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chronic lung disease

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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

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AstraZeneca and Medera’s Novoheart develop first human models of heart failure

The ‘heart-in-a-jar’ model will be used to effectively test drug candidates for HFpEF patient

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EMA grants AZ’s Vaxzevria COVID-19 vaccine full marketing authorisation

Vaxzevria has been shown to be effective against all forms of COVID-19

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Alexion’s Ultomiris shows promise in neuromyelitis optica spectrum disorder phase 3 trial

Ultomiris-treated patients showed zero relapses with a median treatment duration of 73 weeks

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AstraZeneca’s two advanced breast cancer trials meet primary endpoints

Both trials showed significant improvement in progression-free survival

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AstraZeneca’s Imjudo/Imfinzi combination approved by FDA for unresectable liver cancer

Phase 3 trial results demonstrated a 22% reduction in risk of death versus sorafenib

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

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AstraZeneca’s Tezspire receives MHRA approval as add-on treatment for severe asthma

Tezspire demonstrated reduced annualised rate of asthma exacerbations

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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

AstraZeneca’s Tezspire receives EC approval as severe asthma add-on treatment

Tezspire demonstrated superiority across every primary and key secondary endpoint compared to placebo

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