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» gene therapy
gene therapy
JPM2023: Breakthroughs await, but so do roadblocks
Pharmaphorum
Fri, 01/13/23 - 10:09 am
JPMHC 2023
Inflation Reduction Act
cell therapy
gene therapy
clinical trials
CNS disorders
Pfizer pivots from early-stage rare disease R&D, shifting to external innovation and putting assets up for sale
Fierce Biotech
Fri, 01/6/23 - 10:09 am
Pfizer
R&D
externalization
gene therapy
Rare Diseases
Sarepta marches forward with its potential DMD gene therapy and an expanded Catalent manufacturing deal
Endpoints
Thu, 01/5/23 - 04:58 pm
Sarepta Therapeutics
SRP-9001
gene therapy
Duchenne Muscular Dystrophy
CDMOs
Catalent
Lilly's Prevail, Capsida partner to develop gene therapies for nervous system diseases
BioPharma Dive
Wed, 01/4/23 - 05:32 pm
Eli Lilly
gene therapy
Capsida
CNS disorders
Prevail Therapeutics
Pfizer says hemophilia B gene therapy controlled bleeding in key study
BioPharma Dive
Fri, 12/30/22 - 10:42 am
Pfizer
gene therapy
hemophilia B
fidanacogene elaparvovec
Roche gets FDA nod for Lunsumio, offering convenient option to gene therapies in follicular lymphoma
Fierce Pharma
Wed, 12/28/22 - 08:32 pm
Roche
FDA
Lunsumio
gene therapy
follicular lymphoma
Ferring wins FDA approval for bladder cancer gene therapy
BioPharma Dive
Tue, 12/20/22 - 11:07 am
Ferring Pharmaceuticals
bladder cancer
gene therapy
FDA
Adstiladrin
ASH: Despite partial clinical hold, bluebird's sickle cell gene therapy remains on track
Fierce Pharma
Sun, 12/11/22 - 01:34 pm
Bluebird Bio
ASH 2022
Zynteglo
sickle cell disease
gene therapy
lovo-cel
Despite Eye-Popping $3.5 Million Price Tag For Gene Therapy Hemgenix, Budget Impact For Most Payers Will Be Relatively Small
Forbes
Mon, 12/5/22 - 10:48 am
Hemgenix
gene therapy
hemophilia B
drug pricing
CSL
Do Verve's gene edits pass down to patients' children? The FDA wants to know
Fierce Biotech
Mon, 12/5/22 - 10:42 am
Verve Therapeutics
gene editing
gene therapy
FDA
VERVE-101
CTAD 2022 – Lexeo takes baby steps towards an Alzheimer’s gene therapy
EP Vantage
Sun, 12/4/22 - 01:07 pm
Lexeo
Alzheimer's disease
CTAD
gene therapy
Bristol Myers has change of heart on cardiovascular gene therapy pact, terminating uniQure deal
Fierce Biotech
Tue, 11/29/22 - 10:57 am
Bristol Myers Squibb
uniQure
gene therapy
uniQure, CSL claim first okay for hemophilia B gene therapy
Pharmaphorum
Wed, 11/23/22 - 07:53 am
uniQure
CSL
hemophila B
gene therapy
Hemgenix
FDA
Lysogene's lead gene therapy flunks phase 2/3 trial almost a year after Sarepta walked out
Fierce Biotech
Sun, 11/20/22 - 12:54 pm
Lysogene
gene therapy
clinical trials
LYS-SAF302
Sanfillipo Syndrome
Novartis cuts 275 jobs at Illinois site to consolidate Zolgensma manufacturing in North Carolina
Endpoints
Wed, 11/9/22 - 11:13 pm
Novartis
layoffs
gene therapy
Zolgensma
drug manufacturing
ASH preview – waiting for Editas
EP Vantage
Wed, 11/9/22 - 10:40 am
ASH 2022
sickle cell disease
Bluebird Bio
Sangamo BioSciences
Novartis
Editas Medicine
gene therapy
Vertex Pharmaceuticals
Sanofi
BioMarin's hemophilia gene therapy faces further delays over fresh FDA requests
Fierce Biotech
Tue, 11/8/22 - 10:46 am
Biomarin
gene therapy
FDA
hemophilia
Roctavian
FDA finalizes umbrella trial guidance for cell and gene therapies
RAPS.org
Sun, 11/6/22 - 01:28 pm
FDA
regulatory
clinical trials
umbrella trials
gene therapy
cell therapy
Sarepta preps $1.2B war chest as DMD gene therapy nears accelerated FDA nod
Fierce Biotech
Thu, 11/3/22 - 11:56 am
Sarepta Therapeutics
Duchenne Muscular Dystrophy
SRP-9001
gene therapy
Cost watchdog ICER backs multimillion-dollar price tags for BioMarin, CSL hemophilia gene therapies
Fierce Pharma
Thu, 11/3/22 - 11:02 am
ICER
drug pricing
gene therapy
Biomarin
CSL
Bluebird Bio
hemophilia
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