Gene Therapy: Page 2


  • The sign identifying the FDA headquarters in front of its building in White Oak, Maryland.
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    Sarah Silbiger via Getty Images

    BioMarin signals lengthier FDA review for hemophilia gene therapy

    The agency wants to see forthcoming study results for the biotech's treatment, which could potentially push out an approval decision by three months.

    By Nov. 8, 2022
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    Seth Babin/BioPharma Dive
    Gene editing

    FDA halts Verve plans to test gene editing therapy for heart disease in US

    Verve didn’t say what led to the FDA’s decision, but claimed it hasn’t observed any safety issues in an ongoing trial in New Zealand and the U.K, where enrollment will continue.

    By Nov. 7, 2022
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    Spencer Platt via Getty Images
    Trendline

    The BioPharma Dive Outlook on 2023

    Against a stormy backdrop, 2022 did bring bright spots in Alzheimer’s, obesity, breast cancer and rare disease. Dig in to BioPharma Dive’s 2023 outlook for what’s in store this year. 

    By BioPharma Dive staff
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    Jonathan Gardner / BioPharma Dive

    Bispecific cancer drugs and gene therapy advances: What to watch at this year’s ASH meeting

    Competition is increasing behind newly approved drugs from Roche and J&J, while the long-term benefit of gene therapies for chronic blood disorders is being put to the test.

    By , Nov. 4, 2022
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    Getty / Edited by BioPharma Dive

    Abeona to submit cell therapy for approval after positive results for skin disorder treatment

    The company said its therapy, which could become the first drug for a type of epidermolysis bullosa, helped heal wounds and reduce pain in a clinical trial.

    By Nov. 3, 2022
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    Spencer Platt via Getty Images

    String of gene therapy deals spurs cautious optimism on Wall Street

    To some analysts, the recent acquisitions of Akouos and AGTC, as well as Astellas’ equity investment in Taysha, are a positive signal for an area of drug research that’s been “in investors' penalty box for much of 2022.”

    By Nov. 1, 2022
  • The FDA logo on a glass pane at the agency's campus in Silver Spring, Maryland.
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    Jacob Bell / BioPharma Dive

    BioMarin to face FDA advisers before approval decision on hemophilia gene therapy

    Though the panel represents a new regulatory hurdle for Roctavian, BioMarin’s research head said the company has been preparing for such a meeting “over the last few months.”

    By Oct. 27, 2022
  • A photo of a building showing a GSK sign in London.
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    Courtesy of GSK

    GSK, dialing back cell therapy work, ends deals with Lyell and Adaptimmune

    The British drugmaker terminated the two alliances following disappointing results this year for a once highly touted cell-based treatment for solid tumors.

    By Updated Oct. 26, 2022
  • A rendering of Astellas' planned biotech campus in South San Francisco
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    Courtesy of Astellas

    Astellas takes stake in gene therapy developer Taysha

    Already an investor in gene therapy, the Japanese pharma will buy 15% of the Texas biotech’s stock and gain options to two gene therapies for central nervous system disorders.

    By Oct. 24, 2022
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    Leonid Sorokin via Getty Images

    AGTC yields to biotech downturn with gene therapy buyout deal

    The biotech is the latest in a growing list of genetic medicine developers to sell themselves near record stock lows amid a challenging funding environment.  

    By Oct. 24, 2022
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    Permission granted by Aldevron
    Sponsored by Aldevron

    Simplify IP for your gene therapy with OTS backbones, plasmids and enzymes

    Read how IP-simplified products can transform your speed to market without needing to reassess operations midway through to commercialization.

    Oct. 24, 2022
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    JuSun via Getty Images

    Prime Medicine raises $175M in one of the year’s largest biotech IPOs

    Shares in the gene editing startup, one of only 19 biotechs to debut on Wall Street this year, have fallen 10% since trading began last Thursday.

    By Oct. 20, 2022
  • Lilly bets more than $600M on a gene therapy developer targeting hearing loss

    Like with Prevail Therapeutics, Lilly’s first gene therapy acquisition, the pharma’s planned buyout of Akouos includes a contingent value right that could hike the deal’s overall cost.

