Gene Therapy: Page 23
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Intellia cleared to start key CRISPR drug study in UK
The trial would be the first test of a systemically administered gene editing treatment that uses the Nobel Prize-winning technology to alter DNA.
By Ned Pagliarulo • Oct. 19, 2020 -
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Roche, aiming to make better gene therapies, turns to an emerging startup
The Swiss pharma is the third large drugmaker to partner with Harvard spinout Dyno Therapeutics, a small biotech aiming to improve gene therapy technology.
By Ben Fidler • Oct. 14, 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 -
Yujin Kim / MedTech Dive, original photo courtesy of U.S. Food and Drug Administration
FDA puts Voyager study on hold in latest gene therapy speed bump
The biotech won't be able to start human testing of an experimental Huntington's disease treatment until regulators see more manufacturing data.
By Jonathan Gardner • Oct. 13, 2020 -
Pfizer and Sangamo, after rival's setback, kick off late-stage gene therapy trial
The FDA's recent rejection of a hemophilia gene therapy from BioMarin sets a high standard for approval. Will Pfizer fare any better?
By Jonathan Gardner • Oct. 7, 2020 -
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Solid gets all-clear from FDA to restart gene therapy trial
Worrisome immune responses had led the FDA to halt Solid's study. Now, the agency will permit Solid to continue after the biotech made adjustments to its manufacturing and patient enrollment procedures.
By Jonathan Gardner • Oct. 1, 2020 -
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Sponsored by BiomerePreclinical done right: The importance of using relevant animal models in gene therapy studies
Gene therapy is no longer an approach for the future. It's a technique used now.
Oct. 1, 2020 -
Permission granted by Emergent BioSolutions
Sponsored by Emergent BiosolutionsGene therapy solution: The value of a CDMO as your end-to-end partner
As gene therapy research continues to expand, innovators in this space will need CDMOs with highly specific expertise, facilities, and equipment.
Sept. 29, 2020 -
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New data bolsters Sarepta gene therapies for two deadly muscle diseases
The results disclosed at a medical meeting on Monday show that all patients tested have seen their motor skills stabilize or improve when history suggests they wouldn't.
By Ben Fidler • Sept. 28, 2020 -
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Gene therapy company Taysha completes sprint from first funding to IPO
Led by alumni of Zolgensma developer AveXis, the university spinout could have four experimental therapies in the clinic by the end of 2021.
By Jonathan Gardner • Updated Sept. 24, 2020 -
Permission granted by Takeda Pharmaceutical
Takeda adds to cell therapy push with new manufacturing site
The Japanese drugmaker has made cell therapy a focus, advancing three experimental cancer treatments into clinical testing with plans for two more.
By Ned Pagliarulo • Sept. 15, 2020 -
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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 -
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
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