Gene Therapy: Page 5

  • Illustration of a macrophage devouring a cancer cell.
    Image attribution tooltip
    Design Cells via Getty Images
    Image attribution tooltip

    Kincell spins out of cell therapy biotech, joining wave of manufacturing startups

    The company is the latest to emerge with plans to make production of cell-based medicines easier for researchers and biotechs.

    By • July 26, 2023
  • Futuristic 3D cubes showing DNA base pairs and a double helix.
    Image attribution tooltip
    JuSun via Getty Images
    Image attribution tooltip

    Gene therapy biotech Kriya adds $150M in fresh funds

    Led by Vivek Ramaswamy's brother, Kriya has now raised over $600 million to fund gene therapy research spanning ophthalmology, neurology and metabolic disease.

    By Kristin Jensen • July 26, 2023
  • Colorful DNA double helix. Explore the Trendline➔
    Image attribution tooltip
    iStock 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
  • A photograph of Editas Medicine CEO Gilmore O'Neill
    Image attribution tooltip
    Courtesy of Editas Medicine
    Image attribution tooltip

    Editas names Biogen veteran as top scientist in latest executive change

    Linda Burkly’s appointment follows the exit of CSO Mark Shearman, whose departure Editas announced alongside layoffs earlier this year.

    By • July 24, 2023
  • Man in medical lab manufacturing DNA.
    Image attribution tooltip
    Permission granted by Center for Breakthrough Medicines
    Image attribution tooltip
    Sponsored by Center for Breakthrough Medicines

    Cell and gene therapy is coming of age

    Outsourcing in the cell and gene therapy industry is booming and no longer considered just a “trend.”

    July 24, 2023
  • A street sign for Wall Street in front of the facade of the New York Stock Exchange.
    Image attribution tooltip
    JaysonPhotography via Getty Images
    Image attribution tooltip

    A cancer biotech’s collapse gives Neurogene a path to Wall Street

    The startup, a developer of gene therapies for brain diseases like Rett syndrome, will bypass an IPO by merging with struggling drugmaker Neoleukin.

    By • July 18, 2023
  • A piece of DNA stretches vertically along a plain background.
    Image attribution tooltip
    Getty / Edited by BioPharma Dive
    Image attribution tooltip

    Avrobio halts gene therapy research and considers a sale

    The decision to explore “strategic alternatives” comes about six weeks after the company sold its most advanced treatment to Novartis for nearly $90 million.

    By • July 12, 2023
  • Headshot of Morten Sogaard from Astellas
    Image attribution tooltip
    Permission granted by Jennifer Arcure
    Image attribution tooltip

    Astellas buys rights to 4D Molecular’s technology in latest gene therapy deal

    The licensing agreement for the biotech’s viral vector platform follows recent Astellas agreements with Taysha Gene Therapies and Kate Therapeutics.

    By • July 10, 2023
  • Science lab with table in the middle.
    Image attribution tooltip
    Permission granted by Center for Breakthrough Medicine
    Image attribution tooltip
    Sponsored by Center for Breakthrough Medicines

    Overcoming AAV manufacturing challenges

    Overcome issues with AAV vector scalability and reproducibility. Enable a wider application of gene therapies in a cost-effective and sustainable way.

    July 10, 2023
  • A 3D rendering of the CRISPR-cas9 gene editing system.
    Image attribution tooltip
    Meletios Verras via Getty Images
    Image attribution tooltip

    Pfizer backs CRISPR biotech Caribou in latest cell therapy investment

    Five years after helping launch Allogene Therapeutics, Pfizer is backing another developer of “off-the-shelf” cell therapies in a deal that gives the drugmaker the chance to acquire a multiple myeloma treatment. 

    By Kristin Jensen • July 6, 2023
  • A photograph of a Sarepta building in Boston, MA
    Image attribution tooltip
    Courtesy of Sarepta
    Image attribution tooltip

    Sarepta sells FDA fast pass earned from Duchenne gene therapy approval

    The deal enables Sarepta to quickly cash in on the clearance of Elevidys, with $102 million in new funds to help bankroll its drug research.

    By • July 5, 2023
  • Workers gather inside BioMarin’s gene therapy manufacturing plant in Novato, California.
    Image attribution tooltip
    Courtesy of BioMarin Pharmaceutical
    Image attribution tooltip

    With FDA approval in hand, BioMarin lays out plan to sell $2.9M gene therapy

    The agency granted a long-awaited clearance last Thursday, but unexpected aspects of the hemophilia treatment’s label had some investors worried about its commercial prospects.

    By • June 30, 2023
  • An illustration of red blood cells
    Image attribution tooltip
    Brillianata via Getty Images
    Image attribution tooltip

    BioMarin finally secures FDA approval of hemophilia gene therapy

    After a prolonged journey, the medicine, known as Roctavian, is now cleared for certain patients with hemophilia A, the more common form of the rare bleeding disorder.

