Gene Therapy: Page 4


  • An illustration of T cells attacking a cancer cell
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    Peddalanka Ramesh Babu via Getty Images
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    Precision sells lymphoma drug to Imugene in retreat from cell therapy research

    The struggling biotech also plans to offload other cell therapy assets, including a multiple myeloma therapy, and focus exclusively on gene editing medicines.

    By Aug. 16, 2023
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    nopparit via Getty Images
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    HIV cell therapy startup Addimmune heads to Wall Street via blank-check merger

    The deal will help fund a cell-based treatment that completed Phase 1 testing last year and is designed to provide a “functional cure” for patients with HIV infections.

    By Aug. 10, 2023
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    iStock 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
  • Regeneron ups genetic medicine investment with Decibel buyout

    Third Rock-founded Decibel, which has shed much of its value since a 2021 IPO, expected to run out of money within a year.

    By Aug. 9, 2023
  • A photograph of a Sarepta building in Boston, MA
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    Courtesy of Sarepta
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    Sarepta offers early look at closely watched gene therapy launch

    One patient received its Duchenne gene therapy, Elevidys, this week and a handful more could follow shortly, surprising some analysts and raising near-term sales expectations.

    By Aug. 3, 2023
  • An image of Intergalactic Therapeutics' non-viral, closed-loop DNA platform.
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    Permission granted by Intergalactic Therapeutics
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    A once buzzy gene therapy startup lays off staff and shuts down

    Intergalactic, a developer of non-viral gene therapies built by ex-Biogen executive Michael Ehlers, is folding less than two years after its launch. 

    By Kristin Jensen • Aug. 2, 2023
  • Vertex CEO Reshma Kewalramani
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    Permission granted by Vertex Pharmaceuticals
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    Vertex taking ‘flexible’ approach to payment talks for experimental CRISPR therapy

    The company is discussing various reimbursement models for its sickle cell treatment with government and commercial insurers ahead of an FDA approval decision later this year.

    By Aug. 2, 2023
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    Lisa Maree Williams via Getty Images
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    AstraZeneca buys Pfizer’s early gene therapy work for up to $1B

    The British pharma is deepening its investment in genetic medicine through a deal for a group of Pfizer’s gene therapy delivery tools, while the New York giant cuts back. 

    By July 28, 2023
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    Design Cells via Getty Images
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    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
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    JuSun via Getty Images
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    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
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    Courtesy of Editas Medicine
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    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
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    Permission granted by Center for Breakthrough Medicines
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    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
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    JaysonPhotography via Getty Images
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    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
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    Getty / Edited by BioPharma Dive
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    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
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    Permission granted by Jennifer Arcure
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    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
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    Permission granted by Center for Breakthrough Medicine
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    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.
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    Meletios Verras via Getty Images
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    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
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    Courtesy of Sarepta
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    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.
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    Courtesy of BioMarin Pharmaceutical
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    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
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    Brillianata via Getty Images
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    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
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    luismmolina via Getty Images
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    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
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    Grigorii Yalukov via Getty Images
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    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
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    Courtesy of Sarepta
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    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
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    Courtesy of Sarepta
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    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.
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    Courtesy of UniQure
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    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
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    Permission granted by Bluebird bio
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    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