This webcast was developed by the ‘AAV gene therapy in the clinical setting’ meeting faculty. The sponsoring companies have provided sponsorship to Omniamed Communications to support the costs of running the webcast. The sponsoring companies have had no input into the agenda, speaker selection, presentations or collateral content.

AAV gene therapy in the clinical setting:
a practical approach

7 September 2021 / 09.00 – 12.15

Adeno-associated viral (AAV) gene therapy is very rapidly progressing. Many AAV therapies are being tested in early and more advanced clinical trials, with some recently receiving European Medicines Agency (EMA) approval and becoming available under the NHS. In view of the progress in many of the ongoing clinical trials, it is anticipated that many more AAV gene therapy products will become available for patients currently receiving other, or no, therapies within the NHS.

The handling, administration, and clinical surveillance of patients receiving AAV gene therapy is different to other therapeutic interventions, especially regarding the potential for adverse events and how to best manage those. Several UK hospitals and biomedical research centres already have a wealth of knowledge and experience in running AAV gene therapy trials in several indications. But the availability of AAV gene therapy products for larger numbers of paediatric and adult patients with genetic conditions means it is necessary to build expertise on AAV in many other sites.

This meeting has been set up to address the unique challenges of AAV gene therapy, and provide practical suggestions on how these therapeutic interventions can best be delivered.

Which topics will be discussed?

  • Mechanism of action of AAV gene therapy, and how this differs from other forms of gene therapy
  • How to set up a site for AAV gene therapy: from pharmacy, to Genetically Modified Organisms Safety Committee, to therapy delivery location
  • Preparing patients ahead of the AAV gene therapy
  • Post-discharge planning: safety monitoring and hub-and-spoke models of follow-up
  • Healthcare professionals’ experience and patient and public involvement

Who should attend this meeting?

The target audience is primarily those in the delivery team in hospitals who are currently not involved in AAV gene therapy. This includes nurses, coordinators, pharmacists, physicians and other members of the MDT.

In association with

7 September 2021 / 09.00 – 12.15

Speakers:

Professor Francesco Muntoni

Professor of Paediatric Neurology and Theme Lead for Novel Therapies, Great Ormond Street Hospital

Francesco Muntoni is a Professor of Paediatric Neurology at Great Ormond Street Hospital (GOSH), with an interest in the clinical and molecular aspects of neuromuscular disorders. He is the Director of the Dubowitz Neuromuscular Centre – a leading clinical and research institution for children affected by neuromuscular disorders – and is Theme Lead in Novel Therapies at the UCL Great Ormond Street Institute of Child Health. Since 2008, in close collaboration with the team at the UCL Institute of Neurology, he has co-directed the MRC Centre for Neuromuscular Diseases at UCL.

 

Professor Muntoni’s main interests are in pathogenesis, deep phenotyping, gene identification for rare neuromuscular conditions and translational research in Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA). He is involved in several natural history studies and clinical trials and is the UK Chief Investigator for clinical trials on DMD and SMA, including adeno associated virus trials. His research has led to the development and early clinical trials of two morpholino antisense oligonucleotides, now approved by FDA, that induce partial correction of the processing defect of the DMD gene in boys with Duchenne muscular dystrophy.

Dr Simon Jones

Medical Director, NIHR Manchester Children’s Clinical Research Facility

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Professor Mimoun Azzouz

Director of the Gene Therapy Innovation and Manufacturing Centre and Director of Research and Innovation, University of Sheffield

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Professor Jan Kirschner

Medical Director in the Department of Neuropediatrics, University Hospital Bonn

Jan Kirschner is Medical Director in the Department of Neuropaediatrics at the University Hospital Bonn, Germany, with a long-standing interest in the diagnosis and treatment of neuromuscular disorders in children. His main research activities include planning and conducting clinical trials that test innovative treatments for neuromuscular disorders.

 

Jan is a member of the Executive Committee of the European Reference Network for Neuromuscular Diseases EURO-NMD and has been joint coordinator of the German Network for Neuromuscular Disorders MD-NET since 2008. He recently established the SMArtCARE network to collect real-world data from patients with spinal muscular atrophy.

