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Cell and Gene Therapy Catapult Publishes National Cell and Gene Therapy Vision

on Wednesday, 06 April 2022.

The UK's Cell and Gene Therapy Catapult has published a new National Cell and Gene Therapy Vision.

The Vision highlights the exciting opportunities from cell and gene therapies, but also explains some challenges, as well as recommendations to overcome those and keep the UK at the forefront of these cutting-edge treatments.

Cell and gene therapies are exciting new treatments that introduce, remove or change the content of a person's genetic code. They can save multiple invasive treatments and are sometimes described as miracle cures. However, because of the highly personalised nature of the treatments, they are not manufactured at scale, which can make them costly. In addition, the manufacture need to be in specialised centres and close to the place where the patient is treated. Furthermore, highly specialised people are needed in the treatment and in the manufacture. Data needs to be gathered and shared in a better way to help to understand the impact of the treatments.

The Recommendations

Amongst the recommendations of the document are as follows:

  • the development of a UK advanced therapy manufacturing sector
  • enhancing the knowledge and skills of the workforce to prepare for future therapies
  • expanding treatment centre capacity through existing centres, as well as additional new ones
  • NICE and the Scottish Medicines Consortium to consider how their assessments may need to change. Plus more flexibility and use of real world evidence incorporated into decision making
  • effective horizon scanning to prepare the NHS and industry for novel therapies. This will include insights from manufacturers and input from across regulatory, pricing and clinical adoption decision makers
  • attracting further global commercial investment into clinical research. This includes enhancing the NHS's attractiveness as a place to conduct trials as more advanced therapies are introduced
  • strengthening the NHS's data infrastructure to inform continued improvements to clinical practice and strengthen real world evidence
  • improve patient care, education and support

The Stakeholders' Roles

The paper refers to important roles for the following:

  • The Government are to oversee the implementation of a vision and act on commitments from the 2021 Life Sciences Vision. The Department for Health and Social Care will have a role in setting timelines and delivery metrics for each party.
  • NICE and the SMC will need to work in close collaboration with the MHRA, as well as showing flexibility in their approach.
  • The MHRA must adopt innovation in its approach to regulating new medicines, including through the use of the ILAP (Innovative Licensing and Access Pathway) procedure.
  • The NHS will need to carry out horizon scanning to see where and how new treatments are likely to come, and plan future workforce needs to ensure sufficient expertise and capacity across the country to deliver advanced therapies, as well as collect robust data on long-term outcomes to demonstrate the value of the new therapies.
  • Industry must ensure data demonstrates the value for treatments to be approved, and engage early to ensure horizon scanning.
  • Patient and charity groups must educate patients about the therapies.

George Freeman MP, the Minister for Science, Research and Innovation, says: “The UK Cell and Gene Catapult has played a key role in developing UK leadership in advanced therapies. It is no coincidence that 9% of all Advanced Therapy Medicinal Products clinical trials are run in the UK, and that the UK has the third largest cluster for cell and gene therapies in the world. This National Cell and Gene Therapy Vision supports our ambition to cement the UK’s position as a world leader in advanced clinical research and development, while helping to accelerate access both for patients to new therapies and researchers to patients.”

Matthew Durdy, CEO of the Catapult, comments: “The National Cell and Gene Therapy Vision for the UK will help ensure the UK continues its progress in bringing these life-changing therapies to patients. Crucial to this effort is the Advanced Therapy Treatment Centre network, coordinated by the CGT Catapult, which will continue to drive innovation and support the clinical adoption of ATMPs through close collaboration between industry and the NHS. The expert recommendations, along with continued investment in the sector, will ensure that the NHS is prepared to deliver the next generation of ATMPs, and UK retains its status as a world leader in the delivery of these therapies.”

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Hear More About the UK's Global-Leading Role in Life Sciences Innovation

VWV are hosting the 2022 PING Conference on 20 June in the beautiful setting of The Old Palace at Hatfield House. The event is focused on why this time is being described as the golden age for life sciences innovation, and the UK is uniquely position to adopt innovations. One such area is in cell and gene therapies. George Freeman is one of the speakers at the Conference. Another is Matthew Whitty, the NHS England Director of Innovation, Research and Life Sciences and Chief Executive of The Accelerated Access Collaborative. The new NICE Chief Executive, Samantha Roberts, will also be presenting.

Just two years ago, at the 2020 PING Conference, we heard from Matthew Durdy about the fast-growing developments at the CGT Catapult and the opportunities in the area. It is no surprise to see the UK continuing apace in making the running to meet the challenges posed by the opportunities for these new opportunities.


If you would be interested in being invited to attend the 2022 PING Conference, please contact Paul Gershlick in our Pharmaceuticals and Life Sciences team on 07795 570072, or complete the form below.

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