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cell and genetic therapies

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Bluebird bio’s sickle cell gene therapy shows early-study benefit

Closely follows approval of beta thalassaemia therapy Zynteglo

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FDA backs GBT’s sickle cell drug Oxbryta

Second new treatment for blood disorder to be approved this year

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Vertex continues upward trajectory with French Orkambi deal

Comes on the heels of similar reimbursement deal in England

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Vertex reveals promising data from first CRISPR-treated patients

Investigational gene therapy has potentially curative effect

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Provention Bio wins PRIME designation for type 1 diabetes prevention drug

Meanwhile J&J partnered Crohn's drug flops in phase 2

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NIH, Gates pledge $200m for HIV and sickle cell gene therapies

Aim to bring therapies to patients in lower-income countries

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Vertex, NHS England and NICE finally reach agreement for Orkambi

Deal also includes Vertex’s other licensed cystic fibrosis medicines

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Vertex scores FDA approval for CF triple therapy Trikafta

Decision comes only a few months after therapy was filed

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Labour Party takes aim at pharma, calls attention to Orkambi row

Proposal to introduce compulsory licensing faces industry criticism

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Vertex eyes type 1 diabetes cure with biotech acquisition

Will acquire Semma Therapeutics for $950m

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Vertex faces rejection of its CF medicines in Scotland

Another rejection over cost-effectiveness

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Leiden reassures on key role at Vertex

Reshma Kewalramani to be new CEO, but Leiden will retain portfolio

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