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gene therapy

Pfizer leans into gene therapy manufacturing with 'build-buy-partner' strategy

Pfizer leans into gene therapy manufacturing with 'build-buy-partner' strategy

Fierce Pharma

Vivet Therapeutics

WIlson's disease

build-buy-partner

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FDA sets back Novartis plans to expand use of SMA gene therapy

FDA sets back Novartis plans to expand use of SMA gene therapy

BioPharma Dive

clinical trials

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Epilepsy gene therapy candidate moves closer to clinical study phase

Epilepsy gene therapy candidate moves closer to clinical study phase

Biopharma Reporter

Cobra Biologics

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Sarepta faces another gene therapy hiccup as Regenxbio sues over Jim Wilson's patent

Sarepta faces another gene therapy hiccup as Regenxbio sues over Jim Wilson's patent

Endpoints

Sarepta Therapeutics

Duchenne Muscular Dystrophy

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Sarepta cites 'overburdened' FDA as factor in DMD gene therapy delay

Sarepta cites 'overburdened' FDA as factor in DMD gene therapy delay

Fierce Biotech

Sarepta Therapeutics

Duchenne Muscular Dystrophy

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Rocket Pharmaceuticals receives FDA fast track designation for RP-L401 gene therapy for infantile malignant osteopetrosis

Rocket Pharmaceuticals receives FDA fast track designation for RP-L401 gene therapy for infantile malignant osteopetrosis

Pharmaceutical Business Review

Rocket Pharmaceuticals

infantile malignant osteopetrosis[]

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Astellas' Audentes reports 3rd death in gene therapy trial

Astellas' Audentes reports 3rd death in gene therapy trial

Fierce Biotech

Audentes Therapeutics

clinical trials

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Is valrox dead?

Is valrox dead?

EP Vantage

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FDA puts up a red light for Passage Bio’s first gene therapy program

FDA puts up a red light for Passage Bio’s first gene therapy program

Endpoints

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Preparing for an influx of cell and gene therapy approvals

Preparing for an influx of cell and gene therapy approvals

Pharmaforum

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FDA keeps Solid Bio's troubled Duchenne MD gene therapy on clinical lockdown in latest setback

FDA keeps Solid Bio's troubled Duchenne MD gene therapy on clinical lockdown in latest setback

Endpoints

Solid Biosciences

Duchenne Muscular Dystrophy

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Locanabio’s Combo RNA Modification Platform Targets Multiple RNA Dysfunctions with One Injection

Locanabio’s Combo RNA Modification Platform Targets Multiple RNA Dysfunctions with One Injection

BioSpace

RNA modification

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Forge Bio’s Combo Model for Gene Therapies Attracts $40M in Funding

Forge Bio’s Combo Model for Gene Therapies Attracts $40M in Funding

Xconomy

Forge Biologics

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J&J-partnered MeiraGTx aims at Biogen with new eye gene therapy data

J&J-partnered MeiraGTx aims at Biogen with new eye gene therapy data

Endpoints

Retinitis pigmentosa

clinical trials

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Orchard licenses gene therapy tech from GSK

Orchard licenses gene therapy tech from GSK

Fierce Biotech

Orchard Therapeutics

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Biogen boosts gene therapy strategy with Harvard pact focused on inherited eye disease

Biogen boosts gene therapy strategy with Harvard pact focused on inherited eye disease

Fierce Biotech

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Sarepta bags drug to fully unlock DMD gene therapy opportunity

Sarepta bags drug to fully unlock DMD gene therapy opportunity

Fierce Biotech

Sarepta Therapeutics

Duchenne Muscular Dystrophy

Hansa Biopharma

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Takeda and Carmine Enter $900 Million Non-Viral Gene Therapy Collaboration

Takeda and Carmine Enter $900 Million Non-Viral Gene Therapy Collaboration

BioSpace

Carmine Therapeutics

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Two patients die in now-halted study of Audentes gene therapy

Two patients die in now-halted study of Audentes gene therapy

BioPharma Dive

Audentes Therapeutics

clinical trials

myotubular myopathy

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One-and-done gene therapy wipes out Parkinson's symptoms in mouse models

One-and-done gene therapy wipes out Parkinson's symptoms in mouse models

Fierce Biotech

Parkinson's Disease

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