Rare Diseases: Gene Therapies

(asked on 9th December 2025) - View Source

Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what steps his Department is taking to (a) support the development of treatments for Huntington's disease and (b) improve the ability of the NHS to deliver new gene therapies for people living with rare diseases.


Answered by
Zubir Ahmed Portrait
Zubir Ahmed
Parliamentary Under-Secretary (Department of Health and Social Care)
This question was answered on 17th December 2025

We recognise the significant challenges faced by those living with rare diseases such as Huntington’s Disease. The Department supports research into Huntington’s disease through the National Institute for Health and Care Research (NIHR). NIHR infrastructure supports pioneering research into Huntington's, including the positive preliminary results for a novel gene therapy reported this year. NHS England will assess the service delivery impact of any specific gene therapy for Huntingdon's disease within three years of its expected licensing decision by the Medicines and Healthcare products Regulatory Agency. The National Institute for Health and Care Excellence (NICE) will make recommendations for the National Health Service on new medicines based on clinical and cost effectiveness. NHS England is required to fund medicines recommended by NICE, within three months of the publication of final guidance. The NHS has a dedicated team to support the adoption of advanced therapy medicinal products (ATMPs) that are recommended by NICE. NHS England works with a variety of internal and external stakeholders to ensure timely patient access to ATMPs that are on NICE’s technology appraisal and highly specialised technology workplan.

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