UK's NHS to roll out Vertex/CRISPR gene therapy for rare blood disorder (updated)

Crispr Therapeutics AG

08/08/2024 01:19

(Updates with details from statement in paragraphs 2-6, updates
with background in paragraphs 7-8)
       Aug 7 (Reuters) - Britain's National Health Service
(NHS) will begin to roll out a gene therapy from Vertex
Pharmaceuticals  VRTX.O  and CRISPR Therapeutics  CRSP.BN  to
treat rare blood disorder beta thalassemia, Vertex said in a
statement on Wednesday. 
    Casgevy, which requires administration through authorized
treatment centers with experience in stem cell transplantation,
will be made available to eligible patients from Aug. 7, the
company said. 
    The agreement between Vertex and the NHS comes as the
National Institute for Health and Care Excellence (NICE) issued
positive guidance recommending Casgevy's use, Vertex said.
        About 460 patients in England with transfusion-dependent
beta thalassaemia aged 12 and older are potentially eligible for
the treatment, which uses gene-editing technology, the NHS said
in a statement. 
        The NHS said the therapy will be manufactured in the UK,
and is set to be offered at seven of its specialist centres.
    The UK list price for the therapy stands at 1.65 million
pounds ($2.09 million), a spokesperson for Vertex said, adding
that the company cannot comment on a negotiated price. 
    Britain's medical regulator had authorized Casgevy
CRISPR-based gene-editing therapy last November for the
treatment of  sickle-cell disease and another type of inherited
blood disorder for patients aged 12 and over. 
    The medicine is administered by taking stem cells out of a
patient's bone marrow and editing a gene in the cells in a
laboratory, with the modified cells then infused back into the
patient after conditioning treatment to prepare the bone marrow.
         
    ($1 = 0.7890 pounds)
    
    

 (Reporting by Gursimran Kaur and additional reporting by Surbhi
Misra in Bengaluru; Editing by Anil D'Silva and Sherry
Jacob-Phillips)
 ((GursimranKaur.Mehar@thomsonreuters.com;))