Gene Therapy: Page 25
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Muscle data doesn't help Solid build faith in its gene therapy
A regulatory delay due to an adverse event, meanwhile, has put Solid further behind Sarepta in the pursuit of a Duchenne muscular dystrophy gene therapy.
By Jonathan Gardner • Dec. 18, 2019 -
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Homology unveils first data for PKU gene therapy, marking early milestone
Data presented Tuesday are from just three patients, but set the pace in a field that includes BioMarin and, most recently, Sangamo Therapeutics.
By Andrew Dunn • Dec. 17, 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 -
Roche's $4.8B deal for Spark cleared by FTC after long delay
An unconditional OK for the deal, which was unexpectedly the target of regulatory scrutiny, should ease sector-wide concerns about acquisitions of gene therapy biotechs.
By Ned Pagliarulo • Updated Dec. 17, 2019 -
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Value-based pricing for gene therapy? Maybe not ready for hemophilia
A small patient population plus large potential savings could make blood-clotting gene therapies an easier sell to payers, biotech executives say.
By Jonathan Gardner • Dec. 11, 2019 -
UniQure, Pfizer updates hint at gene therapy potential in hemophilia B
The two companies are advancing toward market one-time treatments for the blood disorder, with Takeda about to begin clinical testing of its own therapy.
By Jonathan Gardner • Dec. 10, 2019 -
Dive Awards
Drug Launch of the Year: Zolgensma
While clouded by controversy, Novartis' launch of Zolgensma shows gene therapy can be successfully commercialized, even as questions of affordability remain paramount.
By Ned Pagliarulo • Dec. 9, 2019 -
Jonathan Gardner/BioPharma Dive
Sangamo keeps pressure on rivals with updated hemophilia data
Longer follow-up on Sangamo and Pfizer's gene therapy show treated patients continue to be free of bleeds, but one patient's results could spark new questions.
By Ned Pagliarulo • Dec. 7, 2019 -
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Cancer gene therapy backed by Blackstone gets trial win
Results released Thursday are the first to be presented from a Phase 3 study of the therapy, which was recently made the cornerstone of a new company launched by Blackstone and Ferring.
By Jonathan Gardner • Dec. 5, 2019 -
Thermo Fisher opens $90M viral vector manufacturing plant in Massachusetts
The CDMO expects to create more than 200 jobs at the site, which is part of the company's plans to expand its gene therapy business.
By Kristin Jensen • Dec. 4, 2019 -
Astellas joins gene therapy race with $3B Audentes buy
Manufacturing was a key draw for Astellas, which gains access to a plant owned by Audentes that can support clinical and commercial production.
By Ned Pagliarulo • Dec. 3, 2019 -
Fujifilm to invest $120M in gene therapy, build center in Texas
The Japan-based company plans to spend $55 million on the center while also outfitting its joint venture with new clean rooms and bioreactors.
By Kristin Jensen • Nov. 21, 2019 -
The hemophilia gene therapy race faces a critical year in 2020
While BioMarin looks poised to reach market in hemophilia A first, Sangamo and Spark are advancing rival gene therapies to tackle the blood disorder.
By Andrew Dunn • Nov. 20, 2019 -
Takeda sees cell, gene therapy in its future. Is it too late?
Nearly a year on from completing its $62 billion Shire deal, Takeda is pitching investors on its plans to stay one of the industry's leading rare disease drug developers.
By Ned Pagliarulo • Nov. 20, 2019 -
Sponsored by Yourway
The evolving demands of the temperature-controlled supply chain for cell and gene therapy
Cell and gene therapies are heightening the need for advanced temperature control solutions.
By Gulam Jaffer, President, Yourway • Nov. 20, 2019 -
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First look at CRISPR, Vertex gene-editing therapy hints at treatment potential
Results from two patients, one with sickle cell disease and the other with beta-thalassemia, offer an initial glimpse at the disease-altering potential of CRISPR gene-editing.
By Ned Pagliarulo • Updated Nov. 19, 2019 -
Sarepta brings in more gene therapies with StrideBio deal
The deal adds four more neuromuscular candidates to the Exondys maker's already-rich pipeline.
By Jonathan Gardner • Nov. 14, 2019 -
ICER draws new gene therapy pricing framework
One-time and short-term curative therapies require a different approach to evaluating benefit, the cost watchdog group said.
By Jonathan Gardner • Nov. 13, 2019 -
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Solid gene therapy trial halted again by FDA
Shares in Solid fell to a new low as the biotech's muscular dystrophy treatment dropped further behind the leader Sarepta Therapeutics.
By Jonathan Gardner • Nov. 12, 2019 -
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 -
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
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