Gene Therapy: Page 24
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FDA wants more details on Sarepta gene therapy, potentially delaying final test
A new request from the regulator could tighten the race between Sarepta and Pfizer to prove out a gene therapy for Duchenne muscular dystrophy.
By Ben Fidler • Sept. 10, 2020 -
With latest data, a gene therapy for a deadly brain disease inches closer to market
The results keep Bluebird Bio on track to seek approval of the experimental treatment, known as eli-cel, in Europe later this year and in the U.S. in 2021.
By Jacob Bell • Aug. 29, 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 -
BioMarin's hemophilia gene therapy could have warranted a record price tag, ICER finds
A draft report from the drug pricing watchdog concluded that, under the right conditions, Roctavian may be cost-effective at a price of $2.5 million.
By Jacob Bell • Aug. 28, 2020 -
Danielle Ternes/BioPharma Dive
Deep DiveA cure for hemophilia seemed closer than ever. For many patients, it’s now further out of reach
The surprise rejection of BioMarin's hemophilia A gene therapy delayed a decades-long mission to fix the rare bleeding disorder.
By Jacob Bell • Updated Aug. 19, 2020 -
Editas, AbbVie rework gene editing deal as pioneering CRISPR trial resumes
Development of EDIT-101, the first CRISPR-based therapy to be used in a company-led trial to alter genes within a person's body, will now move forward under Editas' sole ownership.
By Ben Fidler • Aug. 7, 2020 -
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Sponsored by Parexel BiotechCell and gene therapies: For biotechs, collaboration is key to successful innovation
With the right partner, biotech companies can fully embrace cell and gene therapy opportunities.
By Matthew M. Cooney & Roberta L. Steere • July 30, 2020 -
Biogen to study use of key SMA drug after Novartis gene therapy
A planned trial is meant to test whether Spinraza can further help infants and young children who have already received Zolgensma, which, in theory, is meant to be curative if given very early on.
By Ned Pagliarulo • July 21, 2020 -
New data from MeiraGTx help bolster J&J's gene therapy bet
The results are the first from a slate of gene therapies J&J licensed from Meira last year, a deal that remains the pharma's largest investment in the field.
By Ben Fidler • July 17, 2020 -
Spark, chasing BioMarin, plans 2021 start for key hemophilia gene therapy study
The Roche subsidiary said it will begin Phase 3 dosing its hemophilia A gene therapy next year, potentially putting it well behind a competing treatment from BioMarin.
By Jonathan Gardner • July 13, 2020 -
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Third patient dies in halted study of Audentes gene therapy
The three patients, treated for a rare neuromuscular disease, were given a high dose of Audentes' gene therapy.
By Ben Fidler • Updated Aug. 21, 2020 -
In a surprise, UniQure sells its hemophilia gene therapy for $450M
The licensing deal with CSL Behring is one of the largest for a gene therapy, but the partnership could make a buyout of the Dutch firm less likely.
By Ben Fidler • June 25, 2020 -
UniQure gets out the gate first in race for Huntington's gene therapy
The biotech has dosed patients in the first trial of a gene-targeted treatment for the degenerative disease, but competitor Voyager could be chasing soon.
By Jonathan Gardner • June 19, 2020 -
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New CRISPR, gene therapy results strengthen potential for treatment of blood diseases
CRISPR Therapeutics and Vertex can more convincingly say their CRISPR-based medicine is helping three people with beta thalassemia or sickle cell disease, while Bluebird takes another step forward.
By Ned Pagliarulo • June 12, 2020 -
Courtesy of Regeneron Pharmaceuticals
Regeneron expands Intellia gene editing deal as its post-Sanofi future emerges
Regeneron and Intellia will grow their alliance and work on hemophilia, an already competitive space that includes several experimental gene therapies.
By Jonathan Gardner • June 1, 2020 -
Courtesy of Sanofi
With $11B stock sale, Sanofi loosens ties with Regeneron
The French pharma will get a cash windfall by selling most of its 20% stake in Regeneron, spurring speculation as to how it'll spend the money.
By Jonathan Gardner • Updated May 27, 2020 -
Novartis gene therapy Zolgensma cleared for use in Europe
The Swiss pharma estimates 550 to 600 infants are born in Europe each year with spinal muscular atrophy, the hereditary disease Zolgensma treats.
By Kristin Jensen • May 20, 2020 -
Thermo Fisher to invest $180M in new gene therapy plant
The planned facility, to be built in Massachusetts, will rival in size a gene therapy plant in Texas owned by CDMO competitor Lonza.
By Kristin Jensen • May 13, 2020 -
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Dyno spins out of Harvard's laboratories with an aim to make gene therapy better
The startup announced collaborations with Novartis and Sarepta, both leading gene therapy makers, that could be worth $2 billion in total.
By Jonathan Gardner • May 11, 2020 -
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Orchard lays off a quarter of its staff amid major shift
The gene therapy developer has given up on plans to build a new manufacturing facility and is adjusting its clinical resources to focus on more common diseases.
By Kristin Jensen • Updated May 8, 2020 -
Texas university spins out Taysha, a gene therapy startup with 15 programs
The university uses a gene therapy delivery tool that Taysha says can be scaled for both small and large manufacturing needs.
By Jonathan Gardner • April 29, 2020 -
Permission granted by Merck KGaA
Merck KGaA to spend $110M on new gene therapy facility in California
The investment continues a spate of build-outs by Merck KGaA, which, like its contracting peers, sees an opportunity in the fast-growing gene therapy field.
By Kristin Jensen • April 23, 2020 -
Ultragenyx's gene therapy technology finds another interested party
Daiichi Sankyo has agreed to pay $225 million to access a manufacturing platform that Ultragenyx acquired through its buy of Dimension Therapeutics.
By Jacob Bell • April 1, 2020 -
Permission granted by Novartis
No sanctions for Novartis as FDA ends review of gene therapy violations
Quietly completing an inspection review, the FDA indicated it will not penalize Novartis for submitting altered data in its approval application for Zolgensma.
By Ned Pagliarulo • March 30, 2020 -
Permission granted by University of Pennsylvania
Deep DiveBig pharma shied away from gene therapy for years. Academia picked up the slack
The byproduct was richer licensing fees for universities and a throng of startups, but also questions on fair prices and research priorities.
By Jonathan Gardner • March 17, 2020 -
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Sangamo sees vindication in Biogen gene-editing deal
Delivering products nearly ready for clinical testing has sparked greater dealmaking interest from big pharma, Sangamo's CEO told BioPharma Dive.
By Jonathan Gardner • Feb. 28, 2020
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