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» gene therapy
gene therapy
Pfizer says hemophilia gene therapy meets late-stage study goal
BioPharma Dive
Wed, 07/24/24 - 11:53 am
Pfizer
gene therapy
hemophilia A
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
cell therapy
gene therapy
FDA
CBER
OTP
Lexeo links gene therapy to improvements in rare heart disease but disappoints investors
Fierce Biotech
Mon, 07/15/24 - 10:01 am
Lexeo Therapeutics
gene therapy
Friedreich’s ataxia
Leqembi sales inch higher; Spark’s pivot leads to layoffs
BioPharma Dive
Fri, 07/12/24 - 11:29 am
Eisai
Biogen
Leqembi
Alzheimer's disease
Roche
Spark Therapeutics
gene therapy
layoffs
Jaguar Gene’s JAG201 therapy trial for autism receives FDA approval
Clinical Trials Arena
Wed, 07/10/24 - 11:07 am
Jaguar Gene Therapy
FDA
gene therapy
JAG201
autism
UniQure, with sale of plant, outsources Hemgenix manufacturing
BioPharma Dive
Mon, 07/1/24 - 11:31 am
uniQure
gene therapy
drug manufacturing
Genezen
Hemgenix
FDA Rejects Rocket’s Gene Therapy, Cites Need for Additional CMC Info
BioSpace
Sun, 06/30/24 - 10:03 pm
Rocket Pharmaceuticals
FDA
gene therapy
Kresladi.
Rocket gene therapy’s flight to approval delayed by FDA rejection over manufacturing questions
Fierce Biotech
Fri, 06/28/24 - 11:26 am
Rocket Pharmaceuticals
Kresladi
FDA
gene therapy
After full approval, Sarepta lays out its next moves
BioSpace
Wed, 06/26/24 - 11:31 am
Sarepta Therapeutics
DMD
Elevidys
gene therapy
Sarepta Duchenne gene therapy wins broader use from FDA
BioPharma Dive
Thu, 06/20/24 - 06:09 pm
Sarepta Therapeutics
gene therapy
FDA
DMD
Elevidys
Pfizer setback brings questions for Duchenne gene therapy ahead of Sarepta decision
BioPharma Dive
Thu, 06/13/24 - 11:07 pm
Pfizer
DMD
gene therapy
FDA
Sarepta Therapeutics
Elevidys
Regenxbio CEO to step down after 15 years
BioPharma Dive
Wed, 06/12/24 - 11:23 pm
Regenxbio
Pharma CEOs
gene therapy
Kenneth Mills
Merck KGaA to buy gene therapy tools maker for $600M
BioPharma Dive
Thu, 05/23/24 - 09:37 pm
Merck KGaA
M&A
MilliporeSigma
Gamma Biosciences
gene therapy
Cell and Gene Therapies Still Have Long Way to Go in Fulfilling Promise
BioSpace
Fri, 05/10/24 - 11:39 am
ASGCT
cell therapy
gene therapy
Bluebird Boasts Nearly 140% Revenue Jump, Still Misses Target
BioSpace
Fri, 05/10/24 - 11:10 am
Bluebird Bio
earnings
gene therapy
In gene therapy race, bluebird leads Vertex in activating treatment centers but trails in patient starts
Fierce Pharma
Thu, 05/9/24 - 07:17 pm
gene therapy
Vertex Pharmaceuticals
Bluebird Bio
ASGCT24: Regeneron’s Gene Therapy Shows ‘Dramatic’ Hearing Improvement in Deaf Kids
BioSpace
Wed, 05/8/24 - 11:33 am
Regeneron
DB-OTO
genetic deafness
gene therapy
ASGCT
Sarepta Seeks Label Expansion, Bigger Market for DMD Gene Therapy
BioSpace
Wed, 05/8/24 - 11:13 am
Sarepta Therapeutics
DMD
Elevidys
gene therapy
Krystal Biotech touts topical gene therapy uptake, plots overseas launches
Fierce Pharma
Tue, 05/7/24 - 06:27 pm
Krystal Biotech
Vyjuvek
dystrophic epidermolysis bullosa
gene therapy
Verve moves forward with backup base editing therapy
BioPharma Dive
Tue, 05/7/24 - 11:29 am
Verve Therapeutics
clinical trials
gene therapy
heart disease
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