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sickle cell disease

Landmark approval for Vertex's CRISPR drug 2 steps closer after trial wins, PDUFA date granted

Landmark approval for Vertex's CRISPR drug 2 steps closer after trial wins, PDUFA date granted

Fierce Biotech

Vertex Pharmaceuticals

CRISPR Therapeutics

sickle cell disease

beta thalassemia

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Bluebird Bio submits BLA for sickle cell disease gene therapy

Bluebird Bio submits BLA for sickle cell disease gene therapy

Biopharma Reporter

sickle cell disease

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Vertex/CRISPR's gene editing therapy cost effective at $1.9 million - pricing review group

Vertex/CRISPR's gene editing therapy cost effective at $1.9 million - pricing review group

Yahoo/Reuters

Vertex Pharmaceutials

CRISPR Therapeutics

sickle cell disease

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Could This Move Make Vertex's Next Blockbuster Even Bigger?

Could This Move Make Vertex's Next Blockbuster Even Bigger?

Motley Fool

Vertex Pharmaceuticals

beta thalassemia

sickle cell disease

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Vertex and CRISPR Therapeutics Complete Submission of Rolling Biologics License Applications (BLAs) to the US FDA for exa-cel for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

Vertex and CRISPR Therapeutics Complete Submission of Rolling Biologics License Applications (BLAs) to the US FDA for exa-cel for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

Yahoo/Businesswire

Vertex Pharmaceuticals

CRISPR Therapeutics

sickle cell disease

beta thalassemia

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Bluebird to miss Q1 deadline for lovo-cel filing, shares sliced

Bluebird to miss Q1 deadline for lovo-cel filing, shares sliced

Endpoints

sickle cell disease

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Fulcrum's clinical hold caps off tough week for sickle cell drug development

Fulcrum's clinical hold caps off tough week for sickle cell drug development

Fierce Biotech

Fulcrum Therapeuticsl

sickle cell disease

clinical trials

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Novartis Drops SCD Gene Therapy Program with Intellia

Novartis Drops SCD Gene Therapy Program with Intellia

BioSpace

sickle cell disease

Intellia Therapeutics

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Graphite Bio Cuts Sickle Cell Therapy, Half of Staff

Graphite Bio Cuts Sickle Cell Therapy, Half of Staff

BioSpace

sickle cell disease

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Medicaid, with planned payment pilot, girds for influx of pricey gene therapies

Medicaid, with planned payment pilot, girds for influx of pricey gene therapies

BioPharma Dive

sickle cell disease

Vertex Pharmaceuticals

CRISPR Therapeutics

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Vertex Teases Launch of Landmark CRISPR Therapy

Vertex Teases Launch of Landmark CRISPR Therapy

BioSpace

Vertex Pharmaceuticals

sickle cell disease

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Novartis' approved sickle cell disease drug fails to beat placebo in PhIII

Novartis' approved sickle cell disease drug fails to beat placebo in PhIII

Endpoints

sickle cell disease

clinical trials

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EMA validates Vertex's CRISPR application, clearing barrier on road to landmark approval

EMA validates Vertex's CRISPR application, clearing barrier on road to landmark approval

Fierce Biotech

Vertex Pharmaceuticals

CRISPR Therapeutics

sickle cell disease

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Graphite's hopes for sickle cell 'cure' blunted after first patient dosed experiences serious event

Graphite's hopes for sickle cell 'cure' blunted after first patient dosed experiences serious event

Fierce Biotech

sickle cell disease

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We finally have new drugs for sickle cell disease. Why are so few patients taking them?

We finally have new drugs for sickle cell disease. Why are so few patients taking them?

Stat

sickle cell disease

Global Blood Therapeutics

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FDA Lifts Partial Clinical Hold On Bluebird Bio's Sickle Cell Disease Studies

FDA Lifts Partial Clinical Hold On Bluebird Bio's Sickle Cell Disease Studies

NASDAQ

clinical trials

sickle cell disease

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ASH: Despite partial clinical hold, bluebird's sickle cell gene therapy remains on track

ASH: Despite partial clinical hold, bluebird's sickle cell gene therapy remains on track

Fierce Pharma

sickle cell disease

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Editas teases tiny sickle cell sample in effort to climb back into gene editing's upper echelon

Editas teases tiny sickle cell sample in effort to climb back into gene editing's upper echelon

Fierce Biotech

Editas Medicine

sickle cell disease

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ASH preview – waiting for Editas

ASH preview – waiting for Editas

EP Vantage

sickle cell disease

Sangamo BioSciences

Editas Medicine

Vertex Pharmaceuticals

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Vertex given green light to seek US approval of CRISPR-based therapy

Vertex given green light to seek US approval of CRISPR-based therapy

BioPharma Dive

Vertex Pharmaceuticals

CRISPR Therapeutics

beta thalassemia

sickle cell disease

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