sickle cell disease

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Agios' chief medical officer, Sarah Gheuens

Agios mulls early FDA filing for sickle cell therapy

Agios is planning to file its sickle cell disease candidate mitapivat with the FDA under the accelerated pathway within the next few months.

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Agios shares slump on sickle cell disease data

Shares in Agios lost nearly half their value after it posted mixed clinical results from a phase 3 trial of its sickle cell disease therapy mitapivat.

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Pfizer suffers another hit to its GBT sickle cell portfolio

Pfizer's $5.4 billion takeover of Global Blood Therapeutics three years ago is starting to look like a poor deal.

Digital

Enhancing rare disease trials: eCOA technology’s impact on s...

Sickle cell disease (SCD) affects an estimated 20 million people worldwide.

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Sickle cell gene therapy Casgevy finally cleared for NHS use

Vertex's gene-editing therapy for sickle cell disease, Casgevy, has been given the nod for use by the NHS in England after an earlier rejection.

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Editas axes two-thirds of its staff, including its CMO

Editas Medicine ends development of its gene-editing therapy for thalassaemia and sickle cell, shedding 65% of staff and narrowing its R&D focus.

Agios mulls early FDA filing for sickle cell therapy

Agios shares slump on sickle cell disease data

Pfizer suffers another hit to its GBT sickle cell portfolio

Enhancing rare disease trials: eCOA technology’s impact on s...

Sickle cell gene therapy Casgevy finally cleared for NHS use

Editas axes two-thirds of its staff, including its CMO

Ambrosia's sweet $100m round, and other biofinancings

Using AI-driven synthetic personas to take your insights fur...

sickle cell disease

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