Gene Therapy: Page 6

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    Jonathan Gardner / BioPharma Dive
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    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
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    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
  • Explore the Trendline➔
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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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    Spencer Platt via Getty Images
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    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
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    Jacob Bell / BioPharma Dive
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    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
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    Courtesy of GSK
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    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
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    Courtesy of Astellas
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    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
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    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
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    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
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    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
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    Eli Lilly & Co.
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    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
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    Permission granted by Carl June
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    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
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    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.
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    ‘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
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    Jacob Bell / BioPharma Dive
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    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
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    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
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    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
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    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
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    Courtesy of Sarepta
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    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
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    Courtesy of Vertex Pharmaceuticals
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    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
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    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
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    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
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    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
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    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
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    Permission granted by Stig Albansson / Pretzel Therapeutics
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    Mitochondrial drugs, with a twist: Pretzel Therapeutics launches with $72.5M in funding

    Scientists at Pretzel believe fixing mutated mitochondrial DNA with a mix of small molecule therapies and gene editing could be key to solving a number of hard-to-treat diseases.

    By • Sept. 12, 2022
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    Christoph Burgstedt via Getty Images
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    Beam-linked startup launches with an eye on the next generation of RNA drugs

    Orbital co-founder and former Alnylam CEO John Maraganore said the startup aims to “overcome some of the shortcomings that have been there with the first generation of RNA companies.” 

    By • Sept. 7, 2022