UK health system to offer cutting-edge gene therapy for sickle cell disease

Crispr Therapeutics AG

31/01/2025 24:01

LONDON, Jan 31 (Reuters) - Britain's National Health
Service (NHS) will provide a cutting-edge gene therapy that aims
to cure sickle cell disease, the National Institute for Health
and Care Excellence (NICE) said on Friday.
    The therapy from Vertex Pharmaceuticals  VRTX.O  and CRISPR
Therapeutics  CRSP.BN  will cost the state-funded healthcare
system around 1.65 million pounds ($2.1 million) per course.
    Britain's MHRA medical regulator in 2023 became the first in
the world to approve the therapy, which uses the gene-editing
tool CRISPR.
    NICE, which assesses whether new medical technologies can be
used in the NHS, said the treatment would be suitable for around
50 patients per year.
    Sickle cell disease is a serious and lifelong condition
caused by errors in the genes for haemoglobin, which is used by
red blood cells to carry oxygen around the body.
    The therapy involves taking stem cells out of a patient's
bone marrow and editing a gene in the cells in a laboratory and
then infusing them back into the patient.
    The same technology was approved for use in the NHS in
August to treat beta thalassemia, another rare blood disorder.

($1 = 0.8039 pounds)

 (Reporting by Sam Tabahriti, Editing by William James and
Sachin Ravikumar)
 ((sam.tabahriti@thomsonreuters.com; +447585976686;))