gene therapy

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
gene therapy
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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
Takeda
gene therapy
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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
gene therapy
patient deaths
clinical trials
AT132
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
USCD
Parkinson's Disease
gene therapy
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With cell and gene therapy boom coming, experts at Novartis, Kite trumpet need for capacity: panel
With cell and gene therapy boom coming, experts at Novartis, Kite trumpet need for capacity: panel
Fierce Pharma
cell therapy
gene therapy
Novartis
Kite Pharma
Biomarin
Astellas
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Sarepta expands gene therapy toolkit, bets $72.5M to hitch a ride on Codiak's exosome tech
Sarepta expands gene therapy toolkit, bets $72.5M to hitch a ride on Codiak's exosome tech
Endpoints
Sarepta Therapeutics
Codiak Biosciences
gene therapy
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Pfizer, Sangamo flesh out hemophilia gene therapy data ahead of phase 3
Pfizer, Sangamo flesh out hemophilia gene therapy data ahead of phase 3
Fierce Biotech
Pfizer
Sangamo BioSciences
hemophilia A
clinical trials
gene therapy
giroctocogene fitelparvovec
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4DMT to Use $75 Million Series C to Advance Three Key Pipeline Products
4DMT to Use $75 Million Series C to Advance Three Key Pipeline Products
BioSpace
4D Molecular Therapeutics
IPOs
gene therapy
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Magenta, Beam partner to explore use of antibody-based conditioning drug for gene therapies
Magenta, Beam partner to explore use of antibody-based conditioning drug for gene therapies
MedCity News
Magenta Therapeutics
Beam Therapeutics
MGTA-117
gene therapy
sickle cell disease
beta thalassemia
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Bit Bio’s “enter button for the keyboard to the software of life” nabs the company $41.5 million
Bit Bio’s “enter button for the keyboard to the software of life” nabs the company $41.5 million
Tech Crunch
Bit Bio
startups
drug discovery
gene therapy
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New CRISPR, gene therapy results strengthen potential for treatment of blood diseases
New CRISPR, gene therapy results strengthen potential for treatment of blood diseases
BioPharma Dive
CRISPR
gene therapy
beta thalassemia
sickle cell disease
CRISPR Therapeutics
Vertex
Bluebird Bio
EHA
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Scopus Bio preps trials of gene-silencing immuno-oncology drug
Scopus Bio preps trials of gene-silencing immuno-oncology drug
Pharmaforum
Scopus BioPharma
lymphoma
immuno-oncology
CpG-STAT3siRNA
gene therapy
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Sarepta, with new data, plans final push for limb-girdle gene therapy
Sarepta, with new data, plans final push for limb-girdle gene therapy
BioPharma Dive
Sarepta Therapeutics
Limb Girdle Muscular Dystrophy
gene therapy
SRPT-9003
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Add ear gene therapy company Akouos to the ever-growing list of IPOs amid Covid-19
Add ear gene therapy company Akouos to the ever-growing list of IPOs amid Covid-19
Endpoints
IPOs
Akouos
NASDAQ
gene therapy
hearing loss
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BioMarin gene therapy shows sustained benefit in hemophilia A study
BioMarin gene therapy shows sustained benefit in hemophilia A study
Seeking Alpha
Biomarin
hemophilia A
clinical trials
gene therapy
valoctocogene roxaparvovec
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Big gene therapy names line up behind experimental Covid-19 vaccine
Big gene therapy names line up behind experimental Covid-19 vaccine
Stat
COVID-19
gene therapy
Novartis
AveXis
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What Sanofi Might Buy With Its Regeneron Windfall
What Sanofi Might Buy With Its Regeneron Windfall
Barron's
Sanofi
Regeneron
M&A
gene therapy
Ultragenyx
Bluebird Bio
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New Zolgensma 'inflection point' is here as Novartis snags EU nod for SMA gene therapy
New Zolgensma 'inflection point' is here as Novartis snags EU nod for SMA gene therapy
Fierce Pharma
Novartis
Zolgensma
SMA
spinal muscular atrophy
gene therapy
Europe
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Safety questions linger as Pfizer preps Duchenne gene therapy for final test
Safety questions linger as Pfizer preps Duchenne gene therapy for final test
BioPharma Dive
Pfizer
gene therapy
Duchenne Muscular Dystrophy
PF-06939926
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Thermo Fisher to invest $180M in new gene therapy plant
Thermo Fisher to invest $180M in new gene therapy plant
BioPharma Dive
Thermo Fisher
gene therapy
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