Gene Therapy


  • Three business men stand on a city street for a photo
    Image attribution tooltip
    Courtesy of Cure Ventures
    Image attribution tooltip

    Cure Ventures backs a cell therapy startup targeting Parkinson’s

    The startup, Kenai Therapeutics, has raised $82 million and will be chaired by Jeff Jonas, a new Cure partner and former CEO of Sage Therapeutics.

    By Feb. 29, 2024
  • An illustration of red blood cells
    Image attribution tooltip
    ismagilov via Getty Images
    Image attribution tooltip

    BioMarin preaches patience amid slow sales for hemophilia gene therapy

    The company earned only $3.5 million last year from its Roctavian treatment, far below the $50 million to $150 million range it had forecast eight months ago.

    By Feb. 23, 2024
  • 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
  • An illustration of B cells producing antibodies
    Image attribution tooltip
    Love Employee via Getty Images
    Image attribution tooltip

    NEJM paper fills in details on ‘remarkable’ CAR-T result in autoimmune disease

    The full results spotlight cell therapy’s potential to treat lupus and other inflammatory conditions, but also the problems drugmakers must still solve.

    By Feb. 21, 2024
  • Packaging for Iovance Biotherapeutics' cellular medicine Amtagvi is held by a technician wearing gloves.
    Image attribution tooltip
    Courtesy of Iovance Biotherapeutics
    Image attribution tooltip

    Iovance, with approval of ‘TIL’ cell therapy, readies for complex launch

    The biotech set a price of roughly $515,000 per patient for its therapy Amtagvi, the first to be approved based on a decades-old technique of using tumor-infiltrating lymphocytes. 

    By Feb. 20, 2024
  • A photograph of a Sarepta building in Boston, MA
    Image attribution tooltip
    Courtesy of Sarepta
    Image attribution tooltip

    FDA to review expanded use of Sarepta Duchenne gene therapy

    The agency will decide by June 21 whether to broaden eligibility for Elevidys, and won’t convene a group of outside experts beforehand.

    By Feb. 16, 2024
  • An illustration of two lungs on a scientific background colored orange.
    Image attribution tooltip
    Mohammed Haneefa Nizamudeen via Getty Images
    Image attribution tooltip

    Intellia, ReCode partner on genetic medicines for cystic fibrosis

    The partnership will use Intellia's "DNA writing” technology, and focus on people with the lung disease who have limited or no available treatments

    By Feb. 15, 2024
  • An illustration of sickled red blood cells blocking a blood vessel.
    Image attribution tooltip
    Dr_Microbe via Getty Images
    Image attribution tooltip

    New CMS pilot to test payment scheme for pricey sickle cell gene therapies

    The agency is planning a pilot program across states to help sickle cell patients access treatments like the newly approved Casgevy and Lyfgenia.

    By Jan. 31, 2024
  • A sign shows the logo of Eli Lilly's Research Laboratories
    Image attribution tooltip
    jetcityimage via Getty Images
    Image attribution tooltip

    Lilly gene therapy finding puts focus on hearing loss treatment pipeline

    An 11-year-old boy born deaf can hear after receiving Lilly's genetic medicine. Others, including biotechs in China and Regeneron in the U.S., are taking aim at the same drug target.

    By Updated Jan. 25, 2024
  • A rendering of an adeno-associated virus, colored blue-green, on a black background.
    Image attribution tooltip
    Yabusaka Design via Getty Images
    Image attribution tooltip

    Gene therapy biotech Jaguar spins out manufacturing company

    Called Advanced Medicine Partners, the new company will offer specialized cell and gene therapy manufacturing services to biotech and pharma clients.

    By Updated Jan. 18, 2024
  • A photo of Vertex Pharmaceuticals' Boston headquarters.
    Image attribution tooltip
    Courtesy of Vertex Pharmaceuticals
    Image attribution tooltip

    FDA widens approval of Vertex’s CRISPR medicine to treat beta thalassemia

    The agency’s decision to expand use of Casgevy, which won a landmark OK for sickle cell disease in December, comes two months ahead of schedule.

    By Jan. 16, 2024
  • Intellia Therapeutics headquarters in Cambridge, MA
    Image attribution tooltip
    Courtesy of Intellia Therapeutics
    Image attribution tooltip

    Intellia to lay off staff, cut some early research

    The CRISPR specialist joins some of its genetic medicine peers in trimming staff, announcing an “organizational streamlining” to focus resources on prioritiy drug programs.

    By Jan. 4, 2024
  • An illustration of different blood cells types
    Image attribution tooltip
    Grigorii Yalukov via Getty Images
    Image attribution tooltip

    Pfizer wins Canadian OK for hemophilia treatment, its first gene therapy

    The hemophilia B therapy, which Pfizer will sell in Canada as Beqvez, is also under review in the U.S., where the FDA expects to make a decision by the second quarter.

    By Jan. 3, 2024
  • A Novartis logo is seen on the side of an office building.
    Image attribution tooltip
    Permission granted by Novartis
    Image attribution tooltip

    Novartis deepens ties with Voyager via $100M gene therapy deal

    The deal builds on an existing alliance and includes tools Novartis will use in gene therapies for Huntington’s disease and spinal muscular atrophy.

