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Pfizer hemophilia gene therapy arrives in US to uncertain future

Bio Pharma Dive

The Food and Drug Administration approval of Beqvez comes as other gene therapies for the bleeding condition that were approved earlier struggle to gain traction.

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What’s next for AAV gene therapies in 2024?

Pharmaceutical Technology

Upcoming regulatory events for pipeline AAV gene therapies, with a focus on neuromuscular and blood disorders, are setting high expectations for the space in 2024.

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Cell and gene therapy companies trip at scalability hurdle

Pharmaceutical Technology

Experts hold scalability challenges and high costs accountable for market failures within the cell and gene therapy landscape.

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Pfizer receives FDA approval for haemophilia B gene therapy

Pharmaceutical Technology

Pfizer has received approval from the US Food and Drug Administration (FDA) for its one-time gene therapy BEQVEZ for haemophilia B.

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Making the Case for Case Management for Cell and Gene Therapies

As demand for advanced therapies increases, so does the need for more specialized supply chain support, as these products have strict transportation and handling requirements.

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Regeneron gene therapy improves hearing in two children

Bio Pharma Dive

The results add to an early but growing body of evidence suggesting gene therapy may improve outcomes in young kids with a genetic form of deafness.

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AskBio’s AB-1002 gene therapy receives FDA fast-track status

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted fast-track designation to Bayer subsidiary Asklepios BioPharmaceutical’s (AskBio) investigational gene therapy AB-1002, aimed at treating congestive heart failure (CHF).