gene therapy

FDA panel says Vertex/CRISPR to assess safety risks of gene therapy in follow-up study
Yahoo/Reuters
Tue, 10/31/23 - 09:23 pm
Vertex Pharmaceuticals
CRISPR Therapeutics
FDA
gene therapy
exa-cel
Sarepta’s disappointing data put the FDA in a difficult spot
Stat
Tue, 10/31/23 - 10:16 am
Sarepta Therapeutics
Duchenne Muscular Dystrophy
DMD
FDA
gene therapy
Sarepta gene therapy for Duchenne misses main goal of key study
BioPharma Dive
Mon, 10/30/23 - 08:17 pm
Sarepta Therapeutics
gene therapy
Duchenne Muscular Dystrophy
clinical trials
FDA
Elevidys
Bluebird bio to sell sickle cell FDA voucher for $103 mln if therapy approved
Reuters
Mon, 10/30/23 - 10:23 am
Bluebird Bio
priority review vouchers
sickle cell disease
gene therapy
ThermoGenesis opens cell and gene therapy facility in Sacramento, US
Pharmaceutical Business Review
Fri, 10/27/23 - 11:33 am
Thermogenesis
cell therapy
gene therapy
California
Roche builds gene therapy portfolio with SpliceBio deal
Pharmaphorum
Tue, 10/17/23 - 11:00 am
Roche
Spark Therapeutics
SpliceBio
gene therapy
inherited retinal disease
Gene therapy maker UniQure to cut about 20% of workforce
Reuters
Thu, 10/5/23 - 10:10 am
uniQure
gene therapy
layoffs
Bluebird taps Lonza to boost manufacturing capacity for its gene therapies Zynteglo, Skysona
Fierce Pharma
Thu, 09/28/23 - 10:27 am
Bluebird Bio
gene therapy
Lonza
drug manufacturing
CDMOs
Taysha ends work on gene therapy after Astellas walks away, FDA requests randomized trial
Fierce Biotech
Wed, 09/20/23 - 08:47 am
Astellas
Taysha Gene Therapies
gene therapy
FDA
Orchard nears FDA decision on rare disease gene therapy
BioPharma Dive
Mon, 09/18/23 - 12:25 pm
Orchard Therapeutics
gene therapy
metachromatic leukodystrophy
FDA
Have Million-Dollar Gene Therapies Finally Reached An Inflection Point?
Investors Business Daily
Sun, 09/17/23 - 09:42 pm
gene therapy
AlveoGene debuts to develop inhaled gene therapy technology
Pharmaphorum
Thu, 09/14/23 - 10:01 am
Alvogene
UK
startups
lentiviral vectors
gene therapy
drug delivery
Rocket boosted as FDA alignment on pivotal gene therapy trial design sends stock skyward
Fierce Biotech
Wed, 09/13/23 - 11:30 am
Rocket Pharmaceuticals
gene therapy
FDA
clinical trials
RP-A501
Danon disease
With Roctavian as a launching pad, BioMarin looks to up pace of new drug filings
Fierce Biotech
Tue, 09/12/23 - 07:17 pm
Biomarin
Roctavian
hemophilia A
Voxzogo
achrondoplasia
drug development
gene therapy
Novartis Drops Eye Disease Gene Therapy from $800M Gyroscope Acquisition
BioSpace
Mon, 09/11/23 - 12:28 pm
Novartis
GT005
gene therapy
geographic atrophy
Gyroscope Therapeutics
Otsuka, Shape Ink Potential $1.5B Deal to Develop Eye AAV Gene Therapies
BioSpace
Fri, 09/8/23 - 10:11 am
Otsuka
Shape Therapeutics
gene therapy
AAV gene therapy
RNA editing
ocular diseases
Kriya, flush with funding, buys a gene therapy for NASH
BioPharma Dive
Thu, 09/7/23 - 10:51 pm
Kriya Therapeutics
gene therapy
NASH
M&A
Tramontane Therapeutics
Royalty Pharma to Pay Potential $500M for Royalties on Ferring’s Gene Therapy
BioSpace
Thu, 08/24/23 - 08:34 pm
Ferring Pharmaceuticals
Royalty Pharma
gene therapy
bladder cancer
Genethon’s Gene Therapy for Rare Liver Disease Shows Promising Early-Stage Results
BioSpace
Fri, 08/18/23 - 11:37 am
Genethon
gene therapy
Crigler-Najjar Syndrome
liver disease
clinical trials
Thermo Fisher lays off 200-plus at clinical-stage cell and gene therapy factory in Florida
Fierce Pharma
Mon, 08/14/23 - 06:51 pm
ThermoFisher Scientific
layoffs
gene therapy
cell therapy

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