Gene Therapy

  • A 3D rendering of the CRISPR-cas9 gene editing system.
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    Meletios Verras via Getty Images
    Gene editing

    Vertex pays CRISPR to use its gene editing tech for diabetes drugs

    Vertex will license CRISPR technology to develop insulin-producing islet cells that are more resistant to immune rejection, adding to other efforts directed at Type 1 diabetes.

    By March 27, 2023
  • A view of Moderna headquarters on May 08, 2020 in Cambridge, Massachusetts.
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    Maddie Meyer via Getty Images

    Moderna inks second genetic medicine deal in a month

    The biotech plans to use Generation Bio’s nonviral delivery technology to get nucleic acid-based therapies to immune cells and into the liver.

    By March 23, 2023
  • 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
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    Justin Sullivan via Getty Images

    Gilead’s Yescarta extends survival in lymphoma study

    The results, the first for a CAR-T therapy, are further evidence supporting the use of the complex therapies earlier in a patient’s disease course. 

    By March 21, 2023
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    LaylaBird via Getty Images
    Sponsored by MilliporeSigma

    Addressing manufacturing challenges in gene therapy development

    As new gene therapies are developed to treat a growing number of indications, patients may soon have access to novel treatment options and potential cures.

    By Kate Silver • March 20, 2023
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    Evgenii Kovalev via Getty Images

    Switch Therapeutics launches with $52M to capitalize on RNA medicine ‘era’

    The company’s name comes from its goal of developing drugs that only “switch” on in target cells, potentially enabling more precise therapies.

    By March 14, 2023
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    Grigorii Yalukov via Getty Images

    BioMarin, as expected, sees delay to FDA review of hemophilia gene therapy

    The agency is considering three-year follow-up data in its review of Roctavian, pushing back the potential U.S. arrival of BioMarin’s drug once again.

    By Kristin Jensen • March 7, 2023
  • A photo of Alfred Sandrock, former chief medical officer and head of R&D at Biogen
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    Courtesy of Business Wire

    Voyager licenses gene therapy tools to Novartis

    The Swiss drugmaker's decision is the latest in a string of partnership announcements for the gene therapy maker, which aims to bounce back from past research failures.

    By March 6, 2023
  • An Intellia Therapeutics lab
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    Courtesy of Intellia Therapeutics
    Gene editing

    FDA clears Intellia to start US tests of ‘in vivo’ gene editing drug

    The announcement, which follows regulatory setbacks for some of Intellia’s peers, is a “big win” for the gene editing field, according to one analyst.

    By March 2, 2023
  • Chroma Medicine CEO Catherine Stehman-Breen
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    Permission granted by Chroma Medicine

    Amid industry turbulence, Chroma raises fresh funding to edit the epigenome

    As many drug startups struggle to gain cash, Chroma has raised another $135 million to back its research, a new twist on genetic medicine.

    By Updated March 1, 2023
  • Intellia Therapeutics headquarters in Cambridge, MA
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    Courtesy of Intellia Therapeutics

    Sickle cell pipeline narrows as gene therapy developers rethink research plans

    Graphite Bio and Sangamo are stopping work on their respective sickle cell gene therapies, while Intellia revealed partner Novartis ended development of its genetic treatment for the blood disease.

    By Feb. 23, 2023
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    Getty / Edited by BioPharma Dive

    Medicaid, with planned payment pilot, girds for influx of pricey gene therapies

    The proposed model could help state Medicaid agencies explore different kinds of outcomes-based payment schemes, but may come too late to prepare for the first sickle cell gene therapy.

    By Feb. 21, 2023
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    Ozankutsal via Getty Images

    Talaris restructures, ending effort to develop a cell therapy for kidney transplants

    Following a patient death and difficulty enrolling study participants, the company is laying off a third of its staff and ending a pair of studies for a two-decade-old program once owned by Novartis.

    By Feb. 16, 2023
  • A photo of Feng Zhang, founder of Aera Therapeutics, speaking with Akin Akinc, the company's CEO. Both are wearing goggles and lab coats.
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    Permission granted by Aera Therapeutics

    With Aera, CRISPR pioneer Feng Zhang goes after gene therapy’s delivery problem

    The company’s platform technology is built on research from Zhang’s lab at the Broad Institute, which focuses on proteins that could be used to package and deliver nucleic acids.

    By Feb. 16, 2023
  • Astellas' U.S. headquarters in Northbrook, IL.
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    Permission granted by Astellas

    Astellas switches CEOs amid plans to pursue ‘aggressive’ growth

    Naoki Okamura, currently Astellas’ chief strategy officer, will replace current CEO Kenji Yasukawa as the company continues a strategic reboot that’s seen it invest more heavily in gene therapy.

    By Feb. 6, 2023
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    Courtesy of Roche

    Roche writes off $3B on lower sales forecasts for gene therapies, cancer drugs

    Among the drugs Roche recorded impairment charges on are four gene therapies acquired in the company's 2019 deal for Spark Therapeutics. 

    By Feb. 3, 2023
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    Pixelimage via Getty Images

    Magenta to explore sale, merger after study setback

    The biotech recently stopped a trial of its experimental conditioning regimen over safety concerns. Now it’s halting further development work as it undertakes a strategic review.

    By Feb. 3, 2023
  • The headquarters of the FDA, which has recently issued a recall for Teleflex air filters used in hospital respirators.
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    Sarah Silbiger/Getty Images via Getty Images

    Head of FDA gene therapy office set to retire in March

    Wilson Bryan, a key decision maker in the agency’s oversight of genetic medicines, is expected to leave next month amid an organizational revamp of his office, the FDA confirmed.

    By Kristin Jensen • Feb. 1, 2023
  • Employees of biotechnology company UniQure work in a laboratory.
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    Courtesy of UniQure
    ALS drug development

    UniQure nabs another gene therapy for ALS

    For $10 million upfront, UniQure has licensed rights to its second experimental treatment for the condition, a medicine from startup Apic Bio that should enter human testing later this year.

    By Jan. 31, 2023
  • 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
  • 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
  • A photo of Alfred Sandrock, former chief medical officer and head of R&D at Biogen
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    Courtesy of Business Wire

    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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    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
  • An illustration for BioPharma Dive series on gene therapies for different diseases
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    Danielle Ternes/BioPharma Dive
    Gene editing

    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