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beta thalassaemia

This page shows the latest beta thalassaemia news and features for those working in and with pharma, biotech and healthcare.

Vertex to lead development of CRISPR Therapeutics-partnered gene therapy

Vertex to lead development of CRISPR Therapeutics-partnered gene therapy

According to the companies, seven patients with transfusion-dependent beta thalassaemia (TDT), including three who have either a severe or b0/b0 genotype, were transfusion independent at the last follow-up.

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