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gene therapy

Ultragenyx tweaks gene therapy dosing to dial up efficacy after seeing signs of activity

Fierce Biotech

Fri, 10/4/24 - 09:38 am

Ultragenyx Pharmaceutical

Genespire raises €46.6 million (~$52 million) in a Series B round to advance its first pediatric in-vivo gene therapy into the clinic

Yahoo Finance

Wed, 09/25/24 - 11:58 am

Bluebird bio lays off 25% of workforce as gene therapy pioneer struggles to stay afloat

Fierce Pharma

Tue, 09/24/24 - 11:35 am

4DMT’S Gene Therapy for AMD Clears Phase II, Heads to Late-Stage Studies

BioSpace

Thu, 09/19/24 - 11:07 am

clinical trials

wet age-related macular degeneration

Why some gene therapies face sluggish launches

Medical Marketing and Media

Mon, 09/16/24 - 10:21 pm

Gene therapy startup emerges with green light for first-of-its-kind trial

BioPharma Dive

Thu, 09/12/24 - 11:33 am

adeno-associated viruses

BridgeBio slashes gene therapy budget after early-phase data fall short of 'transformational' target

Fierce Biotech

Wed, 09/11/24 - 11:15 am

clinical trials

Novartis ignites new stage of Voyager pact with $15M capsid deal

Fierce Biotech

Thu, 09/5/24 - 11:28 am

Voyager Therapeutics

Gene therapy biotech Medera looks back to SPAC as way to go public

Fierce Biotech

Thu, 09/5/24 - 11:27 am

cardiovascular disease

Is anyone taking the world’s priciest drugs?

Pharma Voice

Fri, 08/30/24 - 11:28 am

CRISPR Therapeutics

Vertex Pharmaceuticals

Sarepta Therapeutics

Cell and gene therapy investment, once booming, is now in a slump

BioPharma Dive

Thu, 08/29/24 - 11:22 am

Q2 2024 update: cell & gene therapy related hiring actvity in the pharma industry

Clinical Trials Arena

Tue, 08/27/24 - 08:09 pm

Vertex lifts sales guidance as launch of gene therapy Casgevy picks up steam

Fierce Pharma

Fri, 08/2/24 - 11:52 am

Vertex Pharmaceuticals

Despite Late-Stage Hemophilia Win, Sangamo Remains in Do-or-Die Situation

BioSpace

Wed, 07/31/24 - 10:48 am

Sangamo BioSciences

Pfizer takes $230M hit after axing failed DMD gene therapy—and tosses out RSV combo vaccine

Fierce Biotech

Tue, 07/30/24 - 11:50 am

Pfizer says hemophilia gene therapy meets late-stage study goal

BioPharma Dive

Wed, 07/24/24 - 11:53 am

clinical trials

giroctocogene fitelparvovec

FDA proposes cell and gene therapy site tours to help regulators learn from industry

Fierce Pharma

Mon, 07/15/24 - 11:18 pm

Lexeo links gene therapy to improvements in rare heart disease but disappoints investors

Fierce Biotech

Mon, 07/15/24 - 10:01 am

Lexeo Therapeutics

Friedreich’s ataxia

Leqembi sales inch higher; Spark’s pivot leads to layoffs

BioPharma Dive

Fri, 07/12/24 - 11:29 am

Alzheimer's disease

Spark Therapeutics

Jaguar Gene’s JAG201 therapy trial for autism receives FDA approval

Clinical Trials Arena

Wed, 07/10/24 - 11:07 am

Jaguar Gene Therapy

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