    By • June 29, 2023
  • NK Cell (Natural Killer Cell) Attacking a Cancer Cell
    Image attribution tooltip
    luismmolina via Getty Images
    Image attribution tooltip

    With new data, Nkarta battles doubts facing ‘off-the-shelf’ cell therapies

    Though a newer regimen showed promise in patients with acute myeloid leukemia, earlier remissions haven’t held up as well as the company hoped.

    By • June 27, 2023
  • An illustration of different blood cells types
    Image attribution tooltip
    Grigorii Yalukov via Getty Images
    Image attribution tooltip

    Pfizer’s hemophilia B gene therapy inches closer to regulatory approval

    The treatment could become Pfizer’s first marketed gene therapy, an area the pharma has poured significant resources into in recent years.

    By • June 27, 2023
  • A photograph of a Sarepta building in Boston, MA
    Image attribution tooltip
    Courtesy of Sarepta
    Image attribution tooltip

    Sarepta prices Duchenne gene therapy at $3.2M

    The cost makes Sarepta’s treatment among the most expensive medicines in the world. But company executives said they don’t expect significant insurer pushback.

    By • June 22, 2023
  • A photograph of a Sarepta building in Boston, MA
    Image attribution tooltip
    Courtesy of Sarepta
    Image attribution tooltip

    First Duchenne gene therapy approved by FDA for young children

    The approval of Elevidys was a milestone for research into the progressive and deadly disease. But the subsequent failure of a confirmatory trial has raised questions for the FDA.

    By • Updated June 22, 2023
  • Employees of biotechnology company UniQure work in a laboratory.
    Image attribution tooltip
    Courtesy of UniQure
    Image attribution tooltip

    UniQure stock tumbles on ‘confusing’ results for Huntington’s gene therapy

    While patients appear to be doing better than historical data suggests, mixed results on other measures perplexed analysts and sent shares falling.

    By • June 21, 2023
  • A photo of Bluebird bio signage in a corporate lobby
    Image attribution tooltip
    Permission granted by Bluebird bio
    Image attribution tooltip

    Bluebird, playing catch-up, gets decision date for sickle cell gene therapy

    The FDA will issue a decision on lovo-cel by Dec. 20, roughly two weeks after a verdict is expected on a rival treatment from Vertex and CRISPR Therapeutics. 

    By Kristin Jensen • June 21, 2023
  • Two lab scientists work inside a research facility. A man turns to talk to his colleague, who is seated.
    Image attribution tooltip
    Permission granted by Landmark Bio
    Image attribution tooltip
    State of Play

    Cell and gene therapy manufacturing: the next generation of startups

    Joining larger CDMOs like Lonza and Catalent, a new group of companies aims to capitalize on persistent bottlenecks in producing complex genetic treatments.

    By • June 20, 2023
  • Lab workers against digitized background
    Image attribution tooltip
    metamorworks via Getty Images
    Image attribution tooltip
    Sponsored by Center for Breakthrough Medicines

    A right-first-time approach to development

    Avoid costly delays, don't let development oversights derail your advanced therapy commercialization. Read the article.

    June 20, 2023
  • A sign for the Food and Drug Administration is seen on July 20, 2020 in Maryland.
    Image attribution tooltip
    Sarah Silbiger via Getty Images
    Image attribution tooltip

    Ferring, with manufacturing update, takes a step towards first gene therapy sales

    The FDA has cleared Ferring’s planned scale-up process for its bladder cancer treatment Adstiladrin, which the company plans to make available in the U.S. later this year.

    By • June 13, 2023
  • Image attribution tooltip
    Christopher Furlong via Getty Images
    Image attribution tooltip

    AstraZeneca teams up with startup Quell, joining ‘Treg’ cell therapy hunt

    The pharma is the latest large drugmaker to show interest in the emerging technology, following similar deals by Bristol Myers and Regeneron.

    By • June 9, 2023
  • An illustration of an adeno-associated virus.
    Image attribution tooltip
    Dr_Microbe via Getty Images
    Image attribution tooltip

    Using twin viruses, startup AAVantgarde aims to extend gene therapy’s reach

    The biotech has raised about $65 million to test two ways to deliver larger genes into the body, which could help gene therapy treat more diseases.

    By • June 6, 2023
  • Lentivirus cell
    Image attribution tooltip
    fpm via Getty Images
    Image attribution tooltip
    Sponsored by Bio-Rad

    Overcoming common pitfalls in lentiviral cell and gene therapies

    See how ddPCR technology can take your lentiviral cell and gene therapies to the next level.

    By Marwan Alsarraj • May 30, 2023
  • An empty laboratory
    Image attribution tooltip
    Alvarez via Getty Images
    Image attribution tooltip

    PTC lays off staff, fires CFO as neuromuscular drug study fails

    The biotech is giving up on early-stage gene therapy research as part of a restructuring meant to cut costs and redirect resources toward therapies most likely to succeed.

    By Kristin Jensen • May 24, 2023