Gene Therapy: Page 5

  • A photograph of a Sarepta building in Boston, MA
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    Courtesy of Sarepta
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    FDA grants speedy review to Sarepta’s Duchenne gene therapy

    The agency will decide on an accelerated approval of Sarepta’s treatment by May 29, months before results are expected from a potentially confirmatory Phase 3 trial.

    By • Nov. 28, 2022
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    DC Studio/Stock.adobe.com

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    Sponsored by Cardinal Health

    Navigate the complexities of cell and gene therapies

    See how the effective use of CMC expertise helped guide a biopharma company’s development of a CGT product.

    Nov. 28, 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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    Permission granted by ICON
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    Sponsored by ICON

    Mainstreaming cell and gene therapy – Realizing its potential

    The challenges biopharma companies face in running clinical trials and tips on how to surmount those obstacles.

    Nov. 28, 2022
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    ismagilov via Getty Images
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    FDA approves first gene therapy for hemophilia B

    The one-time treatment, which is for the less common “B” form of the bleeding disorder, will be sold in the U.S. by maker CSL for $3.5 million per infusion. 

    By • Updated Nov. 23, 2022
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    Permission granted by Editas Medicine
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    Gene editing

    Editas to seek partner for CRISPR medicine after lackluster study results

    While the biotech will not develop the gene editing therapy further on its own, CEO Gilmore O’Neill claimed the data are still a meaningful demonstration of what CRISPR can accomplish. 

    By • Nov. 17, 2022
  • A photo of Ionis CEO Brett Monia and Metagenomi CEO Brian Thomas.
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    Permission granted by Ionis Pharmaceuticals and Metagenomi
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    Ionis teams with Metagenomi and dives into gene editing

    The RNA drug developer is paying the richly funded startup $80 million in a wide-ranging alliance that marks its first foray into DNA editing medicines.

    By • Nov. 14, 2022
  • The sign identifying the FDA headquarters in front of its building in White Oak, Maryland.
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    Sarah Silbiger via Getty Images
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    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
  • A photo of Sekar Kathiresan, CEO of Verve Therapeutics
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    Seth Babin/BioPharma Dive
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    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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    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
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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
  • The FDA logo on a glass pane at the agency's campus in Silver Spring, Maryland.
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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
  • A photo of a building showing a GSK sign in London.
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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
  • A rendering of Astellas' planned biotech campus in South San Francisco
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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
  • A photo of Carl June in his laboratory at the University of Pennsylvania
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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
  • The FDA logo on a glass pane at the agency's campus in Silver Spring, Maryland.
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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
  • Two researchers in safety goggles and lab coats work in a lab.
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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
  • An illustration of T cells attacking a cancer cell
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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
  • An illustration of red blood cells
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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