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.
How AI is streamlining cell and gene therapy manufacturing
AI could deliver substantially greater efficiencies in cell and gene therapy manufacturing and enable more patients to benefit.
'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 multimillion dollar, medically-intensive therapies.
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
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. The therapy from Vertex Pharmaceuticals and CRISPR Therapeutics will cost the state-funded healthcare system around 1.
NHS to offer 'groundbreaking' sickle cell therapy as patients given 'very real prospect of cure'
A groundbreaking £1.65million gene therapy treatment offering hope for sickle cell disease patients has been approved for use on the NHS in England. The one-time treatment, called Casgevy (exa-cel), has shown remarkable success in clinical trials,
Revolutionary gene-editing therapy for sickle cell approved
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‘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 access to gene-editing therapy on the NHS. View on euronews
Managed Healthcare Executive3h
Gene Therapy’s Pipeline Has a Steady Flow of More Than 2,000 Candidates
The majority are in the very early preclinical stage of development, but applications for regulatory approval have been ...
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Gene Therapy Stabilizes Rare Chronic HPV Disease
For recurrent respiratory papillomatosis (RRP), novel gene therapy to jump-start immunity to the human papillomavirus (HPV) ...
Gene therapy and bone marrow transplant alleviate ultra-rare genetic disease symptoms
In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and ...
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CMS Cell and Gene Therapy Access Plan Earns Reprieve After Trump Halt
President Trump signed an executive order on the day of his inauguration that created doubts about the future of a program to ...
Idaho Republican pushes ban of mRNA vaccines against COVID, gene therapy products
The bill would implement a moratorium for a decade. “I am not against vaccination in any way,” the freshman lawmaker said.
Gene therapies have been uneven for DMD — but these companies hope to turn the tide
Despite mixed results using gene therapies to treat Duchenne muscular dystrophy, drug developers are pushing ahead with the ...
Taysha Gene Therapies Is Showing Promise For Rett Syndrome
Taysha Gene Therapies offers hope for Rett syndrome with innovative treatment, strong trial results, and promising regulatory ...
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Rentschler closes UK cell and gene therapy facility and does a pivot to biologics
CDMO Rentschler Biopharma is signalling its intent to cut down cell and gene therapy operations by shutting down its UK manufacturing facility in Stevenage.