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- PMLiVE

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- PMLiVE

FDA advisory committee backs Sarepta’s Duchenne muscular dystrophy gene therapy

The disease occurs in up to one in every 5,000 newborn males worldwide

- PMLiVE

bluebird bio submits US application for sickle cell gene therapy

The genetic disease affects approximately 100,000 people in the US alone

- PMLiVE

bluebird bio sells second FDA priority review voucher for $95m

The company sold its first voucher to Dutch biotech Argenx for $102m

- PMLiVE

Pfizer announces positive phase 3 results for haemophilia B gene therapy

A one-off, intravenous dose of the therapy candidate reduced bleeding by 71%

- PMLiVE

FDA lifts partial clinical hold on bluebird bio’s sickle cell gene therapy studies

The company can now resume the enrolment and treatment of patients aged two to 17 years

- PMLiVE

bluebird bio sells Rare Pediatric Disease Priority Review Voucher

The company was granted two PRVs upon the FDA approvals of Zynteglo and Skysona

- PMLiVE

Sarepta to assess imlifidase as pre-treatment to Duchenne muscular dystrophy gene therapy

The company submitted a Biologics License Application to the FDA for SRP-9001 in September

Cell and gene therapies as depicted via a DNA strand

The fast-paced future of cell and gene therapies

In this report, we interview patients, researchers, and biopharmaceutical leaders to explore the complex landscape for the development of cell and gene therapies and share commercial recommendations from our specialist...

Avalere Health

Biomarin

BioMarin’s application for haemophilia A gene therapy accepted by FDA

If approved, the treatment would be the first gene therapy in the US for this indication

- PMLiVE

The promised land of gene therapy: Commercialization of novel gene-editing technology in beta-thalassemia

With transformative treatments that leverage CRISPR technology, there are many challenges to overcome throughout the journey of development through to patient access. We interviewed a researcher at a top 20...

Avalere Health

- PMLiVE

Pfizer and Sangamo Therapeutics reopen recruitment for haemophilia A gene therapy trial

The trial was voluntarily paused by the two partners last year and later placed on hold by the FDA

- PMLiVE

bluebird bio’s Skysona approved by FDA to treat cerebral adrenoleukodystrophy

The gene therapy was shown to slow the progression of neurologic dysfunction

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