You are here

gene therapy

Sarepta now needs flawless progress for gene therapy

Sarepta now needs flawless progress for gene therapy

Biopharma Dive

Sarepta Therapeutics

Duchenne Muscular Dystrophy

Flag link:

Gene therapies to test FDA's approach to regulating drugs after launch, experts say

Gene therapies to test FDA's approach to regulating drugs after launch, experts say

Bizjournals

Flag link:

Treating glioblastoma with a Ziopharm drug and IL-12 gene therapy

Treating glioblastoma with a Ziopharm drug and IL-12 gene therapy

Fierce Biotech

Ziopharm Oncology

clinical trials

Flag link:

Patient in Sarepta gene therapy trial develops serious illness; shares plunge

Patient in Sarepta gene therapy trial develops serious illness; shares plunge

Yahoo/Reuters

Sarepta Therapeutics

Duchenne Muscular Dystrophy

Flag link:

More M&A Activity Expected Around AAV-Based Gene Therapies, Analyst Says

More M&A Activity Expected Around AAV-Based Gene Therapies, Analyst Says

BioSpace

Audentes Therapeutics

Flag link:

Novartis used manipulated data in winning Zolgensma approval, FDA says

Novartis used manipulated data in winning Zolgensma approval, FDA says

Biopharma Dive

spinal muscular atrophy

Flag link:

BioMarin drops lower dose of its hemophilia gene therapy as it eyes submissions by year-end

BioMarin drops lower dose of its hemophilia gene therapy as it eyes submissions by year-end

Fierce Biotech

valoctocogene roxaparvovec

Flag link:

Amicus shares early look at Batten disease gene therapy

Amicus shares early look at Batten disease gene therapy

Fierce Biotech

Amicus Therapeutics

Flag link:

RegenxBio and Pfizer Partner on Gene Therapy for Friedreich's Ataxia

RegenxBio and Pfizer Partner on Gene Therapy for Friedreich's Ataxia

BioSpace

Friedreich's Ataxia

Flag link:

Massachusetts General and ElevateBio Ink 10-Year Cell and Gene Therapy Deal

Massachusetts General and ElevateBio Ink 10-Year Cell and Gene Therapy Deal

BioSpace

Massachusetts General Hospital

Flag link:

Ultragenyx close to learning whether gene therapy gamble will pay off

Ultragenyx close to learning whether gene therapy gamble will pay off

Biopharma Dive

Dimension Therapeutics

Flag link:

Payers, patients coming on board for Novartis’ Zolgensma

Payers, patients coming on board for Novartis’ Zolgensma

BioCentury

Flag link:

Billions on the line: BioMarin bustles past enthused rival, planning to launch world’s first hemophilia A gene therapy

Billions on the line: BioMarin bustles past enthused rival, planning to launch world’s first hemophilia A gene therapy

Endpoints

Flag link:

Sangamo backs up early success for hemophilia gene therapy

Sangamo backs up early success for hemophilia gene therapy

Biopharma Dive

Spark Therapeutics

Flag link:

Novartis struggling to win payer coverage for $2.1M gene therapy Zolgensma: analysts

Novartis struggling to win payer coverage for $2.1M gene therapy Zolgensma: analysts

Fierce Pharma

Flag link:

Thermo Fisher to manufacture gene therapies being developed by Amicus

Thermo Fisher to manufacture gene therapies being developed by Amicus

Fierce Pharma

Amicus Therapeutics

Flag link:

3 Small-Cap Biotech Stocks That Soared Last Week

3 Small-Cap Biotech Stocks That Soared Last Week

Motley Fool

Dova Pharmaceuticals

Krystal Biotech

Flag link:

Pfizer's DMD Gene Therapy Shows Severe Side Effects in Study

Pfizer's DMD Gene Therapy Shows Severe Side Effects in Study

Yahoo/Zacks.com

Duchenne Muscular Dystrophy

clinical trials

Flag link:

Krystal looks to Phase III as stock soars on data for gene therapy

Krystal looks to Phase III as stock soars on data for gene therapy

BioCentury

Krystal Biotech

Flag link:

Pharmacquired: Is $11B enough to secure Sanofi's spot in hemophilia?

Pharmacquired: Is $11B enough to secure Sanofi's spot in hemophilia?

Biopharma Dive

Flag link:

Pages