Gene Therapy: Page 4


  • A 3D illustration showing swollen neurons with lamellar inclusions due to accumulation of gangliosides in lysosomes.
    Image attribution tooltip
    Dr Microbe via Getty Images
    Image attribution tooltip

    Freeline cuts more jobs and halts Fabry gene therapy work

    The U.K. biotech will lay off 30% of its workforce and focus on an experimental Gaucher disease treatment, five months after an initial restructuring that cut a hemophilia gene therapy.

    By April 4, 2023
  • An illustration of CRISPR-cas9 gene editing
    Image attribution tooltip
    del Aguila III, Ernesto. (2018). "CRISPR Cas9" [Illustration]. Retrieved from Flickr.
    Image attribution tooltip

    Vertex, CRISPR finish US filing for gene editing drug approval

    The approval application is the first in the U.S. for a CRISPR-based medicine and puts the partners ahead of a rival therapy from Bluebird bio. 

    By April 3, 2023
  • Explore the Trendline
    Image attribution tooltip
    Mario Tama via Getty Images
    Image attribution tooltip
    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
  • DNA strand zoomed in
    Image attribution tooltip

    BillionPhotos via Adobe Stock 

    Image attribution tooltip
    Sponsored by Advanced Clinical

    Changing cell & gene therapy landscape and implications on clinical trials

    Cell & gene therapy clinical research may help treat diseases and change how we approach healthcare.

    By Elizabeth Dugan, Director, Business Development • April 3, 2023
  • Gene therapy of the DNA cell, low-poly design of a human hand with a syringe and a spiral-shaped chromosome. Blue background.
    Image attribution tooltip

    Ilya/Stock.adobe.com

    Image attribution tooltip

    Gene therapy startup Vedere to close two years after launch

    The startup, a successor to a biotech that Novartis bought in 2020, will wind down after preclinical experiments didn’t meet the bar set by its leaders.

    By Updated April 2, 2023
  • Electron microscope image of T regulatory cells interacting with antigen-presenting cells
    Image attribution tooltip
    NIAID. (2019). "T Regulatory Cells" [Microscope image]. Retrieved from Flickr.
    Image attribution tooltip

    Regeneron bets on ‘Treg’ cell therapy with Sonoma deal

    The biotech will work with Sonoma, a well-funded startup run by prominent immunologist Jeffrey Bluestone, to develop cell-based medicines for ulcerative colitis and Crohn’s disease.

    By March 28, 2023
  • A 3D rendering of the CRISPR-cas9 gene editing system.
    Image attribution tooltip
    Meletios Verras via Getty Images
    Image attribution tooltip
    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.
    Image attribution tooltip
    Maddie Meyer via Getty Images
    Image attribution tooltip

    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
  • A sign showing Gilead's logo
    Image attribution tooltip
    Justin Sullivan via Getty Images
    Image attribution tooltip

    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
  • Shot of a young woman using a machine to conduct a medical test in a laboratory
    Image attribution tooltip
    LaylaBird via Getty Images
    Image attribution tooltip
    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
  • A 3D visualization of neurons and neural network with signals.
    Image attribution tooltip
    Evgenii Kovalev via Getty Images
    Image attribution tooltip

    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
  • An illustration of different blood cells types
    Image attribution tooltip
    Grigorii Yalukov via Getty Images
    Image attribution tooltip

    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
    Image attribution tooltip
    Courtesy of Business Wire
    Image attribution tooltip

    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
    Image attribution tooltip
    Courtesy of Intellia Therapeutics
    Image attribution tooltip
    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
    Image attribution tooltip
    Permission granted by Chroma Medicine
    Image attribution tooltip

    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
    Image attribution tooltip
    Courtesy of Intellia Therapeutics
    Image attribution tooltip

    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
  • A piece of DNA stretches vertically along a plain background.
    Image attribution tooltip
    Getty / Edited by BioPharma Dive
    Image attribution tooltip

    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
  • A blurred photo of an empty laboratory
    Image attribution tooltip
    Ozankutsal via Getty Images
    Image attribution tooltip

    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.
    Image attribution tooltip
    Permission granted by Aera Therapeutics
    Image attribution tooltip

    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.
    Image attribution tooltip
    Permission granted by Astellas
    Image attribution tooltip

    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
  • A Roche logo is seen on the side of a building.
    Image attribution tooltip
    Courtesy of Roche
    Image attribution tooltip

    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
  • An illustration of red blood cells.
    Image attribution tooltip
    Pixelimage via Getty Images
    Image attribution tooltip

    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.
    Image attribution tooltip
    Sarah Silbiger/Getty Images via Getty Images
    Image attribution tooltip

    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.
    Image attribution tooltip
    Courtesy of UniQure
    Image attribution tooltip

    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.
    Image attribution tooltip
    OGphoto via Getty Images
    Image attribution tooltip

    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
  • An illustration of red blood cells
    Image attribution tooltip
    ismagilov via Getty Images
    Image attribution tooltip

    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