gene therapy

Novartis, Voyager launch another gene therapy mission with $100M upfront deal
Fierce Biotech
Tue, 01/2/24 - 10:05 am
Novartis
gene therapy
Voyager Therapeutics
Huntington's disease
SMA
Takeda’s gene therapy pivot leaves JCR behind after proof-of-concept preclinical test
Fierce Biotech
Thu, 12/21/23 - 09:57 am
Takeda
gene therapy
JCR Pharma
Benefit of uniQure's Huntington's gene therapy will be revealed 'in the fullness of time'
Fierce Biotech
Tue, 12/19/23 - 11:14 pm
uniQure
Huntington's disease
gene therapy
Bluebird’s Newly Approved SCD Gene Therapy Faces Rough Road with Hefty Price Tag, Boxed Warning
BioSpace
Mon, 12/11/23 - 11:20 am
Bluebird Bio
sickle cell disease
Lyfgenia
gene therapy
CRISPR
Sen. Bill Cassidy Eyes Legislation to Improve Access to Gene Therapies
BioSpace
Wed, 12/6/23 - 10:26 am
Senate
legislation
gene therapy
patient access
BioMarin secures hemophilia gene therapy coverage in Germany
BioPharma Dive
Wed, 11/29/23 - 11:35 pm
Biomarin
Roctavian
gene therapy
Germany
hemophilia
Cell & Gene Therapy Space Gears Up for First CRISPR Approval
BioSpace
Mon, 11/13/23 - 11:48 am
CRISPR
gene therapy
cell therapy
sickle cell disease
exa-cel
Vertex Pharmaceuticals
CRISPR Therapeutics
Bayer adds a new life science incubator in China
Pharmaphorum
Tue, 11/7/23 - 10:04 am
Bayer
Co.Lab
incubators
China
oncology
cell therapy
gene therapy
Krystal's topical gene therapy gains steam, prompting analyst group to boost sales projection
Fierce Biotech
Mon, 11/6/23 - 07:27 pm
Krystal Biotech
gene therapy
Lexeo drops share price at last minute in attempt to raise $100M IPO
Fierce Biotech
Fri, 11/3/23 - 11:53 am
Lexeo Therapeutics
IPOs
gene therapy
Sarepta enjoys early sales ramp for DMD gene therapy, maintains confidence in label expansion
Fierce Pharma
Thu, 11/2/23 - 05:15 pm
Sarepta Therapeutics
DMD
Duchenne Muscular Dystrophy
Elevidys
gene therapy
Two Sickle Cell Disease Gene Therapies May Soon Be Approved By FDA
Forbes
Thu, 11/2/23 - 05:07 pm
sickle cell disease
gene therapy
lovo-cel
exa-cel
Vertex Pharmaceutical
CRISPR Therapeutics
Bluebird Bio
AstraZeneca Drops $245M on Cell and Gene Therapies in Deal with Cellectis
BioSpace
Wed, 11/1/23 - 11:07 am
AstraZeneca
Cellectis
cell therapy
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

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