Gene Therapy


  • A micrograph of a myeloma neoplasm in a bone marrow biopsy.
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    OGphoto via Getty Images

    J&J, Legend’s cancer cell therapy has early success in key clinical trial

    The trial is an important test of Carvykti’s potential for earlier use in multiple myeloma and found treatment helped extend progression-free survival over standard regimens.

    By Jan. 27, 2023
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    ismagilov via Getty Images

    Magenta halts study of targeted conditioning drug after participant’s death

    The biotech said one patient died after receiving an experimental antibody drug designed to deplete certain blood and bone marrow cells before a transplant.

    By Updated Jan. 26, 2023
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    Trendline

    The BioPharma Dive Outlook on 2023

    Against a stormy backdrop, 2022 did bring bright spots in Alzheimer’s, obesity, breast cancer and rare disease. Dig in to BioPharma Dive’s 2023 outlook for what’s in store this year. 

    By BioPharma Dive staff
  • A photograph of an infusion bag of Carvkyti, J&J and Legend Biotech's cancer cell therapy
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    Permission granted by Johnson & Johnson

    Sales of J&J, Legend cell therapy plateau amid production challenges

    Carvykti, which was approved a year ago for hard-to-treat multiple myeloma, generated $55 million in fourth-quarter sales, on par with the previous quarter.

    By Jan. 25, 2023
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    The image by Pixabay is licensed under CC BY 1.0

    FDA lifts hold on Astellas gene therapy for Pompe disease

    The regulator’s decision ends a seven-month study pause that followed a report of one participant experiencing mild symptoms of peripheral neuropathy.

    By Jan. 20, 2023
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    Courtesy of Business Wire
    Q&A

    Al Sandrock on his short retirement and taking on a biotech turnaround project

    In a conversation at the J.P. Morgan Healthcare conference, the longtime Biogen executive discussed his hesitance to jump back into an executive role and why an opportunity to run Voyager Therapeutics drew him in.

    By Jan. 17, 2023
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    Novartis
    Gene therapy safety

    EMA weighs new safety guidance for Novartis’ gene therapy Zolgensma

    The discussions come months after two patients died from acute liver injury following treatment with the spinal muscular atrophy therapy.

    By Jan. 13, 2023
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    Danielle Ternes/BioPharma Dive

    5 questions facing gene therapy in 2023

    Buoyed by recent approvals, the field faces a pivotal year that’s likely to bring new treatments as well as more challenges.

    By Jan. 12, 2023
  • A sign for Pfizer is seen outside the Pfizer headquarters on November 9, 2020 in New York City.
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    David Dee Delgado via Getty Images

    Pfizer pares back early-stage research in rare disease, cancer

    The drugmaker said it is considering licensing out some assets to shorten development timelines, and will stop work on early viral-based gene therapies. 

    By Jan. 6, 2023
  • A general view of the Pfizer Headquarters sign on November 10, 2020 in Tadworth, England.
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    Dan Kitwood via Getty Images

    Pfizer says hemophilia B gene therapy controlled bleeding in key study

    The Phase 3 results showed the one-time treatment outperformed standard factor replacement in adults with the bleeding disorder, but suggest it may be less potent than CSL and UniQure’s rival therapy. 

    By Dec. 29, 2022
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    Sarah Silbiger via Getty Images

    Ferring wins FDA approval for bladder cancer gene therapy

    The OK caps a long development journey for the treatment, which was previously turned back by the agency. Ferring doesn’t expect it to be available until the second half of next year, however.

    By Dec. 19, 2022
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    Permission granted by Bluebird bio

    FDA lifts hold on Bluebird’s sickle cell gene therapy

    Removal of the partial study suspension, which the agency imposed last December after a case of persistent anemia, helps clear the way for Bluebird to submit an approval application early next year. 

    By Dec. 19, 2022
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    Leonid Sorokin via Getty Images

    Sio Gene Therapies to dissolve after years of setbacks

    Having failed to find a buyer or potential partner, the biotech company has opted instead to shut down and liquidate its assets.

    By Dec. 15, 2022
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    Jonathan Gardner / BioPharma Dive

    Roche’s hemophilia gene therapy holds steady with longer-term data

    Roche’s subsidiary Spark has offered few updates on its hemophilia A treatment since being acquired in 2019. New data at ASH show the therapy can maintain levels of a key blood-clotting protein for years. 

    By Dec. 12, 2022
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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
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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
  • Futuristic 3D cubes showing DNA base pairs and a double helix.
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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
  • An illustration of a gene being inserted into a helix strand.
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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
  • A photo of an Editas Medicine scientist in the lab.
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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
  • A photo of Ionis CEO Brett Monia and Metagenomi CEO Brian Thomas.
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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