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Sarepta

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FDA grants surprise OK to Sarepta’s DMD therapy after earlier rejection

Initially had concerns about infection risks and kidney toxicity

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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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Astellas makes gene therapy a core focus with $3bn Audentes buy

Deal includes a late-stage candidate for a rare disease

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Private equity firm backs Ferring gene therapy venture

Joint venture will focus on development of therapies for bladder cancer

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Solid Bio falls as FDA halts DMD trial over safety concerns

Shares in the biotech dropped dramatically following the announcement

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Personalised medicine can improve treatment, but access still an issue

Barriers to access could harm uptake of potentially transformative therapies

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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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Sarepta bounces back with new gene therapy data

Announces positive results in treatment of limb-girdle muscular dystrophy

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Sarepta rival NS Pharma files Duchenne drug with FDA

Another blow for Sarepta following FDA rejection earlier this year

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bluebird bio reveals further encouraging data for CALD gene therapy

Study shows continued benefit in treatment of rare disease

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Vertex eyes type 1 diabetes cure with biotech acquisition

Will acquire Semma Therapeutics for $950m

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NICE backs NHS use of Novartis’ gene therapy Luxturna

First and only therapy for rare, inherited form of blindness

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