Gene Therapy: Page 25
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BioMarin inches closer to a gene therapy first
A decision by the FDA on whether to approve valrox, potentially the first gene therapy for hemophilia, should come by late August.
By Jacob Bell • Feb. 21, 2020 -
With supply tight, Novartis readies gene therapy plant for production
Bringing the facility in Durham, North Carolina, online is a critical step for the Swiss pharma's plans to expand the market for Zolgensma, its gene therapy for spinal muscular atrophy.
By Ned Pagliarulo • Feb. 21, 2020 -
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 -
Audentes to spend $110M on new gene therapy plant in North Carolina
The biotech, which was bought last year by Astellas, joins Pfizer, Bluebird bio and AveXis in choosing to locate gene therapy manufacturing in the state.
By Ned Pagliarulo • Feb. 18, 2020 -
Decibel CEO steps down as company pares workforce
The Third Rock-backed company put an acting executive in place as it repositions for development in gene therapy and regenerative medicine.
By Jonathan Gardner • Jan. 29, 2020 -
Zolgensma sales grow, signaling gene therapy momentum
Another 100 infants received Novartis' gene therapy in the fourth quarter, supported by broad insurance coverage and more newborn screening.
By Ned Pagliarulo • Jan. 29, 2020 -
Pfizer lays out gene therapy aspirations
With more Phase 3 trials on the near horizon, the big pharma plans to add one new gene therapy project per year to its pipeline.
By Jonathan Gardner • Jan. 28, 2020 -
FDA, expecting a gene therapy boom, firms up policies
In seven guidance documents, the agency gave drugmakers guidelines on manufacturing, testing and long-term follow-up for the one-time treatments.
By Ned Pagliarulo • Jan. 28, 2020 -
Danielle Ternes/BioPharma Dive
Orchard isn't for the picking, Biohaven's Allergan headache, and who's on Vascepa?
Amarin CEO John Thero had one question for investors eager to hear about his company's plans for its heart pill, while Orchard made a case for independence.
By Jacob Bell • Jan. 16, 2020 -
Permission granted by Novartis
Q&ANovartis' David Lennon on next steps for AveXis, selling Zolgensma's price to the public
While Zolgensma's launch has gone well, the pharma still faces challenges in expanding the gene therapy's market and addressing criticism over its price.
By Andrew Dunn • Jan. 15, 2020 -
Nationwide Children's, a gene therapy leader, launches manufacturing spinout
Andelyn Biosciences, named after two children treated with gene therapy at Nationwide, will operate as a for-profit subsidiary of the Ohio-based hospital.
By Ned Pagliarulo • Updated Jan. 15, 2020 -
Danielle Ternes/BioPharma Dive
Alexion's neuro bet, Rubius' trial troubles and biotech's gene therapy milestones
On the first day of the JPM conference, Alexion mapped out ambitious plans in neuroscience, while Rubius did its best to explain delays in testing its first drug.
By Jacob Bell , Andrew Dunn • Jan. 14, 2020 -
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BioMarin sets gene therapy sights beyond hemophilia
At the JPM conference, the biotech announced a manufacturing lift and a new trial start that affirm its ambitions in gene therapy go beyond its closely watched hemophilia A treatment.
By Jacob Bell • Jan. 13, 2020 -
Ultragenyx boosted by trial data for 2nd gene therapy
One analyst called the results, which sparked a 30% jump in Ultragenyx's stock price, better than expected. The biotech is now preparing a Phase 3 trial.
By Jonathan Gardner • Jan. 10, 2020 -
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Solid cuts one-third of staff, goes all in on delayed gene therapy
Re-starting a halted muscular dystrophy trial is Solid's priority as it puts a second drug and an assistive device on the backburner.
By Jonathan Gardner • Jan. 9, 2020 -
Permission granted by Roche
Roche hands $1B to Sarepta in major return to gene therapy deals
The Swiss pharma acquired a muscular dystrophy treatment for markets outside the U.S. in a pact that could reach a value of $10 billion.
By Jonathan Gardner • Dec. 23, 2019 -
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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 -
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 -
Getty / Edited by BioPharma Dive
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
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