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gene therapy

Sen. Bill Cassidy Eyes Legislation to Improve Access to Gene Therapies

Sen. Bill Cassidy Eyes Legislation to Improve Access to Gene Therapies

BioSpace

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BioMarin secures hemophilia gene therapy coverage in Germany

BioMarin secures hemophilia gene therapy coverage in Germany

BioPharma Dive

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Cell & Gene Therapy Space Gears Up for First CRISPR Approval

Cell & Gene Therapy Space Gears Up for First CRISPR Approval

BioSpace

sickle cell disease

Vertex Pharmaceuticals

CRISPR Therapeutics

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Bayer adds a new life science incubator in China

Bayer adds a new life science incubator in China

Pharmaphorum

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Krystal's topical gene therapy gains steam, prompting analyst group to boost sales projection

Krystal's topical gene therapy gains steam, prompting analyst group to boost sales projection

Fierce Biotech

Krystal Biotech

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Lexeo drops share price at last minute in attempt to raise $100M IPO

Lexeo drops share price at last minute in attempt to raise $100M IPO

Fierce Biotech

Lexeo Therapeutics

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Sarepta enjoys early sales ramp for DMD gene therapy, maintains confidence in label expansion

Sarepta enjoys early sales ramp for DMD gene therapy, maintains confidence in label expansion

Fierce Pharma

Sarepta Therapeutics

Duchenne Muscular Dystrophy

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Two Sickle Cell Disease Gene Therapies May Soon Be Approved By FDA

Two Sickle Cell Disease Gene Therapies May Soon Be Approved By FDA

Forbes

sickle cell disease

Vertex Pharmaceutical

CRISPR Therapeutics

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AstraZeneca Drops $245M on Cell and Gene Therapies in Deal with Cellectis

AstraZeneca Drops $245M on Cell and Gene Therapies in Deal with Cellectis

BioSpace

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FDA panel says Vertex/CRISPR to assess safety risks of gene therapy in follow-up study

FDA panel says Vertex/CRISPR to assess safety risks of gene therapy in follow-up study

Yahoo/Reuters

Vertex Pharmaceuticals

CRISPR Therapeutics

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Sarepta’s disappointing data put the FDA in a difficult spot

Sarepta’s disappointing data put the FDA in a difficult spot

Stat

Sarepta Therapeutics

Duchenne Muscular Dystrophy

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Sarepta gene therapy for Duchenne misses main goal of key study

Sarepta gene therapy for Duchenne misses main goal of key study

BioPharma Dive

Sarepta Therapeutics

Duchenne Muscular Dystrophy

clinical trials

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Bluebird bio to sell sickle cell FDA voucher for $103 mln if therapy approved

Bluebird bio to sell sickle cell FDA voucher for $103 mln if therapy approved

Reuters

priority review vouchers

sickle cell disease

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ThermoGenesis opens cell and gene therapy facility in Sacramento, US

ThermoGenesis opens cell and gene therapy facility in Sacramento, US

Pharmaceutical Business Review

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Roche builds gene therapy portfolio with SpliceBio deal

Roche builds gene therapy portfolio with SpliceBio deal

Pharmaphorum

Spark Therapeutics

inherited retinal disease

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Gene therapy maker UniQure to cut about 20% of workforce

Gene therapy maker UniQure to cut about 20% of workforce

Reuters

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Bluebird taps Lonza to boost manufacturing capacity for its gene therapies Zynteglo, Skysona

Bluebird taps Lonza to boost manufacturing capacity for its gene therapies Zynteglo, Skysona

Fierce Pharma

drug manufacturing

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Taysha ends work on gene therapy after Astellas walks away, FDA requests randomized trial

Taysha ends work on gene therapy after Astellas walks away, FDA requests randomized trial

Fierce Biotech

Taysha Gene Therapies

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Orchard nears FDA decision on rare disease gene therapy

Orchard nears FDA decision on rare disease gene therapy

BioPharma Dive

Orchard Therapeutics

metachromatic leukodystrophy

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Have Million-Dollar Gene Therapies Finally Reached An Inflection Point?

Have Million-Dollar Gene Therapies Finally Reached An Inflection Point?

Investors Business Daily

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