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gene therapy
More trouble for Astellas as AT132 gene therapy trial is halted again
Pharmaforum
Wed, 09/1/21 - 11:02 am
Astellas
gene therapy
AT132
clinical trials
X-linked myotubular myopathy
Vertex boosts cell and genetic therapies toolkit in deal worth up to $1.2bn with Arbor
Biopharma Reporter
Mon, 08/30/21 - 10:57 am
Vertex
cell therapy
gene therapy
CRISPR
gene editing
Arbor Biotechnologies
Cell, gene therapy company funding reaches new heights, despite setbacks
BioPharma Dive
Thu, 08/19/21 - 12:08 pm
cell therapy
gene therapy
funding
FDA lifts clinical hold on Rocket gene therapy trial, creating launchpad for start of pediatric dosing
Fierce Biotech
Mon, 08/16/21 - 11:08 am
Rocket Pharmaceuticals
gene therapy
FDA
Danon disease
Bluebird bio Takes Hit with FDA Clinical Hold on Gene Therapy
BioSpace
Mon, 08/9/21 - 11:48 pm
Bluebird Bio
FDA
clinical hold
gene therapy
eli-cel
cerebral adrenoleukodystrophy
Ring Hopes to Bring First Re-dosable Gene Therapy to the Clinic with $117M Series B
BioSpace
Wed, 07/28/21 - 11:26 pm
Ring Therapeutics
gene therapy
funding
Half of Adverum pipeline 'snaps out of existence' as adverse events force pivot to more common eye disease
Fierce Biotech
Fri, 07/23/21 - 11:07 am
Adverum Biotechnologies
ADVM-022
diabetic macular edema
gene therapy
Gene therapy for Fabry: early stages, promising results
BioPharma Dive
Thu, 07/22/21 - 10:02 pm
gene therapy
Fabry disease
uniQure
Avrbio
Freeline Therapeutics
Sangamo Therapeutics
Bluebird, with little fanfare, is first to bring a second gene therapy to market
BioPharma Dive
Wed, 07/21/21 - 08:43 pm
Europe
Bluebird Bio
Skysona
cerebral adrenoleukodystrophy
gene therapy
Coave raises $25M ahead of pivotal retinal gene therapy trial
Fierce Biotech
Wed, 07/21/21 - 11:02 am
Coave Therapeutics
retinal dystrophies
gene therapy
The 'dovetail' partnership meant to turn Boston Children's into a hub for gene therapy startups
BioPharma Dive
Thu, 07/1/21 - 10:35 pm
Elevate Bio
Boston Children's Hospital
gene therapy
cell therapy
Novartis, GBT sickle cell drugs face coverage hurdles as gene therapy threats loom: survey
Fierce Pharma
Wed, 06/30/21 - 12:09 am
Novartis
Adakveo
Global Blood Therapeutics
Oxbryta
sickle cell disease
gene therapy
AskBio, Syndax and Catalyst All on the Fast Track
BioSpace
Mon, 06/28/21 - 06:56 pm
FDA
fast track status
AskBio
Bayer
gene therapy
LGMD
Syndax Pharma
leukemia
Catalyst Biosciences
episodic bleeding
Biopharma M&A set for a strong H2 despite political noise—and Biogen and Gilead look like buyers: analysts
Fierce Pharma
Mon, 06/28/21 - 11:54 am
M&A
Joe Biden
Biogen
Gilead Sciences
oncology
gene therapy
With FDA filing for key gene therapy pushed back 6 months, uniQure opens the pocketbook to refresh the pipeline
Endpoints
Tue, 06/22/21 - 10:37 pm
uniQure
M&A
Corlieve Therapeutics
AMT-260
gene therapy
temporal lobe epilepsy
BioMarin's gene therapy lead jumps ship to rival biotech Vedere Bio II
Fierce Biotech
Tue, 06/22/21 - 11:11 am
Vedere Bio II
gene therapy
Biomarin
VectorY raises $38M to develop Alzheimer's, ALS gene therapies
Fierce Biotech
Tue, 06/15/21 - 11:37 pm
VectorY Therapeutics
gene therapy
ALS
Alzheimer's disease
Nightstar becomes nightmare, but Biogen has bigger fish to fry
EP Vantage
Tue, 06/15/21 - 10:30 am
Biogen
Nightstar Therapeutics
M&A
gene therapy
BIIB111
BIIB112
Novartis’ $2 Million Gene Therapy Zolgensma Shows ‘Remarkable’ Results and Offers Hope for Children with SMA
Xtalks
Tue, 06/8/21 - 12:19 pm
Novartis
Zolgensma
SMA
gene therapy
Bluebird's Zynteglo trials set to resume, putting gene therapy back on flight path to FDA filing
Fierce Pharma
Mon, 06/7/21 - 11:09 am
Bluebird Bio
Zynteglo
gene therapy
beta-thalassemia
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