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gene therapy

FDA Awards Orphan Drug Designation to Prevail's Gene Therapy for Gaucher Disease

FDA Awards Orphan Drug Designation to Prevail's Gene Therapy for Gaucher Disease

BioSpace

Prevail Therapeutics

Gaucher disease

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Upcoming events – Avadel takes on Jazz and Axovant awaits early results

Upcoming events – Avadel takes on Jazz and Axovant awaits early results

EP Vantage

Jazz Pharmaceuticals

Parkinson's Disease

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Novartis faces criticism over Zolgensma ‘health lottery’

Novartis faces criticism over Zolgensma ‘health lottery’

PM Live

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Drugmaker BioMarin Seeks New CFO Amid Coming Release Of Potentially Costliest Drug In The World

Drugmaker BioMarin Seeks New CFO Amid Coming Release Of Potentially Costliest Drug In The World

Forbes

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Catalent laying out $315M to snatch another gene therapy CDMO

Catalent laying out $315M to snatch another gene therapy CDMO

Fierce Pharma

Paragon Services

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FDA Expects Explosion in Gene Therapy Treatments, Updates Review Policy

FDA Expects Explosion in Gene Therapy Treatments, Updates Review Policy

Motley Fool

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Deerfield vaults to the top of cell and gene therapy CDMO game with $1.1B facility at Philadelphia's newest biopharma hub

Deerfield vaults to the top of cell and gene therapy CDMO game with $1.1B facility at Philadelphia's newest biopharma hub

Endpoints

Deerfield Management

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BioMarin sets gene therapy sights beyond hemophilia

BioMarin sets gene therapy sights beyond hemophilia

BioPharma Dive

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Generation Bio Pockets $110M for Next Wave of Gene Therapy, IPO Plans

Generation Bio Pockets $110M for Next Wave of Gene Therapy, IPO Plans

Xconomy

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Ultragenyx shares jump on 'better than expected' gene therapy data

Ultragenyx shares jump on 'better than expected' gene therapy data

Fierce Biotech

clinical trials

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Solid Bio Restructures to Get Halted Gene Therapy Study Back on Track

Solid Bio Restructures to Get Halted Gene Therapy Study Back on Track

Xconomy

Solid Biosciences

Duchenne Muscular Dystrophy

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3 trends in biotech to watch in 2020

3 trends in biotech to watch in 2020

Stat

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Pharma's gene and cell therapy ambitions will kick into high gear in 2020—despite some major hurdles

Pharma's gene and cell therapy ambitions will kick into high gear in 2020—despite some major hurdles

Fierce Pharma

drug manufacturing

Bristol-Myers Squibb

Sarepta Therapeutics

Gilead Sciences

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Zolgensma for free? Yes, but advocates worry about Novartis' lottery-style giveaway

Zolgensma for free? Yes, but advocates worry about Novartis' lottery-style giveaway

Fierce Pharma

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Value-based pricing for gene therapy? Maybe not ready for hemophilia

Value-based pricing for gene therapy? Maybe not ready for hemophilia

Biopharma Dive

valoctocogene roxaparvovec

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Hopkins team invents non-viral system for getting gene therapy into cells

Hopkins team invents non-viral system for getting gene therapy into cells

Fierce Biotech

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Limelight Bio emerges with $75m to develop next generation gene therapies to overcome limitations of current treatments

Limelight Bio emerges with $75m to develop next generation gene therapies to overcome limitations of current treatments

Pharmaceutical Business Review

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FerGene’s Bladder Cancer Phase III Trial Hits Its Mark

FerGene’s Bladder Cancer Phase III Trial Hits Its Mark

BioSpace

Ferring Pharmaceuticals

Blackstone Life Sciences

clinical trials

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Here's Why BioMarin Pharmaceutical Leapt 10.2% in November

Here's Why BioMarin Pharmaceutical Leapt 10.2% in November

Motley Fool

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Gene Therapy M&A Appetite Provides Lift After Audentes Deal

Gene Therapy M&A Appetite Provides Lift After Audentes Deal

Yahoo/Bloomberg

Audentes Therapeutics

Medicines Company

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