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Vertex Resolves To Reverse English Funding Rejection For CRISPR Sickle Cell Gene Therapy

 
• By Neena Brizmohun

The health technology assessment institute, NICE, is not yet ready to recommend Casgevy for sickle cell disease and says it wants more data. Meanwhile, an access agreement relating to the treatment’s use for transfusion-dependent β-thalassemia is making progress in England, as are reimbursement talks for SCD in other European countries.

3d graphic of sickled blood cell
Casgevy is a one-off gene therapy for sickle cell disease • Source: Shutterstock

Vertex is hoping to reverse NICE’s interim decision to recommend against funding its CRISPR gene editing therapy, Casgevy, for severe sickle cell disease (SCD) in England because of uncertainties over the clinical and economic evidence submitted.

The company, which developed the one-off treatment with CRISPR Therapeutics, said it was disappointed by the health technology assessment institute’s...

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More from United Kingdom

UK MHRA: Bacteriophage Developers Wanted ‘More Clarity’ On UK Framework

 
• By Eliza Slawther

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MHRA Backs Bacteriophage Innovation To Fight Antimicrobial Resistance

 
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Scientists Work With AI To Uncover ‘Unexpected’ Drug Combos For Breast Cancer

 
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More from Europe

EU Pharma Reform Package Clears Big Political Hurdle After Council Adopts Position

 
• By Neena Brizmohun

The Council of the EU has made several key amendments to the legislative reform package, which it will now negotiate with the European Parliament.

MHRA Backs Bacteriophage Innovation To Fight Antimicrobial Resistance

 
• By Eliza Slawther

The UK regulator wants to help companies to develop bacteriophages for the treatment of infections. Its first guidance on this topic offers advice to researchers and sets out the regulatory requirements they will need to meet.

Scientists Work With AI To Uncover ‘Unexpected’ Drug Combos For Breast Cancer

 
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