Gene Therapy: Page 25
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FDA
Regenxbio, stung by trial hold on gene therapy, sues FDA
The biotech seeks to overturn clinical holds placed by the FDA on two gene therapy programs, arguing the agency action was "arbitrary and capricious."
By Ned Pagliarulo • Nov. 11, 2019 -
Lonza taps Cryoport to bolster cell and gene therapy delivery
The Swiss manufacturer inked a deal with the cold chain specialist, which works with sector leaders like Gilead, Novartis and Bluebird bio.
By Kristin Jensen • Nov. 7, 2019 -
Explore the Trendline➔
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TrendlineGene Therapy
Rapid scientific advances have put the gene therapy field at the forefront of biomedical research. But, as recent setbacks have shown, researchers and drugmakers still face major challenges.
By BioPharma Dive staff -
Regenxbio hit with FDA clinical hold, pushes back gene therapy timelines
It's the second trial hold for the gene therapy field in recent weeks, after the FDA partially suspended a Novartis study testing the SMA treatment Zolgensma.
By Andrew Dunn • Nov. 6, 2019 -
Novartis gene therapy ambitions dealt another blow by FDA hold on Zolgensma
A partial trial suspension threatens to delay the gene therapy's expansion into older children, as the FDA works through safety findings from an animal study.
By Jonathan Gardner • Oct. 30, 2019 -
Gilead CAR-T sales slow, renewing growth doubts
Yescarta, the cell therapy Gilead acquired in its $12 billion deal for Kite, appeared to hit a snag, with sales from July to September declining versus the second quarter.
By Ned Pagliarulo • Oct. 25, 2019 -
Bluebird gets European green light for gene therapy production
Although Bluebird won EU approval for Zynteglo in June, the biotech delayed launching the one-time treatment to finalize manufacturing specifications.
By Kristin Jensen • Oct. 24, 2019 -
Novartis' Zolgensma starts strong as about 100 infants are treated with gene therapy
Sales totaled $160 million in the gene therapy's first full quarter of commercial availability, well above Wall Street's expectations.
By Andrew Dunn • Oct. 22, 2019 -
Gene therapy biotechs caught in manufacturing crunch
Rising demand for contract manufacturing services could push companies to invest more in their own capabilities despite the high cost.
By Ned Pagliarulo • Updated Oct. 11, 2019 -
ProQR eye-disease data lend confidence that pivotal trial will succeed
The positive data also helps to keep the Netherlands drugmaker ahead of gene-editing specialists Editas and its partner, Allergan.
By Jonathan Gardner • Oct. 10, 2019 -
Feeling pressure in hemophilia, Novo signs gene editing deal with Bluebird
The deal may come too late, though, as the launch of Hemlibra and approach of gene therapies dampens demand for Novo's factor replacement therapies.
By Jonathan Gardner • Oct. 9, 2019 -
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Sarepta, in shadow of FDA setback, marks gene therapy progress
The biotech released the first functional data from a trial of an experimental gene therapy treating a different type of muscular dystrophy than Exondys 51.
By Jonathan Gardner • Oct. 4, 2019 -
Gene therapy stocks have taken a beating. Their recovery may be slow
Share prices of at least 26 gene therapy biotechs were down on the year in October, a sign of some investor uncertainty on how the drugs will be sold.
By Jacob Bell • Oct. 3, 2019 -
Permission granted by Novartis
Novartis to speed AveXis integration in wake of data manipulation
AveXis will be folded into Novartis' quality organization as a result of a scandal involving altered testing data for the company's gene therapy Zolgensma.
By Ned Pagliarulo • Sept. 24, 2019 -
Novartis, shadowed by data scandal, renews case for Zolgensma
Presymptomatic infants treated with the gene therapy reached motor milestones consistent with normal development, updated study results showed.
By Ned Pagliarulo • Sept. 19, 2019 -
Adverum sinks on first gene therapy data for eye disease
A decline in eyesight post-treatment reflects poorly on the therapy's potential, lengthening the company's odds of catching rival Regenxbio.
By Jonathan Gardner • Sept. 12, 2019 -
BioMarin talks down worries on gene therapy data differences
BioMarin's R&D head said timing of steroid treatment impacted interim Phase 3 data, which had spurred questions on valrox's durability.
By Andrew Dunn • Sept. 10, 2019 -
After scandal, Novartis pledges faster action on data integrity
CEO Vas Narasimhan committed to informing the FDA within five business days of any credible data integrity issues tied to a pending drug application.
By Ned Pagliarulo • Sept. 9, 2019 -
Brian Tucker/BioPharma Dive
ColumnPharmacquired: Vertex has an appetite for deals. Gene therapy is on the menu
CEO Jeffrey Leiden told BioPharma Dive that one or two gene therapy deals wouldn't be surprising as the biotech builds out what he calls its tool kit of technologies.
By Jacob Bell • Sept. 5, 2019 -
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Ultragenyx gene therapy data disappoints investors
The biotech said the current dose of DTX401 will be taken into Phase 3 testing, although a less potent dosage level showed similar efficacy.
By Jonathan Gardner • Sept. 5, 2019 -
Novartis secures NICE nod for Luxturna with discount offer
The U.K. cost agency recommended Luxturna after reaching a deal with Novartis, which sells the Spark gene therapy outside the U.S.
By Ned Pagliarulo • Sept. 4, 2019 -
UniQure stays a step ahead in hemophilia B
Seeing high interest from patients and study investigators, uniQure is enrolling six more participants than planned in its Phase 3 gene therapy study.
By Ned Pagliarulo • Sept. 3, 2019 -
Jacob Bell/BioPharma Dive
Immatics shows Celgene isn't done stocking cell therapy pipeline
As its takeover by Bristol-Myers nears closing, Celgene is committing $75 million to research into T-cell redirecting technology.
By Jonathan Gardner • Aug. 28, 2019 -
Getty / Edited by BioPharma Dive
Gene editing companies oppose altering of embryos
Editing should be restricted to altering faulty genes in tissues, said Sangamo, Bluebird bio and 11 other biotechs in a statement of principles.
By Jonathan Gardner • Updated Aug. 27, 2019 -
Pfizer injects $500M more into gene therapy manufacturing
The money is going toward a North Carolina plant that will make the inactivated viruses used to deliver gene therapies and employ 300 staff.
By Jacob Bell • Aug. 21, 2019 -
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Sarepta now needs flawless progress for gene therapy
An unexpected rejection for the biotech's Vyondys 53 puts investor focus even more tightly on the biotech's earlier-stage muscular dystrophy pipeline.
By Jonathan Gardner • Aug. 20, 2019
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