gene therapy

'Not a horse and pony show': At FDA cell and gene roundtable, officials look for ways to cut red tape
Fierce Biotech
Fri, 06/6/25 - 12:06 pm
cell therapy
gene therapy
Robert F Kennedy Jr.
FDA
CRISPR
regulatory
China
uniQure’s regulatory progress sparks optimism for ‘desperate’ Huntington’s space
BioSpace
Thu, 06/5/25 - 10:01 pm
uniQure
Huntington's disease
AMT-130
gene therapy
Chasing Sarepta, RegenXBio links DMD gene therapy to improved outcomes
Fierce Biotech
Thu, 06/5/25 - 11:01 am
Regenxbio
DMD
gene therapy
Sarepta Therapeutics
Intellia's stock sinks on gene therapy patient's raised enzyme levels, but analysts keep faith
Fierce Biotech
Thu, 05/29/25 - 11:11 am
Intellia Therapeutics
clinical trials
gene therapy
nex-z
transthyretin amyloid cardiomyopathy
Rocket crashes as gene therapy patient dies, FDA imposes hold
Fierce Biotech
Tue, 05/27/25 - 11:35 am
Rocket Pharmaceuticals
gene therapy
patient death
FDA
Danon disease
Thermo Fisher, Mirai Bio link up to advance and produce gene therapies
Fierce Pharma
Wed, 05/21/25 - 11:28 am
Thermo Fisher Scientific
Mirai Bio
CDMOs
gene therapy
RegenXBio cashes in $250M worth of payments for Zolgensma, 2 other gene therapies
Fierce Biotech
Tue, 05/20/25 - 10:18 am
Regenxbio
Novartis
Zolgensma
gene therapy
spinal muscular atrophy
Prime Medicine CEO exits as biotech shelves sole clinical gene therapy, lays off 25% of staff
Fierce Biotech
Mon, 05/19/25 - 10:35 pm
Prime Medicine
gene therapy
layoffs
PM359
Neurogene introduces gene therapy monitoring system after patient death
Clinical Trials Arena
Mon, 05/19/25 - 11:04 am
Neurogene
gene therapy
clinical trials
Rett Syndrome
FDA accepts Regenxbio’s application for MPS II gene therapy
World Pharmaceuticals Frontiers
Wed, 05/14/25 - 11:15 am
Regenxbio
FDA
RGX-121
Hunter Syndrome
gene therapy
Bluebird bio warns of potential bankruptcy after another buyout delay
Fierce Pharma
Tue, 05/13/25 - 11:16 am
Bluebird Bio
bankruptcy
M&A
gene therapy
Lexeo laid off 15% of staff in April to focus on lead cardiac gene therapies
Fierce Biotech
Mon, 05/12/25 - 12:05 pm
Lexeo Therapeutics
layoffs
gene therapy
Sarepta tries to quell concerns over DMD gene therapy Elevidys as patients delay treatment after death report
Fierce Pharma
Wed, 05/7/25 - 11:25 am
Sarepta Therapeutics
DMD
Duchenne Muscular Dystrophy
earnings
Elevidys
gene therapy
Neurocrine hands back 2 CNS gene therapy programs to Voyager
Fierce Biotech
Wed, 05/7/25 - 11:13 am
Neurocrine Biosciences
gene therapy
Voyager Therapeutics
CNS
Vertex discontinues AAV research for gene therapies
Fierce Biotech
Sun, 05/4/25 - 12:06 pm
Vertex Pharmaceuticals
AAV gene therapy
gene therapy
As Pfizer backs out of hemophilia gene therapy space, CSL hopes Hemgenix is here to stay
Fierce Pharma
Fri, 04/11/25 - 11:32 am
Pfizer
gene therapy
hemophilia
hemophilia B
Hemgenix
CSL Behring
Lexeo locks in pivotal trial plan as clinical data pump up prospects of cardiac gene therapy
Fierce Biotech
Mon, 04/7/25 - 11:22 am
Lexeo Therapeutics
Friedreich’s ataxia
gene therapy
clinical trials
Eli Lilly helps struggling Sangamo with $1.4B gene therapy deal
Fierce Biotech
Thu, 04/3/25 - 10:14 pm
Sangamo Therapeutics
Eli Lilly
neurotropics
gene therapy
Sarepta’s Duchenne Gene Therapy Placed on Hold in Europe Pending Death Investigation
BioSpace
Thu, 04/3/25 - 11:26 am
Sarepta Therapeutics
Roche
gene therapy
patient deaths
Europe
Eleviys
DMD
Biopharmas laid off staffers at least 63 times in Q1: Fierce Biotech analysis
Fierce Biotech
Tue, 04/1/25 - 11:46 am
layoffs
biotech
cell therapy
gene therapy

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