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FDA probing cancer cases with Bluebird's CALD gene therapy

The FDA is scrutinising reports of haematological cancers in patients treated with bluebird bio's Skysona gene therapy for neurological disease CALD

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New film aims to raise awareness of rare disease cALD

Rare Disease Day saw the debut of a film that aims to raise awareness of cerebral adrenoleukodystrophy (cALD), a devastating and fatal condition with no pharmacological tr

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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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Vertex Pharmaceutical's Boston headquarters

FDA clears two sickle-cell gene therapies, including CRISPR ...

The US FDA has cleared two novel gene therapies for sickle cell disease: Vertex Pharmaceuticals' Casgevy and bluebird bio's Lyfgenia.

News

FDA sets Dec deadline for Vertex, CRISPR sickle cell therapy

The FDA has started a priority review of Vertex Pharma and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel) in sickle cell disease (SCD), with a decision on approva

News

ICER backs sickle cell gene therapies at one-off cost of $2m

The influential Institute for Clinical and Economic Review (ICER) in the US has issued draft guidance suggesting that new gene therapies for sickle cell disease (SCD) from

FDA probing cancer cases with Bluebird's CALD gene therapy

New film aims to raise awareness of rare disease cALD

First CRISPR drug Casgevy gets swift second approval

FDA clears two sickle-cell gene therapies, including CRISPR ...

FDA sets Dec deadline for Vertex, CRISPR sickle cell therapy

ICER backs sickle cell gene therapies at one-off cost of $2m

World Without Disease initiative: 2024 update

The AI effect in pharma commercial strategy: From data to re...

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