Agenda: 12:30-12:35 – Welcome and introduction of the TINs (Asha Recino, Therapeutic Innovations Networks Coordinator) 12:35-12:45 – Patient and Public Involvement (PPI) in Research - An introduction (Linda von Nerée, PPI Lead at NIHR Blood and Transplant Research Unit (BTRU) in Stem Cells and Immunotherapies at UCL) 12:45-12:55– PPI engagement and benefits in gene therapy research (Matthew Walker, Consultant Neurologist at the National Hospital for Neurology and Neurosurgery) 12:55 -13:05 – Patient experience and involvement in hemophilia gene therapy studies (Luke Pembroke, Gene Therapy Patient) 13:05 -13:15 – Patient involvement in stem cell and immunotherapies for blood cancer (Gillian Murphy, Patient & Public Research Panel Member, Blood and Transplant Research Unit at UCL) 13:15- 13:25 – PPI in pre-clinical regenerative medicine research (Nick Hamilton, NIHR Clinical Lecturer, UCL) https://www.ucl.ac.uk/therapeutic-innovation-networks/join-community/subscribe-tins-newsletter

90

4/30/2021

This Cell & Gene Therapy TIN event hosted by COVANCE, covers considerations for nonclinical development of viral vectored gene therapies. Dr Robert Evans (Associate Drug Development Leader, COVANCE), Dr Bruce Hamilton (Drug Development Leader, COVANCE), Dr Keith McLuckie (Associate Drug Development Leader, COVANCE) and Jenny Pope (Senior Business Development Director, COVANCE) will guide you through the pivotal GLP toxicology and biodistribution studies, which are crucial for progressing gene therapy candidates in to the clinical setting. Also hear an academic’s perspective’ as Prof. Stephanie Schorge (SoP, UCL) and Prof. Paul Gissen (Dept of Genetics & Genomic Medicine, UCL) will describe practical steps which could make the process more manageable. Please note, this COVANCE recording is intended for educational purposes only, and cannot be copied, repurposed, or published elsewhere. https://www.ucl.ac.uk/therapeutic-innovation-networks/join-community/subscribe-tins-newsletter

50

3/25/2021

This educational webinar will cover the practical aspects of using Adeno-Associated Viruses (AAV) and lentiviruses to deliver and express Your Favourite Gene (YFG) in vivo for both basic research and pre-clinical studies of gene therapy treatments. With expertise in translational neuroscience, Professor Stephanie Schorge, Dr Ahad Rahim and Dr Giulia Massaro, will highlight tips and tricks to help any researcher use viral delivery for basic and translational neuroscience. This will include: How to design a viral vector and which capsid to use What is needed to make viral particles How many are needed for different studies What resources are available for help, including the new UCL GeneTxNeuro Facility based at the UCL School of Pharmacy.

25

9/29/2020

This educational webinar recording covers the practical aspects of using Adeno-Associated Viruses (AAV) and lentiviruses to deliver and express Your Favourite Gene (YFG) in vivo for both basic research and pre-clinical studies of gene therapy treatments. With expertise in translational neuroscience, Professor Stephanie Schorge, Dr Ahad Rahim and Dr Giulia Massaro, highlight tips and tricks to help any researcher use viral delivery for basic and translational neuroscience. This includes: How to design a viral vector and which capsid to use What is needed to make viral particles; How many are needed for different studies; What resources are available for help, including the new UCL GeneTxNeuro Facility based at the UCL School of Pharmacy GeneTxNeuro is a facility that gives all UCL researchers access to the equipment and expertise needed to make AAV and lentis for research and preclinical work. The webinar highlights the services and benefits offered by the facility.

24

9/25/2020