sickle cell disease

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Agios shares slide as it abandons sickle cell candidate

Agios Pharma has said it will halt clinical development of tebapivat, a PK activator that had reached phase 2 testing in sickle cell disease.

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Vertex gets paediatric FDA okay for Casgevy gene therapy

For the first time, children in the US as young as two with blood disorders sickle cell disease and beta thalassaemia are eligible for gene therapy.

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Novo Nordisk preps filings for sickle cell drug etavopivat

Novo Nordisk has claimed a key win in its head-to-head race with Agios to bring an oral PKR activator to the market for 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.

Agios shares slide as it abandons sickle cell candidate

Vertex gets paediatric FDA okay for Casgevy gene therapy

Novo Nordisk preps filings for sickle cell drug etavopivat

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

Vogenx surpasses expectations with $81M IPO

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sickle cell disease

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