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To mark World Haemophilia Day, we sit down with Professor Amit Nathwani of the UCL Cancer Institute to explore his journey to commercialising the groundbreaking haemophilia A treatment, Roctavian.
Host Nigel Campbell is joined by Prof Nathwani and Dr. Rick Fagan, UCLB Director of Biopharm, to explore how Amit’s work looking after patients with haemophilia in the NHS instilled a mission to develop a 'one and done' vaccine type approach to the condition.
Amit’s lab to market story, working with UCLB, led to the creation of Roctavian, a one-time gene therapy used for the treatment of adults with severe haemophilia A. The therapy, which was licensed to BioMarin Pharmaceutical Inc, received marketing approval in Europe in 2022 and US FDA approval in 2023, and is now available to patients commercially.
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4/15/2024
This is an information session about the MSc Genetics of Human Disease. You will hear from the course leaders about the programme and the career opportunities that this can give you.
1
2/20/2024
Dr Qasim Rafiq and Dr Rana Khalife discuss cell and gene therapy. Dr Rafiq is an Associate Professor in Cell and Gene Therapy Bioprocess Engineering at UCL, and is also programme director for the Manufacture and Commercialisation of Cell and Gene Therapies MSc at UCL Biochemical Engineering. Dr Khalife is one of the lecturers on the programme.
91
6/16/2021
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
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4/30/2021
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.
24
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