    By Jan. 2, 2024
  • Signs for Johnson & Johnson are seen on company offices in California.
    Image attribution tooltip
    Mario Tama via Getty Images
    Image attribution tooltip

    J&J bets bigger on MeiraGTx’s eye gene therapy

    Ahead of a Phase 3 readout that could come next year, the pharma is paying $130 million in upfront and near-term cash for rights to the retinitis pigmentosa treatment that it didn’t already own.

    By Dec. 21, 2023
  • Employees of biotechnology company UniQure work in a laboratory.
    Image attribution tooltip
    Courtesy of UniQure
    Image attribution tooltip

    Investors still aren’t sold on UniQure’s gene therapy for Huntington’s

    The biotechnology company’s share price fell more than 10% Tuesday after the disclosure of more data from a small study.

    By Dec. 19, 2023
  • A 3D rendering of the CRISPR-cas9 gene editing system.
    Image attribution tooltip
    Meletios Verras via Getty Images
    Image attribution tooltip

    Editas cashes in on CRISPR patent with Vertex deal

    The deal, struck after Editas was awarded ownership of a key patent for the landmark gene editing technology, could be the first of many like it.

    By Kristin Jensen • Dec. 14, 2023
  • A dias lit up in red with a sign reading 65th ASH Annual Meeting & Exposition
    Image attribution tooltip

    ASH/Todd Buchanan

    Image attribution tooltip

    At ASH, doctors acclaim new sickle cell gene therapies, but are cautious on details

    Uptake of Casgevy and Lyfgenia may be slow despite their dramatic benefit, physicians said, citing complexities in treatment, manufacturing and reimbursement.

    By , Dec. 13, 2023
  • Nicole Gormley on stage at ASH23, presenting a data visualization from the FDA.
    Image attribution tooltip
    Courtesy of Nick Agro/American Society of Hematology
    Image attribution tooltip

    ASH23: Pharma branding, Editas’ high bar and clinical trial diversity

    Editas had the tall task Monday of convincing ASH attendees its gene therapy for sickle cell disease could improve on Casgevy and Lyfgenia.

    By , Dec. 11, 2023
  • A needle is seen piercing a cell membrane in a composite image behind an office building.
    Image attribution tooltip

    Photo illustration: Shaun Lucas/Industry Dive; CRISPR Therapeutics; Gregor Fischer/DPA/Newscom

    Image attribution tooltip
    Deep Dive // Gene editing

    ‘No tolerance for failure’: An oral history of the first CRISPR medicine

    A new sickle cell disease therapy developed by CRISPR Therapeutics and Vertex Pharmaceuticals is now approved in the U.S. and U.K. This is the story of how it came to be.

    By , Dec. 10, 2023
  • A man in a suit walks by a sign that reads American Society of Hematology
    Image attribution tooltip

    ASH/Todd Buchanan 2023

    Image attribution tooltip

    Bluebird gene therapy, now approved for sickle cell, shows durable benefit in study update

    Follow-up data continue to show Lyfgenia can address the pain crises people with sickle cell experience, although Bluebird’s therapy will be compared to Vertex and CRISPR Therapeutics’ Casgevy.

    By Dec. 9, 2023
  • A CRISPR Therapeutics sign is seen on the side of a building viewed through trees.
    Image attribution tooltip
    Permission granted by CRISPR Therapeutics
    Image attribution tooltip
    Gene editing

    CRISPR therapy for sickle cell approved by FDA in gene editing milestone

    Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, Casgevy is the first drug based on the Nobel Prize-winning gene editing technology to reach market.

    By Updated Dec. 8, 2023
  • A CRISPR Therapeutics sign is seen above a door to an office building
    Image attribution tooltip
    Permission granted by CRISPR Therapeutics
    Image attribution tooltip

    CRISPR eyes autoimmune disease in revamp of cell therapy plans

    The gene editing biotech is shelving two of its most advanced cancer drugs, and joining a growing group of companies exploring cell-based medicines for inflammatory diseases like lupus.

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

    BioMarin secures hemophilia gene therapy coverage in Germany

    Drawn-out negotiations led to a lower price than initially expected, but analysts called the agreement a step forward for the biotech company.

    By Kristin Jensen • Nov. 29, 2023
  • People walk past the Nasdaq MarketSite on October 12, 2022 in New York City.
    Image attribution tooltip
    Michael M. Santiago via Getty Images
    Image attribution tooltip

    Freeline, after cuts, agrees to take-private deal with Syncona

    The planned acquisition adds to a string of bargain buyouts and reverse mergers in the gene therapy field. 

    By Nov. 22, 2023
  • A 3D rendering of the CRISPR-cas9 gene editing system.
    Image attribution tooltip
    Meletios Verras via Getty Images
    Image attribution tooltip

    World’s first CRISPR medicine approved in UK for sickle cell, beta thalassemia

    Clearance of Vertex Pharmaceuticals and CRISPR Therapeutics' Casgevy in the U.K. comes ahead of expected regulatory decisions in the U.S. and Europe.

    By Nov. 16, 2023