Gene Therapy: Page 4

  • A 3D illustration showing swollen neurons with lamellar inclusions due to accumulation of gangliosides in lysosomes.
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    Dr Microbe via Getty Images
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    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
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    del Aguila III, Ernesto. (2018). "CRISPR Cas9" [Illustration]. Retrieved from Flickr.
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    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
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    Mario Tama via Getty Images
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    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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    BillionPhotos via Adobe Stock 

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    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.
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    Ilya/Stock.adobe.com

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    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
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    NIAID. (2019). "T Regulatory Cells" [Microscope image]. Retrieved from Flickr.
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    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.
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    Meletios Verras via Getty Images
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    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
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    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
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    Justin Sullivan via Getty Images
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    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
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    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
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    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
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    Grigorii Yalukov via Getty Images
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    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
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    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
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    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
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    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
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    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.
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    Getty / Edited by BioPharma Dive
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    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
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    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
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    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
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    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.
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    Courtesy of Roche
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    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
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    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
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    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
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    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
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    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
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    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