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gene therapy

Bluebird Bio Announces More Good News for LentiGlobin Gene Therapy – Exciting Results in Sickle Cell Disease

Bluebird Bio Announces More Good News for LentiGlobin Gene Therapy – Exciting Results in Sickle Cell Disease

CP Wire

sickle cell disease

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Bluebird Bio Presents Promising Results for Gene Therapy in Rare Blood Disorder

Bluebird Bio Presents Promising Results for Gene Therapy in Rare Blood Disorder

CP Wire

beta thalassemia

sickle cell disease

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Novartis' brand-new gene therapy player AveXis will build $55M plant, hire 200

Novartis' brand-new gene therapy player AveXis will build $55M plant, hire 200

Fierce Pharma

drug manufacturing

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Is biopharma’s first $1.5M therapy now in Phase III?

Is biopharma’s first $1.5M therapy now in Phase III?

Endpoints

Spark Therapeutics

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First Patient Dosed in Avexis' Phase 3 Trial of AVXS-101 in Pre-Symptomatic Spinal Muscular Atrophy

First Patient Dosed in Avexis' Phase 3 Trial of AVXS-101 in Pre-Symptomatic Spinal Muscular Atrophy

CP Wire

clinical trials

spinal muscular atrophy

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Bluebird’s gene therapy for beta-thalassemia falls short of a cure, but still wows in 22 patients

Bluebird’s gene therapy for beta-thalassemia falls short of a cure, but still wows in 22 patients

Endpoints

beta-thalassemia

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[video]Did Novartis Overpay for AveXis?

[video]Did Novartis Overpay for AveXis?

Motley Fool

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GSK slims portfolio with sale of rare disease gene therapy drugs

GSK slims portfolio with sale of rare disease gene therapy drugs

Yahoo/Reuters

Orchard Therapeutics

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Pfizer Commences Ph Ib Trial With Mini-Dystrophin Gene Therapy for the Treatment of Duchenne Muscular Dystrophy

Pfizer Commences Ph Ib Trial With Mini-Dystrophin Gene Therapy for the Treatment of Duchenne Muscular Dystrophy

CP Wire

Duchenne Muscular Dystrophy

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Lonza opens largest gene therapy plant in the world and hires 200 scientists

Lonza opens largest gene therapy plant in the world and hires 200 scientists

GoinPharma

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Why Did Novartis Spend $9 Billion On Gene Therapy? CEO Says: 'A Journey To Focus On Data And Digital'

Why Did Novartis Spend $9 Billion On Gene Therapy? CEO Says: 'A Journey To Focus On Data And Digital'

Forbes

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Novartis buying AveXis for $8.7 billion to strengthen gene therapy capabilities

Novartis buying AveXis for $8.7 billion to strengthen gene therapy capabilities

Stat

spinal muscular atrophy

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‘That’s $425,000 right there’ — the anxious launch of a gene therapy with a record sticker price

‘That’s $425,000 right there’ — the anxious launch of a gene therapy with a record sticker price

Stat

Spark Therapeutics

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Solid Bio's DMD gene therapy graduates from partial FDA hold to full-on clinical hold

Solid Bio's DMD gene therapy graduates from partial FDA hold to full-on clinical hold

Fierce Biotech

Solid Biosciences

Duchenne Muscular Dystrophy

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Prevail grabs $75M A round in the hunt for new gene therapies to stop the destruction caused by Parkinson’s

Prevail grabs $75M A round in the hunt for new gene therapies to stop the destruction caused by Parkinson’s

Endpoints

Prevail Therapeutics

Parkinson's Disease

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Tiny U.K. Biotech Takes On Glaxo’s $730,000 Gene Therapy

Tiny U.K. Biotech Takes On Glaxo’s $730,000 Gene Therapy

Bloomberg

bubble boy syndrome

Orchard Therapeutics

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Spark lays groundwork for US Luxturna launch

Spark lays groundwork for US Luxturna launch

Biopharma Dive

Spark Therapeutics

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With $69M Deal, AbbVie Turns to Voyager for an Alzheimer’s Gene Therapy

With $69M Deal, AbbVie Turns to Voyager for an Alzheimer’s Gene Therapy

Xconomy

Voyager Therapeutics

Alzheimer's disease

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Novartis’ Kymriah: The First Gene Therapy to Be Approved in the US

Novartis’ Kymriah: The First Gene Therapy to Be Approved in the US

Market Realist

acute lymphoblastic leukemia

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Bioverativ inks $100M gene therapy deal with Oxford BioMedica

Bioverativ inks $100M gene therapy deal with Oxford BioMedica

Endpoints

Oxford BioMedica

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