Gene Therapy


  • A photo of an Editas Medicine scientist in the lab.
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    Permission granted by Editas Medicine
    Gene editing

    Trailing rivals, Editas shares first study data for sickle cell treatment

    Editas said its gene editing therapy appeared safe and was well tolerated in two patients. The data comes five months after U.S. regulators lifted a partial hold on the trial.

    By Kristin Jensen • Dec. 7, 2022
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    Danielle Ternes/BioPharma Dive

    Gene therapy approvals bring validation as field closes year on high

    It’s been a busy fall in gene therapy, with FDA nods for new treatments from CSL and Bluebird as well as a few deals. Catch up on our best stories here. 

    By Dec. 6, 2022
  • Trendline

    Gene 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
  • A photo of Sekar Kathiresan, CEO of Verve Therapeutics
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    Seth Babin/BioPharma Dive
    Gene therapy safety

    Verve shares slide after company reveals details on FDA trial hold

    In a letter to the biotech, the regulator asked for more information on its gene editing medicine for heart disease as well as on its potential risks.

    By Dec. 6, 2022
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    JuSun via Getty Images
    Gene editing

    FDA lifts pause on Beam plans to test base editing cancer therapy

    The FDA’s decision is a step forward for a type of drugmaking known as “multiplex editing,” which involves editing several genes simultaneously.

    By Dec. 2, 2022
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    iStock / Getty Images Plus via Getty Images
    Gene editing

    Gene editing startup iECURE returns to investors for fresh funding

    The company, which draws on research by Jim Wilson’s lab at UPenn, says the $65 million it raised will support the start of human testing of its lead drug through to initial clinical data.

    By Nov. 30, 2022
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    Meletios Verras via Getty Images

    AstraZeneca reveals its cell therapy ambitions with deal for startup

    The British drugmaker will pay as much as $320 million to acquire Neogene Therapeutics, a developer of cell-based treatments for solid tumors — its first significant investment in the field.

    By Nov. 29, 2022
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    Courtesy of Sarepta

    FDA grants speedy review to Sarepta’s Duchenne gene therapy

    The agency will decide on an accelerated approval of Sarepta’s treatment by May 29, months before results are expected from a potentially confirmatory Phase 3 trial.

    By Nov. 28, 2022
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    DC Studio/Stock.adobe.com

    Sponsored by Cardinal Health

    Navigate the complexities of cell and gene therapies

    See how the effective use of CMC expertise helped guide a biopharma company’s development of a CGT product.

    Nov. 28, 2022
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    Permission granted by ICON
    Sponsored by ICON

    Mainstreaming cell and gene therapy – Realizing its potential

    The challenges biopharma companies face in running clinical trials and tips on how to surmount those obstacles.

    Nov. 28, 2022
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    ismagilov via Getty Images

    FDA approves first gene therapy for hemophilia B

    The one-time treatment, which is for the less common “B” form of the bleeding disorder, will be sold in the U.S. by maker CSL for $3.5 million per infusion. 

    By Updated Nov. 23, 2022
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    Permission granted by Editas Medicine
    Gene editing

    Editas to seek partner for CRISPR medicine after lackluster study results

    While the biotech will not develop the gene editing therapy further on its own, CEO Gilmore O’Neill claimed the data are still a meaningful demonstration of what CRISPR can accomplish. 

    By Nov. 17, 2022
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    Permission granted by Ionis Pharmaceuticals and Metagenomi
    Gene editing

    Ionis teams with Metagenomi and dives into gene editing

    The RNA drug developer is paying the richly funded startup $80 million in a wide-ranging alliance that marks its first foray into DNA editing medicines.

    By Nov. 14, 2022
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    Sarah Silbiger via Getty Images

    BioMarin signals lengthier FDA review for hemophilia gene therapy

    The agency wants to see forthcoming study results for the biotech's treatment, which could potentially push out an approval decision by three months.

    By Nov. 8, 2022
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    Seth Babin/BioPharma Dive
    Gene editing

    FDA halts Verve plans to test gene editing therapy for heart disease in US

    Verve didn’t say what led to the FDA’s decision, but claimed it hasn’t observed any safety issues in an ongoing trial in New Zealand and the U.K, where enrollment will continue.

    By Nov. 7, 2022
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    Jonathan Gardner / BioPharma Dive

    Bispecific cancer drugs and gene therapy advances: What to watch at this year’s ASH meeting

    Competition is increasing behind newly approved drugs from Roche and J&J, while the long-term benefit of gene therapies for chronic blood disorders is being put to the test.

    By , Nov. 4, 2022
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    Getty / Edited by BioPharma Dive

    Abeona to submit cell therapy for approval after positive results for skin disorder treatment

    The company said its therapy, which could become the first drug for a type of epidermolysis bullosa, helped heal wounds and reduce pain in a clinical trial.

    By Nov. 3, 2022
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    Spencer Platt via Getty Images

    String of gene therapy deals spurs cautious optimism on Wall Street

    To some analysts, the recent acquisitions of Akouos and AGTC, as well as Astellas’ equity investment in Taysha, are a positive signal for an area of drug research that’s been “in investors' penalty box for much of 2022.”

    By Nov. 1, 2022
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    Jacob Bell / BioPharma Dive

    BioMarin to face FDA advisers before approval decision on hemophilia gene therapy

    Though the panel represents a new regulatory hurdle for Roctavian, BioMarin’s research head said the company has been preparing for such a meeting “over the last few months.”

    By Oct. 27, 2022
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    Courtesy of GSK

    GSK, dialing back cell therapy work, ends deals with Lyell and Adaptimmune

    The British drugmaker terminated the two alliances following disappointing results this year for a once highly touted cell-based treatment for solid tumors.

    By Updated Oct. 26, 2022
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    Courtesy of Astellas

    Astellas takes stake in gene therapy developer Taysha

    Already an investor in gene therapy, the Japanese pharma will buy 15% of the Texas biotech’s stock and gain options to two gene therapies for central nervous system disorders.

    By Oct. 24, 2022
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    Leonid Sorokin via Getty Images

    AGTC yields to biotech downturn with gene therapy buyout deal

    The biotech is the latest in a growing list of genetic medicine developers to sell themselves near record stock lows amid a challenging funding environment.  

    By Oct. 24, 2022
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    Permission granted by Aldevron
    Sponsored by Aldevron

    Simplify IP for your gene therapy with OTS backbones, plasmids and enzymes

    Read how IP-simplified products can transform your speed to market without needing to reassess operations midway through to commercialization.

    Oct. 24, 2022
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    JuSun via Getty Images

    Prime Medicine raises $175M in one of the year’s largest biotech IPOs

    Shares in the gene editing startup, one of only 19 biotechs to debut on Wall Street this year, have fallen 10% since trading began last Thursday.

    By Oct. 20, 2022
  • Lilly bets more than $600M on a gene therapy developer targeting hearing loss

    Like with Prevail Therapeutics, Lilly’s first gene therapy acquisition, the pharma’s planned buyout of Akouos includes a contingent value right that could hike the deal’s overall cost.

    By Oct. 18, 2022
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    Emerging biotech

    CAR-T pioneer Carl June on founding startups and cell therapy’s next act

    The University of Pennsylvania immunologist and inventor of Kymriah spoke with BioPharma Dive about working with pharma, starting companies and the future of the cell therapy field.  

    By Oct. 18, 2022