beta thalassaemia

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Chinese trial backs base-editing drug for thalassaemia

A landmark study in China has shown that a base-editing therapy developed by CorrectSequence achieved stellar results in beta-thalassaemia.

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After a no in sickle cell, NICE OKs Casgevy for thalassaemia

Vertex and CRISPR Therapeutics have won the backing of NICE for their gene-editing therapy Casgevy as a treatment for the blood disorder beta thalassaemia, a few months after turning it dow

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A giemsa stained blood smear from a person with beta thalassaemia

First CRISPR drug Casgevy gets swift second approval

The FDA has lost no time in approving Vertex Pharma and CRISPR Therapeutics pioneering gene-editing therapy Casgevy for its second use, approving the drug for transfusion-

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CHMP backs Biogen’s Skyclarys for Friedreich’s ataxia

Biogen’s Skyclarys is on course to becoming the first approved medicine for the inherited neurological disease Friedreich’s ataxia (FA) in the EU after it was recommended

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UK first to approve Vertex, CRISPR drug for blood disorders

The first-ever CRISPR-based gene-editing therapy has been approved for marketing in the UK for inherited blood disorders sickle cell disease (SCD) and transfusion-dependen

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