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gene therapy

Moderna's gene therapy expansion picks up pace with $76M upfront Generation Bio collab

Moderna's gene therapy expansion picks up pace with $76M upfront Generation Bio collab

Fierce Biotech

Moderna Therapeutics

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FDA's Peter Marks backs accelerated approval for gene therapies

FDA's Peter Marks backs accelerated approval for gene therapies

BioSpace

accelerated approval

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Novartis: SMA Drug Zolgensma Long-term Data Shows Sustained Durability Up To 7.5 Years Post-dosing

Novartis: SMA Drug Zolgensma Long-term Data Shows Sustained Durability Up To 7.5 Years Post-dosing

NASDAQ

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FDA Reverses Course; Will Hold Adcomm for Sarepta’s DMD Gene Therapy

FDA Reverses Course; Will Hold Adcomm for Sarepta’s DMD Gene Therapy

BioSpace

Sarepta Therapeutics

Duchenne Muscular Dystrophy

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Duchenne muscular dystrophy: major trials and events to watch in 2023

Duchenne muscular dystrophy: major trials and events to watch in 2023

Clinical Trials Arena

Duchenne Muscular Dystrophy

clinical trials

Sarepta Therapeutics

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CRISPR/ViaCyte and Genprex Vie for First Diabetes Gene Therapy

CRISPR/ViaCyte and Genprex Vie for First Diabetes Gene Therapy

BioSpace

CRISPR Therapeutics

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FDA delays review of BioMarin's hemophilia gene therapy after data update

FDA delays review of BioMarin's hemophilia gene therapy after data update

Endpoints

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Sarepta: FDA finds ‘no significant safety issues of concern’ with DMD gene therapy

Sarepta: FDA finds ‘no significant safety issues of concern’ with DMD gene therapy

Biopharma Reporter

Sarepta Therapeutics

Duchenne Muscular Dystrophy

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Sangamo scraps public offering ‘after evaluating market conditions’

Sangamo scraps public offering ‘after evaluating market conditions’

Endpoints

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Spark Breaks Ground on Gene Therapy Innovation Center on Drexel Campus in Philadelphia

Spark Breaks Ground on Gene Therapy Innovation Center on Drexel Campus in Philadelphia

BioBuzz

Spark Therapeutics

Drexel University

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CBER director Peter Marks says FDA is growing to keep up with cell and gene therapies

CBER director Peter Marks says FDA is growing to keep up with cell and gene therapies

Endpoints

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Sarepta's DMD gene therapy inches closer to market as FDA declines to hold advisory meeting

Sarepta's DMD gene therapy inches closer to market as FDA declines to hold advisory meeting

Fierce Biotech

Sarepta Therapeutics

Duchenne Muscular Dystrophy

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FDA plans to launch communications pilot for promising rare disease gene therapies

FDA plans to launch communications pilot for promising rare disease gene therapies

RAPS.org

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AbbVie, Capsida Set Sights on Gene Therapies for Eye Diseases

AbbVie, Capsida Set Sights on Gene Therapies for Eye Diseases

BioSpace

Capsida Biotherapeutics

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First Gene Therapy for Hemophilia B, CSL's HEMGENIX®, Approved by the European Commission

First Gene Therapy for Hemophilia B, CSL's HEMGENIX®, Approved by the European Commission

BioSpace

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'It's the right call': GSK expresses no regrets on abandoning cell therapy as oligo strategy heats up

'It's the right call': GSK expresses no regrets on abandoning cell therapy as oligo strategy heats up

Fierce Biotech

oligonucleotides

Wave Life Sciences

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A renewed push into early bladder cancer

A renewed push into early bladder cancer

EP Vantage

Ferring Pharmaceuticals

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Taysha’s GAN Gene Therapy Hits FDA Speedbump

Taysha’s GAN Gene Therapy Hits FDA Speedbump

BioSpace

Taysha Gene Therapies

axonal neuropathy

clinical trials

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Amid application tsunami, FDA's top cell and gene therapy leader to retire in March

Amid application tsunami, FDA's top cell and gene therapy leader to retire in March

Endpoints

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UniQure nabs another gene therapy for ALS

UniQure nabs another gene therapy for ALS

BioPharma Dive

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