Professor Hildegard Büning

President of the European Society of Gene and Cell Therapy and Professor of Infection Biology & Gene Transfer, Hannover Medical School

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Ms Anne Black

Regional QA Specialist Pharmacist, North-East and North Cumbria

Anne Black is Regional QA Specialist Pharmacist at the Pharmacy Quality Control/Production Unit at Newcastle upon Tyne Hospitals. She developed her expertise and interest in advanced therapy medicinal products as she became involved with the quality management of Newcastle Advanced Therapies. She has since used this expertise to emphasise the important role of pharmacy to optimise patient safety in the delivery of these medicines.

Anne spent most of her career in NHS Quality Assurance at Newcastle upon Tyne Hospitals, before becoming Regional QA Specialist for the North East and North Cumbria in July 2017. She is currently Chair of the NHS Pan-UK Pharmacy Working Group for ATMPs and a steering group member of the Northern Alliance Advanced Therapy Treatment Centre. Anne also sits on the NHS Pharmaceutical QA Committee and the National Pharmacy Clinical Trials Advisory Group.

Ms Shingirai Mutamba

Pharmacist, University Hospital Southampton

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Ms Jocelyn Walters

Research Delivery Manager, University Hospital Southampton

Jocelyn Walters is Research Delivery Manager at the University Hospital Southampton overseeing cancer, paediatrics, genetics, haematology, reproductive health and childbirth across Wessex. She began her career as a translational researcher at University College London, and in 1992, moved to the USA to set up and run a portfolio of paediatric clinical trials with a focus on neonatal bone mineralization at the University of Tennessee, Memphis.  She returned to London in 1995 to coordinate an international antenatal screening trial at the Institute of Preventative Medicine, St Bartholomew’s Hospital, London, before joining the University of Southampton in 2000 to lead the CR-UK Clinical Research Team. In 2002 she established the Central South Coast Cancer Research Network, one of the first cancer research networks in the UK and led this until 2014 when the NIHR was restructured. Since then, she has managed research at the NIHR and overseen the Southampton Cancer Research team. In 2019 she was awarded an MBE by the Queen for services to cancer research.

Ms Katie Groves

Senior Research Nurse at Great Ormond Street Hospital

Katie Groves is a Senior Research Nurse at the NIHR Clinical Research Facility at Great Ormand Street Hospital (GOSH), specialising in neuromuscular research. She has been a qualified nurse for 13 years and has worked in research for nine years, in which time she has set up and run a large portfolio of clinical trials from early phase to phase 3.

Katie has opened and conducted gene therapy trials that deliver treatment to children with spinal muscular atrophy (SMA) and set up gene therapy studies to deliver treatment to patients with Duchenne muscular dystrophy (DMD). She works with the multidisciplinary team at GOSH, as well as other organisations including pharmaceutical companies, advocacy groups and charities. She is currently completing her Advance Clinical Practice (ACP) training and is managing a portfolio of over 15 open trials with 30 in set up.

Ms Helen Dunn

Consultant Nurse and Director for Infection Prevention & Control, Great Ormond Street Hospital

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Dr Bryan Pukenas

Assistant Professor of Radiology, Hospital of the University of Pennsylvania

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Professor Laurent Servais

Professor of Paediatric Neuromuscular Diseases, MDUK Oxford Neuromuscular Centre

Laurent Servais is Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre and Invited Professor of Child Neurology at Liège University.

After graduating from Louvain Medical School, Brussels in 1999, Laurent completed a PhD in Neuroscience from Free University of Brussels, followed by residencies in child neurology at the Free University of Brussels and Robert Debré Hospital, Paris. In 2008, he took a position in neuromuscular disease and clinical research at the Institute of Myology in Paris, where his interest and expertise in neuromuscular diseases flourished. He was subsequently appointed Head of Clinical Trials and Database Services. Most recently, Laurent served as Head of the Institute of Myology’s I-Motion (Institute of Muscle-Oriented Translational Innovation), and Head of the Neuromuscular Centre in Liège, Belgium. He joined MDUK Oxford Neuromuscular Centre and the University of Oxford in September 2019.

Laurent has been involved as principal investigator in numerous clinical trials to test treatments for Duchenne muscular dystrophy and spinal muscular atrophy (SMA). He is the leader of the Newborn Screening for SMA programme in southern Belgium where they are conducting a medico-economic analysis of newborn screening.

