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companies
» gene therapy
gene therapy
Sarepta Enters Uncharted Territory with Adcomm Approval
BioSpace
Mon, 05/22/23 - 10:22 am
Sarepta Therapeutics
SRP-9001
DMD
Duchenne Muscular Dystrophy
FDA
gene therapy
DMD gene therapy death exposes risks of treating older patients
Fierce Biotech
Mon, 05/22/23 - 10:09 am
Cure Rare Disease
gene therapy
clinical trials
DMD
patient deaths
Krystal's Vyjuvek becomes first topical gene therapy with FDA nod to treat rare skin disease
Fierce Pharma
Sun, 05/21/23 - 10:48 pm
Krystal Biotech
Vyjuvek
dystrophic epidermolysis bullosa
gene therapy
FDA
Siren emerges from stealth with plans to marry gene therapy to cancer immunotherapy
BioPharma Dive
Wed, 05/17/23 - 11:21 am
Siren Biotechnology
gene therapy
cancer immunotherapy
brain cancer
eye cancer
Gene therapy consortium targets eight rare diseases for clinical trials
BioPharma Dive
Tue, 05/16/23 - 10:57 pm
gene therapy
Rare Diseases
clinical trials
Biotech startup Ray raises $100M for vision-restoring gene therapies
BioPharma Dive
Tue, 05/16/23 - 10:30 pm
Ray Therapeutics
gene therapy
blindness
Scribe's CRISPR tech Prevails in new $1.5B biobucks deal with Lilly unit
Fierce Biotech
Tue, 05/16/23 - 12:01 pm
Scribe Therapeutics
Eli Lilly
CRISPR
Prevail Therapeutics
gene therapy
biobucks
Jennifer Doudna
After Hemgenix approvals, uniQure cashes out some of its royalty rights for $375M-plus
Fierce Pharma
Mon, 05/15/23 - 06:23 pm
uniQure
Hemgenix
gene therapy
hemophilia A
FDA advisers narrowly vote in favor of Sarepta's DMD gene therapy
Fierce Biotech
Mon, 05/15/23 - 11:18 am
Sarepta Therapeutics
FDA
DMD
gene therapy
Duchenne Muscular Dystrophy
Takeda cuts 2 midstage GI assets including Crohn's hopeful in latest pipeline cleanup
Fierce Biotech
Thu, 05/11/23 - 11:02 am
Takeda
R&D
gene therapy
earnings
Crohn’s disease
FDA reviewers flag concerns over Sarepta's gene therapy for DMD
Yahoo/Reuters
Wed, 05/10/23 - 10:08 am
Sarepta Therapeutics
FDA
DMD
Duchenne Muscular Dystrophy
gene therapy
Gene & cell therapy manufacturer Ascend launches with more than $130M
Labiotech.eu
Tue, 05/9/23 - 12:20 pm
Ascend Gene & Cell Therapies
gene therapy
drug manufacturing
Life Bio, Forge to develop gene therapies for aging-linked diseases
Pharmaceutical Business Review
Tue, 05/9/23 - 10:41 am
Life Biosciences
Forge Biologics
gene therapy
cGMP manufacturing
Gene Therapies Are Still Hampered By Substantial Delays Between Approval And Launch
Forbes
Wed, 05/3/23 - 11:48 am
gene therapy
drug approvals
Biomarin
Bluebird Bio
FDA
Go or no go? Sarepta’s gene therapy faces scrutiny
EP Vantage
Thu, 04/27/23 - 10:29 am
Sarepta Therapeutics
gene therapy
SRP-9001
Roche
DMD
Duchenne Muscular Dystrophy
Busy with 3 program launches this year, Biogen trims pipeline
Fierce Biotech
Tue, 04/25/23 - 11:40 am
Biogen
stroke
gene therapy
ophthalmology
earnings
Sarepta shares drop as report says FDA almost rejected under-review gene therapy
Yahoo/Reuters
Fri, 04/14/23 - 10:16 am
Sarepta Therapeutics
FDA
gene therapy
DMD
Duchenne Muscular Dystrophy
Vertex/CRISPR's gene editing therapy cost effective at $1.9 million - pricing review group
Yahoo/Reuters
Thu, 04/13/23 - 10:01 am
Vertex Pharmaceutials
CRISPR Therapeutics
gene therapy
sickle cell disease
ICER
A new biotech wants to ease a bottleneck in cell and gene therapy production
BioPharma Dive
Tue, 04/11/23 - 07:28 pm
cell therapy
gene therapy
VintaBio
Layoffs loom as Takeda trims early-stage efforts in AAV gene therapy, rare hematology
Fierce Biotech
Fri, 04/7/23 - 01:45 pm
Takeda
R&D
gene therapy
hematology
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