April 6


02:00 pm - 03:00 pm

Event Category:


Gene and cell therapies have the potential to be one of the most significant success stories of the twenty-first century. The dream of a cure is now within the reach of some patients. Many genetic diseases were once seen as incurable and embedded into the DNA of those affected.

The UK is at a crossroads. As the country exits the EU and with the NHS under pressure from the pandemic there are lingering questions about whether the promise of the treatment pipeline will translate into treatments for the thousands of patients suffering from long-term and rare conditions.

Despite these challenges there is cause for optimism. Life sciences has been identified as a key building block of the future UK economy, and a new regulatory system being developed by the MHRA and being supported with changes to the HTA process. These changes are underpinned by the Life Sciences Vision and the Rare Disease Framework, and together this suite of life sciences policy provides the opportunity for the UK to embrace ATMPs and become the world leader in both their development and use.

This is a new era of medicine and one we should not view as a challenge, but as a huge opportunity to treat people who would otherwise not have access to therapies. It is an opportunity that those who contributed to this white paper are ready to embrace.

About the report: Advanced Treatments | Gene and Cell Therapy

Public Policy Projects’ latest report will explore how the impact the introduction of cell and gene therapies will have, through the following three areas:

  • The Patient Voice
  • Considering the Economics of Advanced Therapies in Rare Disease
  • New Treatments for a Modern Age


  • Samantha Barber, CEO, Gene People
  • Sheela Upadhyaya, Rare Diseases & RAPID-C19 Strategic Advisor, Centre for Health Technology Evaluation, National Institute for Health and Care Excellence (NICE)
  • Dr Panos Kefalas, Director of Access Strategy, Cell and Gene Therapy Catapult

The report ‘Advanced Treatments | Gene and Cell Therapy‘ was supported by sponsorship from Pfizer, Gilead and BMS. Public Policy Projects has retained full editorial control.

To attend this report launch on Zoom please register here.

  • 06 Apr, 2022 14:00 - 15:00 BST
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Public Policy Projects is part of the Dorson Inform Group of companies which includes Dorson Transform.

Publications associated with PPP