gene therapy

FDA delays decision on Duchenne gene therapy and considers narrower approval
FDA delays decision on Duchenne gene therapy and considers narrower approval
BioPharma Dive
Sarepta Therapeutics
FDA
DMD
Duchenne Muscular Dystrophy
gene therapy
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ElevateBio Announces $401 Million Series D Financing to Further Accelerate Growth
ElevateBio Announces $401 Million Series D Financing to Further Accelerate Growth
BioSpace
ElevateBio
funding
cell therapy
gene therapy
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Novartis to buy Avrobio gene therapy for $88M, leaving rest of the biotech on the shelf
Novartis to buy Avrobio gene therapy for $88M, leaving rest of the biotech on the shelf
Fierce Biotech
Novartis
gene therapy
Avrobio
AVR-RD-04
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Sarepta Enters Uncharted Territory with Adcomm Approval
Sarepta Enters Uncharted Territory with Adcomm Approval
BioSpace
Sarepta Therapeutics
SRP-9001
DMD
Duchenne Muscular Dystrophy
FDA
gene therapy
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DMD gene therapy death exposes risks of treating older patients
DMD gene therapy death exposes risks of treating older patients
Fierce Biotech
Cure Rare Disease
gene therapy
clinical trials
DMD
patient deaths
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Krystal's Vyjuvek becomes first topical gene therapy with FDA nod to treat rare skin disease
Krystal's Vyjuvek becomes first topical gene therapy with FDA nod to treat rare skin disease
Fierce Pharma
Krystal Biotech
Vyjuvek
dystrophic epidermolysis bullosa
gene therapy
FDA
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Siren emerges from stealth with plans to marry gene therapy to cancer immunotherapy
Siren emerges from stealth with plans to marry gene therapy to cancer immunotherapy
BioPharma Dive
Siren Biotechnology
gene therapy
cancer immunotherapy
brain cancer
eye cancer
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Gene therapy consortium targets eight rare diseases for clinical trials
Gene therapy consortium targets eight rare diseases for clinical trials
BioPharma Dive
gene therapy
Rare Diseases
clinical trials
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Biotech startup Ray raises $100M for vision-restoring gene therapies
Biotech startup Ray raises $100M for vision-restoring gene therapies
BioPharma Dive
Ray Therapeutics
gene therapy
blindness
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Scribe's CRISPR tech Prevails in new $1.5B biobucks deal with Lilly unit
Scribe's CRISPR tech Prevails in new $1.5B biobucks deal with Lilly unit
Fierce Biotech
Scribe Therapeutics
Eli Lilly
CRISPR
Prevail Therapeutics
gene therapy
biobucks
Jennifer Doudna
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After Hemgenix approvals, uniQure cashes out some of its royalty rights for $375M-plus
After Hemgenix approvals, uniQure cashes out some of its royalty rights for $375M-plus
Fierce Pharma
uniQure
Hemgenix
gene therapy
hemophilia A
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FDA advisers narrowly vote in favor of Sarepta's DMD gene therapy
FDA advisers narrowly vote in favor of Sarepta's DMD gene therapy
Fierce Biotech
Sarepta Therapeutics
FDA
DMD
gene therapy
Duchenne Muscular Dystrophy
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Takeda cuts 2 midstage GI assets including Crohn's hopeful in latest pipeline cleanup
Takeda cuts 2 midstage GI assets including Crohn's hopeful in latest pipeline cleanup
Fierce Biotech
Takeda
R&D
gene therapy
earnings
Crohn’s disease
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FDA reviewers flag concerns over Sarepta's gene therapy for DMD
FDA reviewers flag concerns over Sarepta's gene therapy for DMD
Yahoo/Reuters
Sarepta Therapeutics
FDA
DMD
Duchenne Muscular Dystrophy
gene therapy
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Gene & cell therapy manufacturer Ascend launches with more than $130M
Gene & cell therapy manufacturer Ascend launches with more than $130M
Labiotech.eu
Ascend Gene & Cell Therapies
gene therapy
drug manufacturing
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Life Bio, Forge to develop gene therapies for aging-linked diseases
Life Bio, Forge to develop gene therapies for aging-linked diseases
Pharmaceutical Business Review
Life Biosciences
Forge Biologics
gene therapy
cGMP manufacturing
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Gene Therapies Are Still Hampered By Substantial Delays Between Approval And Launch
Gene Therapies Are Still Hampered By Substantial Delays Between Approval And Launch
Forbes
gene therapy
drug approvals
Biomarin
Bluebird Bio
FDA
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Go or no go? Sarepta’s gene therapy faces scrutiny
Go or no go? Sarepta’s gene therapy faces scrutiny
EP Vantage
Sarepta Therapeutics
gene therapy
SRP-9001
Roche
DMD
Duchenne Muscular Dystrophy
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Busy with 3 program launches this year, Biogen trims pipeline
Busy with 3 program launches this year, Biogen trims pipeline
Fierce Biotech
Biogen
stroke
gene therapy
ophthalmology
earnings
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Sarepta shares drop as report says FDA almost rejected under-review gene therapy
Sarepta shares drop as report says FDA almost rejected under-review gene therapy
Yahoo/Reuters
Sarepta Therapeutics
FDA
gene therapy
DMD
Duchenne Muscular Dystrophy
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