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approval

Bluebird faces longer wait for FDA verdict on experimental gene therapies

Dive Brief: Bluebird bio will need to wait longer for a decision from the Food and Drug Administration on approval of two gene therapies it …

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blood-clotting

Fresh data show UniQure’s hemophilia gene therapy appears to hold up

Gene therapies have the potential to essentially fix many diseases. Yet, those currently in development are administered just once, raising questions about whether their effects …

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brain

A startup led by Novartis veteran raises $80M to develop gene therapies for the brain

The U.K. startup has aims to improve on neurodegenerative therapy by using technology developed in a King’s College laboratory run by Christopher Shaw. Shaw is …

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cell and gene therapies

Regulatory Challenges Associated with the Development of Cell and Gene Therapies

The development of safe and effective cell and gene therapies (CGTs) relies on regulatory compliance and critical selection and qualification of raw materials. Regulatory classifications …

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cellular machinery

Viral Vector Engineering to Improve Clinical Performance and Accelerate Timeline to Success for Novel Gene Therapies

Viruses are known to be dangerous pathogens which invade host cells and hi-jack their cellular machinery to direct the replication and transcription of their own …

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