specific cell types, and is passionate about teaching and science communication. Email: riccardo.privolizzi.13@ucl.ac.uk
Senior Research Fellow, Cellular Myologist: Coming soon
Research Fellow, Dr Laia Torres Masjoan: Laia obtained her PhD in Neuroscience at King's College London, where she worked on direct neuronal reprogramming, an experimental approach for neuronal regeneration based on the forced expression of key neurogenic factors for the direct conversion of non-neuronal cells (i.e. glial cells) into induced neurons. In 2023 she joined the GTAC team as a Research Fellow to work on the TransNAT project, directed by Professor Muntoni and Professor Zhou. Her motivation to move her career towards translational research led Laia to be part of the team where she will be testing the efficacy of potentially therapeutic antisense oligonucleotides on in vitro and in vivo models of Duchene Muscular Dystrophy. Laia's work aims to optimize the efficacy, safety and delivery of exon-skipping antisense oligonucleotides (ASOs) for the treatment of the neuromuscular disorder Duchenne Muscular Dystrophy (DMD). Some of DMD are caused by exon deletions altering the reading frame of the dystrophin gene (DMD), and, although four exon-skipping ASOs for the treatment of DMD have been approved by the US FDA, their treatment efficacy remains low. Laia's work at GTAC involves the use of different models of human DMD skeletal and cardiac muscle cells, including hiPSCs-derived and immortalized cells, to test new generation exon-skipping ASOs with different chemical modifications and conjugations to different moieties, and to analyse their exon-skipping efficacy, dystrophin protein restoration, and intracellular trafficking. These analyses will allow the identification of the best candidates for further tests on DMD mouse models. Email: i.masjoan@ucl.ac.uk

Research Fellow, Dr Rebeca Gil: Dr Rebeca Gil is a research fellow at GTAC working on gene therapies for neurological disorders with no current effective treatment. She completed her PhD at Royal Holloway University of London focused on gene therapies to treat Duchenne Muscular Dystrophy. She received her MSc on "Genes, drugs and stem cells - novel therapies" at Imperial College London. She studied Biotechnology Engineering for her undergrad, where she developed an interest in novel therapies to treat diseases that had no previous cure. She is currently an early career representative on the board of the British Society for Gene and Cell Therapies (BSGCT). She has interest in supporting translation of novel therapies, spreading the word on gene & cell therapies to the public and helping early career researchers to get involved in the field; in alignment with her role as BSGCT Early Career Development & Collaboration Subcommittee co-chair and chair of the BSGCT Promotions and Communication Subcommittee.. Email: rebeca.gil@ucl.ac.uk; Research Assistant, Arta Aghaeipour Dehkari
Research Assistant and PhD Candidate , Hester Chu: Hester Chu is a Research Assistant and PhD Candidate. Coming from a pharmacist background, she is keen to develop novel gene therapy for neurological diseases currently without effective treatments. During her undergraduate, she was awarded the Wellcome Trust Biomedical Vacation Scholarship, and the BSGCT Undergraduate Research Bursary, to conduct adeno-associated virus (AAV) projects at UCL. She is also interested in improving gene therapy efficacy and safety profiles by regulatory element optimisation and other methods. Email hester.chu.16@ucl.ac.uk Twitter: @HesterChu

Research Assistant, Arta Aghaeipour: Arta Aghaeipour obtained her BSc in Biomedical Sciences from Royal Holloway, University of London and an MSc in Human Molecular Genetics from Imperial College London. Her studies at Imperial College focused on Human Molecular Genetics coupled with more than three years of dedicated research in nucleic acid therapy and paediatric diseases, equipped her with a deep understanding of drug development and disease modelling for rare paediatric disorders. Arta worked at the Dubowitz Neuromuscular Centre based in UCL Institute of Child Health, working on Duchenne Muscular Dystrophy and its comorbidities in the brain, as part of the brain Involvement in Dystrophinopathies (BIND) consortium. In 2023, she joined the GTAC team, directed by Professor Muntoni and Professor Zhou on screening safe targeted nucleic acid therapies on muscle as a part of the TransNAT consortium. Arta is passionate about bridging the gap between scientific discoveries and tangible societal improvements. She is particularly drawn to the challenges of understanding disease mechanisms, creating accurate disease models, and developing drugs to enhance the quality of life for patients. The project she is currently working on focuses on developing nucleic acid therapies with better efficacy for Duchenne Muscular Dystrophy as a part of the TransNAT consortium. Email: a.dehkari@ucl.ac.uk
Research Assistant, Camila Vallve Maine: Camila received a MSci in Applied Biological Research from the Royal Veterinary College, where she undertook three years of a bachelor's degree and one integrated master's year. Her bachelor's thesis was on inflammation markers in genes linked with autism spectrum disorder and her master's work was on adeno-associated virus (AAV) gene therapy for Dravet Syndrome, a rare channelopathy in children. Currently, she is developing AAV therapies for infantile Parkinsonism. Camila is passionate about making science more accessible for people of all backgrounds, supporting the Equality, Diversity and Inclusion (EDI) group missions in extending the reach of research, and ultimately wants to improve the lives of children across the globe throughout education and novel genetic therapies development. She is open for collaboration across different sectors, contact her on email for discussion. Email : c.maine@ucl.ac.uk

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