Red blood cell, CRISPR gene editing

NHS to offer 'groundbreaking' sickle cell gene therapy
The gene therapy Casgevy, which will now be offered to some patients in England, could help people live disease-free, experts say.
UK health system to offer cutting-edge gene therapy for sickle cell disease
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.
Groundbreaking one-off gene therapy approved for severe sickle cell disease
People in England to benefit from groundbreaking one-off gene therapy for severe sickle cell disease approved by NICE
Revolutionary gene-editing therapy for sickle cell ‘offers hope of a cure’ for NHS patients
NHS patients with sickle cell disease will be able to benefit from a groundbreaking gene-editing treatment that offers the prospect of a cure for the condition. The one-off gene therapy, known as exagamglogene autotemcel (or ‘exa-cel’),
NHS England to offer Casgevy for sickle cell disease patients
The UK’s Medicines and Healthcare Regulatory Agency (MHRA) approved Casgevy for sickle cell disease in 2023, followed by approval for transfusion-dependent beta-thalassemia (TDT) in January 2024. NHS England reached an earlier reimbursement agreement for Casgevy in TDT in August 2024.
Sickle cell disease ‘cure’ to be offered to NHS patients
Around 1,700 people could be eligible for the one-time treatment, which is seen as a less risky alternative to a donor stem cell transplant
Vertex In CASGEVY Reimbursement Deal With NHS England For Sickle Cell Disease Patients
Vertex Pharmaceuticals Inc. (VRTX), a commercial-stage global biotechnology company, announced Friday a reimbursement agreement with
Vertex wins reimbursement deal for CRISPR-partnered sickle cell therapy in England
Vertex Pharmaceuticals (VRTX) stock gains as England opts to reimburse its CRSPR (CRSP)-partnered gene therapy Casgevy to treat sickle cell disease. Read more here.
Vertex Announces CASGEVY® Reimbursement Agreement for the Treatment of Sickle Cell Disease in England
Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with NHS England for eligible sickle cell disease (SCD) patients to access the CRISPR/Cas9 gene-edited therapy, CASGEVY ® (exagamglogene autotemcel).
Vertex, U.K. reach reimbursement deal for CRISPR-based medicine for sickle cell disease
People in England with sickle cell will be able to get the world’s first CRISPR-based medicine under an agreement between the U.K. and Vertex.
‘Much-needed hope’ as CRISPR gene editing therapy recommended for sickle cell disease
Experts hailed exciting news for some people with sickle cell disease in England as the health watchdog decided to grant ...
NBC News on MSN13d
'Will it really work?': Young sickle cell patient among the first to start new gene therapy
Since two groundbreaking treatments were approved in late 2023, only a handful of patients have been able to access the ...
Sickle cell patients share delight over approval of new treatment
NHS officials estimate that around 50 people a year will receive treatment now that it has been approved for use for certain ...
‘Sickle cell pain is the worst I have ever felt in my life - it’s hard to put into words'
Everything you need to know about agonising sickle cell disease and the ‘revolutionary’ new DNA therapy that's being rolled ...
ENDPOINTS NEWS7h
Af­ter more de­lib­er­a­tion, Eng­land agrees to pay for Ver­tex’s gene ther­a­py for sick­le cell dis­ease
NHS England & Vertex reach deal for sickle cell gene therapy Casgevy, making it available at an undisclosed reduced price ...
Hosted on MSN9h
Revolutionary gene-editing therapy for sickle cell approved
Billie Eilish and Green Day kicked off the FireAid benefit concert in Los Angeles on Thursday night - a two-venue, five-hour ...