gene therapy

BioMarin nets conditional approval for valrox in Europe, marking the first hemophilia gene therapy
Endpoints
Thu, 08/25/22 - 10:59 am
Biomarin
hemophilia A
valrox
Europe
gene therapy
Roctavian
Ovid takes a novel approach to gene therapy
EP Vantage
Thu, 08/25/22 - 10:54 am
Ovid Therapeutics
gene therapy
epilepsy
phages
bluebird bio Scores First U.S. Approval for Lentiviral Vector Gene Therapy
BioSpace
Wed, 08/17/22 - 11:34 pm
Bluebird Bio
FDA
gene therapy
beta thalassemia
Zynteglo
beti-cel
Novartis reports deaths of two patients treated with Zolgensma gene therapy
BioPharma Dive
Thu, 08/11/22 - 11:32 pm
Novartis
gene therapy
Zolgensma
SMA
patient deaths
spinal muscular atrophy
Freeline's One Dose Gene Therapy could Transform Hemophilia B Treatment
BioSpace
Sun, 07/24/22 - 07:56 pm
hemophilia
hemophilia B
clinical trials
Freeline Therapeutics
gene therapy
FLT180a
Gene therapy for ovarian cancer fails PhIII study — sinking VBL Therapeutics' shares
Endpoints
Wed, 07/20/22 - 09:33 am
VBL Therapeutics
gene therapy
ovarian cancer
VB-111
ofra-vec
Ultragenyx buys gene therapy partner after new study results
BioPharma Dive
Tue, 07/19/22 - 10:45 am
Ultragenyz
gene therapy
M&A
Angelman Syndrome
GeneTx Biotherapeutics
Myocarditis case mars Sarepta DMD gene therapy readout
Pharmaforum
Thu, 07/7/22 - 10:23 am
Sarepta Therapeutics
gene therapy
Duchenne Muscular Dystrophy
SRP-9001
myocarditis
uniQure shares spike as investors see bright spot in 12-month Huntington's gene therapy data
Fierce Biotech
Thu, 06/23/22 - 07:51 pm
uniQure
Huntington's disease
gene therapy
AMT-130
Carbon Biosciences Launches with $38 Million Series A Financing to Advance Novel Gene Therapy Platform and Pipeline
BioSpace
Tue, 06/21/22 - 12:06 pm
Carbon Biosciences
funding
gene therapy
Bluebird bio’s future in balance as FDA weighs gene therapy approvals
BioPharma Dive
Thu, 06/9/22 - 10:32 am
Bluebird Bio
gene therapy
Astellas, despite recent setbacks, opens $100M gene therapy plant
BioPharma Dive
Wed, 06/8/22 - 11:05 pm
Astellas
gene therapy
drug manufacturing
North Carolina
Precision hails phase 1/2 data as proof it could deliver the first approved off-the-shelf CAR-T
Fierce Biotech
Wed, 06/8/22 - 11:00 pm
Precision Biosciences
CAR-T
gene therapy
gene editing
non-Hodgkin lymphoma
Ahead of 2-day adcomm, FDA sounds positive on one bluebird gene therapy but questions remain for another
Endpoints
Tue, 06/7/22 - 10:08 am
FDA
Bluebird Bio
gene therapy
eli-cel
beti-cel
Even at $2M+, ICER calls bluebird's potential gene therapy for blood disorder 'superior' to standard of care
Endpoints
Thu, 06/2/22 - 10:22 am
Bluebird Bio
ICER
gene therapy
FDA
beti-cel
CSL-UniQure Hemophilia B Gene Therapy Scores Priority Review
BioSpace
Tue, 05/24/22 - 04:34 pm
CSL Behring
uniQure
hemophilia B
gene therapy
FDA
priority review
etranacogene dezaparvovec
Ohio gene therapy industry gets 'buzz' – but needs to sell itself more
Bizjournals
Sun, 05/22/22 - 09:02 pm
Ohio
gene therapy
Columbus
100% overall survival data for gene therapy fuel Rocket for flight to regulators
Fierce Biotech
Fri, 05/20/22 - 10:48 am
Rocket Pharmaceuticals
gene therapy
Leukocyte Adhesion Deficiency-I
FDA explains plans to bolster cell and gene therapy approvals through wider messaging
RAPS.org
Thu, 05/19/22 - 09:01 pm
FDA
regulatory
cell therapy
gene therapy
With its lead gene therapy nearing the clinic, SwanBio leans heavily on Syncona to close $56M series B
Fierce Biotech
Wed, 05/18/22 - 10:25 am
Sycona
SwanBio Therapeutics
funding
gene therapy

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