Vertex, CRISPR prep filing for gene-editing blood disorder therapy
beta thalassaemia, CRISPR Therapeutics, CRISPR/Cas9, gene editing, sickle cell disease, Vertex
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beta thalassaemia
bluebird unveils $2.8m price for gene therapy Zynteglo on FDA approval
beta thalassaemia, bluebird bio, fda, gene therapy, regulatory approval, Zynteglo
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Precision Bio climbs on near-$1.5bn sickle cell pact with Novartis
beta thalassaemia, gene editing, Haematology, Novartis, partnering, Precision BioSciences, sickle cell disease
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EHA: Vertex, CRISPR build case for gene-editing drug
beta thalassaemia, CRISPR Therapeutics, CRISPR/Cas9, EHA 2022, gene editing, sickle cell disease, Vertex Pharma
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Second unanimous FDA adcomm vote boosts bluebird bio
beta thalassaemia, bluebird bio, fda, gene therapy, rare disease, regulatory
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ICER says bluebird bio’s $2.1m gene therapy is cost-effective
beta thalassaemia, bluebird bio, gene therapy, HEOR, ICER
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Vertex builds in gene editing yet again, with $1.2bn Arbor deal
Arbor Biotechnologies, beta thalassaemia, CRISPR/Cas9, gene editing, sickle cell disease, type 1 diabetes, Vertex Pharma
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EMA finds possible link between mRNA COVID jabs and myocarditis
beta thalassaemia, bluebird bio, coronavirus, COVID-19, COVID-19 vaccine, ema, mRNA, PRAC, Rare diseases, Zynteglo
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