Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what steps he is taking to support the development of gene therapy treatments for rare genetic diseases such as tyrosinemia and Gaucher disease.

The Government is committed to improving the lives of those living with rare diseases, such as tyrosinemia and Gaucher disease. Through the UK Rare Diseases Framework, the Government supports access to specialist care, treatment, and drugs as a priority. This has included NHS England developing a strategic approach to commissioning advanced therapy medicinal products (ATMPs) to facilitate rapid access to National Institute for Health and Care Excellence approved therapies.

We recognise the transformative potential of gene therapies and other advanced therapy medicinal products (ATMPs) for rare diseases and have stood up an advanced therapies co-ordination group spanning public sector partners. This group aims to create a joined-up ecosystem that will support the development, regulation, and delivery of ATMPs in the United Kingdom. We continue to invest in ATMPs through initiatives such as the Cell and Gene Therapy Catapult. With £17.9 million of new funding via the National Institute of Health and Care Research (NIHR), the Advanced Therapy Treatment Centres is driving forward work to enhance the UK’s environment for ATMP clinical trials and to progress research within the National Health Service to ensure patients can get safe access to these innovative therapies.

NIHR’s ‘Be Part of Research’ allows individuals to find and take part in current research. There are currently a number of studies on tyrosinemia and Gaucher disease where researchers are actively looking for participants. Further information on NIHR’s ‘Be Part of Research’ is avaiable at the following link:

https://bepartofresearch.nihr.ac.uk/

This includes a clinical trial assessing the safety of a gene therapy for those with peripheral manifestations of Gaucher disease, with further information avaiable at the following link:

https://bepartofresearch.nihr.ac.uk/trial-details/trial-detail?trialId=55560&location=&distance=