gene therapy

BioMarin says gene therapy hold will endure as FDA questions cancer risk
BioPharma Dive
Sun, 02/20/22 - 11:54 am
Biomarin
FDA
gene therapy
clinical trials
FDA grounds BioMarin gene therapy trial for months with demand for tumor risk data
Fierce Biotech
Fri, 02/18/22 - 10:32 am
Biomarin
FDA
clinical trials
clinical hold
BMN 307
PKU
gene therapy
SwanBio gets FDA Fast Track designation for adrenomyeloneuropathy therapy
Pharmaceutical Business Review
Thu, 02/17/22 - 10:47 am
SwanBio Therapeutics
gene therapy
FDA
fast track
SBT101
AMN
Regenxbio expands MPS I gene therapy trial after first look at data
Pharmaforum
Fri, 02/11/22 - 10:28 am
Regenxbio
gene therapy
Hurler Syndrome
RGX-111
Pfizer aims to restart late-stage trial of Duchenne gene therapy following safety setback
BioPharma Dive
Thu, 02/10/22 - 08:04 pm
Pfizer
gene therapy
Duchenne Muscular Dystrophy
clinical trials
GPB Scientific becomes Curate Biosciences, targets 50% reduction in autologous cell therapy costs
Biopharma Reporter
Thu, 02/10/22 - 10:21 am
GPB Scientific
Curate Biosciences
rebranding
cell therapy
gene therapy
Sarepta reveals gene editing alliance with biotech exploring alternative delivery methods
BioPharma Dive
Tue, 02/1/22 - 11:18 pm
Sarepta Therapeutics
gene editing
gene therapy
GenEdit
Homology scratches cash itch with manufacturing deal, bagging $130M to fund gene therapy trials
Fierce Biotech
Fri, 01/28/22 - 10:25 am
Homology Medicines
funding
gene therapy
Court ruling on bluebird bio’s gene therapy marketing campaign 'sparks' dismay at Roche's Spark
Fierce Pharma
Thu, 01/27/22 - 10:38 am
Bluebird Bio
gene therapy
sickle cell disease
Roche
Spark Therapeutics
No stranger to headwinds, bluebird runs into two PDUFA delays unrelated to new safety issues
Endpoints
Tue, 01/18/22 - 10:40 am
Bluebird Bio
beti-cel
eli-cel
FDA
gene therapy
beta thalassemia
JPM 2022: Bluebird bio hopes for clear skies ahead as it gears up for trio of gene therapy launches
Fierce Pharma
Thu, 01/13/22 - 10:32 pm
JPMHC 2022
Bluebird Bio
gene therapy
sickle cell disease
beti-cel
5 questions facing gene therapy in 2022
BioPharma Dive
Thu, 01/13/22 - 10:21 pm
gene therapy
funding
FDA
Sarepta, with new Duchenne results, to broach 'possibility' of speedy gene therapy approval
BioPharma Dive
Mon, 01/10/22 - 11:39 pm
Sarepta Therapeutics
Duchenne Muscular Dystrophy
gene therapy
BioMarin's hemophilia A gene therapy hits goal in phase 3, teeing up FDA filing despite durability doubts
Fierce Biotech
Mon, 01/10/22 - 11:32 am
Biomarin
hemophilia A
gene therapy
valrox
clinical trials
Gene therapy player Affinia aims to go public, hoping to raise big money in effort to remake AAVs
Endpoints
Wed, 01/5/22 - 10:37 am
Affinia Therapeutics
IPOs
gene therapy
funding
What are the prospects for the US gene therapy industry in 2022?
Biopharma Reporter
Mon, 12/27/21 - 11:11 am
gene therapy
Novartis to acquire Gyroscope Therapeutics, gaining ocular gene therapy
Biopharma Reporter
Wed, 12/22/21 - 11:16 am
Novartis
Gyroscope Therapeutics
ocular gene therapy
gene therapy
M&A
Pfizer reports patient death in early-stage Duchenne gene therapy trial, halts enrollment
Fierce Biotech
Tue, 12/21/21 - 10:28 am
Pfizer
clinical trials
Duchenne Muscular Dystrophy
gene therapy
patient deaths
PF-06939926
bluebird bio Announces Partial Clinical Hold for Patients Under 18 in Sickle Cell Gene Therapy Clinical Program
Yahoo Finance
Mon, 12/20/21 - 10:54 am
Bluebird Bio
lovo-cel
gene therapy
sickle cell disease
FDA
clinical hold
clinical trials
Spark Therapeutics will invest $575M in a cell and gene therapy innovation center on Drexel’s campus
Technical.ly
Fri, 12/17/21 - 10:58 am
Spark Therapeutics
cell therapy
gene therapy
drug manufacturing
expansions

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