    By Oct. 18, 2022
  • A photo of Carl June in his laboratory at the University of Pennsylvania
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    Permission granted by Carl June
    Emerging biotech

    CAR-T pioneer Carl June on founding startups and cell therapy’s next act

    The University of Pennsylvania immunologist and inventor of Kymriah spoke with BioPharma Dive about working with pharma, starting companies and the future of the cell therapy field.  

    By Oct. 18, 2022
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    Meletios Verras via Getty Images
    State of Play

    ‘In vivo’ cell therapy: expanding beyond CAR-T

    At least five startups have emerged with new ways to genetically modify immune cells within the body, an approach that, if successful, could widen the field of CAR-T treatment.

    By Oct. 18, 2022
  • A photo of FDA CBER Director Peter Marks delivering remarks at a public workshop on March 3, 2020.
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    Ermath, Michael. (2020). "Individualized Therapies Workshop" [Photograph]. Retrieved from Flickr.

    ‘We have to find a way’: FDA seeks solutions to aid bespoke gene therapy

    Gene therapies could help treat many ultra-rare diseases. But they may not get developed if drugmakers can’t build a sustainable business around them, CBER director Peter Marks said at a conference. 

    By Oct. 13, 2022
  • The FDA logo on a glass pane at the agency's campus in Silver Spring, Maryland.
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    Jacob Bell / BioPharma Dive

    BioMarin gets FDA decision date for hemophilia gene therapy, but questions on review remain

    After issuing a surprise rejection of the therapy in 2020, the FDA has now agreed to again review BioMarin’s Roctavian therapy. An approval decision is expected by March 31.

    By Kristin Jensen • Oct. 13, 2022
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    Permission granted by Kevin Trimmer/Ascidian Therapeutics

    Ascidian starts up with $50M and a twist on RNA editing

    The startup, run by Translate Bio cofounder Romesh Subramanian, believes its RNA “exon editing” approach could have long-lasting effects without the risks that come with editing DNA. 

    By Oct. 12, 2022
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    Peddalanka Ramesh Babu via Getty Images

    Allogene starts first pivotal trials of an ‘off-the-shelf’ cell therapy for cancer

    The biotech believes the two Phase 2 trials initiated Thursday can support approval applications for what could be the first allogeneic cancer cell therapy.  

    By Oct. 7, 2022
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    ismagilov via Getty Images

    BioMarin resubmits its hemophilia gene therapy to the FDA

    The resubmission has been long awaited after BioMarin’s original application was unexpectedly rejected by the FDA two years ago. The company expects a decision around the middle of next year.

    By Sept. 30, 2022
  • A photograph of a Sarepta building in Boston, MA
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    Courtesy of Sarepta

    Sarepta asks FDA to approve first gene therapy for Duchenne muscular dystrophy

    The application comes a year earlier than previously had been expected, as company says drug reviewers are open to accelerated review based on data from early-stage trials.

    By Sept. 29, 2022
  • A photo of Vertex Pharmaceuticals' Boston headquarters.
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    Courtesy of Vertex Pharmaceuticals

    Vertex given green light to seek US approval of CRISPR-based therapy

    The company and its development partner, CRISPR Therapeutics, will begin submitting a rolling application in November. The blood disease treatment is the first of its kind to near an FDA review. 

    By Sept. 27, 2022
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    ismagilov via Getty Images

    Pfizer, Sangamo set to resume gene therapy study after safety delay

    Concerns over blood clotting risk had derailed testing of the hemophilia treatment last year, leading the companies to adjust their trial protocol.

    By Sept. 23, 2022
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    Permission granted by Bluebird bio

    Back-to-back gene therapy approvals give Bluebird shot at survival

    The FDA’s clearances of Zynteglo and Skysona are a boost to Bluebird, and could help lift the research field after a series of setbacks. But selling the high-priced therapies will be a challenge.

    By Sept. 19, 2022
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    Courtesy of Bluebird bio

    Bluebird wins FDA approval of gene therapy for rare brain disorder

    The therapy, called Skysona and cleared to treat cerebral adrenoleukodystrophy, is the product of more than a decade of work by Bluebird. It will cost $3 million per patient. 

    By Updated Sept. 17, 2022
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    Danielle Ternes/BioPharma Dive

    BioMarin reports cancer case in hemophilia gene therapy trial

    The development comes three weeks after Roctavian was approved in Europe and ahead of a planned regulatory submission in the U.S. Drug regulators have not ordered a trial hold, however.

    By Sept. 13, 2022