• Work will commence in May, with publication expected in September 2024.
  • We will be accepting stakeholder contributions until 31 July 2024.
  • More information on how to contribute to POST research is available in our guidance for contributors.

Inherited blood diseases include anaemias (beta-thalassemia and sickle cell anaemia) and bleeding disorders (Von Willebrand and haemophilia). Advances in personalised therapies, such as gene editing, offer improved treatments and potential cures. In January 2024, the NHS offered personalised blood group genotyping, to improve outcomes from blood transfusions. In November 2023, the MHRA approved an innovative CRISPR gene editing therapy that could offer a cure for thalassemia and sickle cell anaemia. Whether these treatments will be determined to be a cost-effective treatment recommended for use on the NHS is contingent on a current National Institute for Health Care and Excellence (NICE) appraisal.

This POSTnote will outline the health burden of these diseases, explain key concepts of emerging treatments and examine perspectives regarding patient awareness and access to innovative medicines such as cell and gene therapy.

Work will commence in May, with publication expected in September 2024. We will be accepting stakeholder contributions until 31 July 2024.


By Odua Imagesstock.adobe.com).

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