Ms Liz Ryburn

Support Team Manager, Spinal Muscular Atrophy UK (SMA UK)

Liz Ryburn is Support Team Manager at Spinal Muscular Atrophy UK (SMA UK), a charity and patient group that advocates for better health and social care with and for the spinal muscular atrophy community. SMA UK support many families who have a child with SMA type 1 and they have been actively engaged in advocacy for access to Zolgensma (a gene therapy medicine) with NICE and the SMC, presenting community views on access to this treatment.

 

Liz has an extensive professional background in social work and disability rights in both the voluntary and statutory sectors. She gained her MA in Social Policy and Social Work from the London School of Economics and has worked as a Probation Officer in Brixton, and in children’s services for South Warwickshire Health and Social Services, where she also developed supported living options for people with learning disabilities. In Christchurch, New Zealand she worked as a paediatric social worker, Executive Officer at the Champion Centre Early Intervention Centre and Support Services Manager for the Canterbury and West Coast Branch of CCS Disability Action. For the last ten years she has been Support Team Manager at SMA UK.

Professor Volker Straub

Director of the John Walton Muscular Dystrophy Research Centre, Newcastle University

Volker Straub is a Professor of Neuromuscular Genetics at Newcastle University. He trained as a paediatric neurologist at the University of Dussledorf and the University of Essen, Germany. Following this, he completed his PhD on Duchenne muscular dystrophy (DMD) and took up a postgraduate post in Dr Kevin Campbell’s laboratory at the University of Iowa, on limb girdle muscular dystrophies (LGMD). In 2003, he joined Newcastle University as the Deputy Director of the Institute of Genetic Medicine (until 2019), and now leads the John Walton Muscular Dystrophy Research Centre.

Professor Volker has interests in the pathogenesis of genetic muscle diseases and translational research. He was the co-founder of the EU FP6 funded network of excellence for genetic neuromuscular diseases, TREAT-NMD. He is the Chief Investigator/Principal Investigator for a number of natural history and interventional trials in DMD, LGMD, Pompe disease, spinal muscular atrophy and other NMDs. He is currently the president of the World Muscle Society and author of over 375 peer-reviewed publications.

Dr Savita Rangarajan

Associate Professor of Clinical Haematology and Honorary Consultant, University of Southampton

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Agenda

Site preparation for AAV therapies

Round table discussion

9.10 – 9.25
9.25 – 9.40

Case study

9.40 – 9:50

Case study

9.50 – 10.00
10.00 – 10.10

Clinical experience with AAV in different conditions

Different modalities to administer AAV

10.25 – 10.40
10.40 – 10.55

Case studies and advocacy groups perspectives

10.55 – 11.05
11.05 – 11.15

Preparing the patients and the post AAV pathways

Round table discussion

11.30 – 12.10
  • Implementing a vaccincation schedule
  • Pre-treatment with steriods – when is it necessary?
  • Duration of steriod treatment and tapering regimens
  • Other immosuppresive regimes that are in use
  • What to monitor to assess the incidence and severity of adverse events in the first few weeks and months of treatment
  • Which contingency plans should be available in case of SUSARs
  • Different versions of the hub-and-spoke model

Agenda

Site preparation for AAV therapies

Round table discussion

9.10 – 9.25
9.25 – 9.40

Case study

9.40 – 9:50

Case study

9.50 – 10.00
10.00 – 10.10

Clinical experience with AAV in different conditions

Different modalities to administer AAV

10.25 – 10.40
10.40 – 10.55

Case studies and advocacy groups perspectives

10.55 – 11.05
11.05 – 11.15

Preparing the patients and the post AAV pathways

Round table discussion

11.30 – 12.10
  • Implementing a vaccincation schedule
  • Pre-treatment with steriods – when is it necessary?
  • Duration of steriod treatment and tapering regimens
  • Other immosuppresive regimes that are in use
  • What to monitor to assess the incidence and severity of adverse events in the first few weeks and months of treatment
  • Which contingency plans should be available in case of SUSARs
  • Different versions of the hub-and-spoke model

Headline Sponsor

Sponsors

7 September 2021 / 09.00 – 12.15

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