Gene Therapy: Page 4

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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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    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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    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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    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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    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
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    Dr Microbe via Getty Images
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    Sangamo presses ahead with Fabry disease gene therapy

    Preliminary results from a Phase 1 study show Sangamo’s treatment to be safe and suggest it is working as intended, leading the biotech to move into the trial’s next phase.

    By • Sept. 1, 2022
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    Beam details reasons for FDA hold on base editing cancer therapy

    The regulator has asked Beam for a range of technical data before it can start testing the blood cancer treatment in humans.

    By Kristin Jensen • Aug. 31, 2022
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    Courtesy of BioMarin Pharmaceutical
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    With European approval secured, BioMarin puts roughly $1.5M price tag on hemophilia gene therapy

    On a conference call, BioMarin executives revealed the anticipated price for Roctavian and the company's initial launch plans, which include pay-for-performance deals customized to different markets.

    By • Aug. 25, 2022
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    Dr_Microbe via Getty Images
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    ElevateBio finds a new manufacturing home and long-term gene therapy partner in Pittsburgh

    The biotech signed a 30-year deal with the University of Pittsburgh to establish a gene and cell therapy manufacturing hub that’s being built with a $100 million grant.  

    By Kristin Jensen • Aug. 25, 2022
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    iLexx via Getty Images
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    Ovid turns to gene therapy startup to restock drug pipeline

    The New York biotech will invest in and develop up to three drugs with Gensaic, an emerging startup aiming to use the viruses that infect bacteria to deliver genetic medicines.

    By • Aug. 24, 2022
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    With $2.8M gene therapy, Bluebird sets new bar for US drug pricing

    Approved for severe beta thalassemia, Zynteglo will test insurers’ willingness to pay for expensive one-time treatments. Its market launch is likely to be watched carefully by other gene therapy developers.

    By • Aug. 18, 2022
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    Sarah Silbiger via Getty Images
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    Bluebird gene therapy approved by FDA for rare blood disease

    The regulator cleared the biotech’s medicine Zynteglo for transfusion-dependent beta thalassemia, giving patients a powerful new treatment option. But it will come at a cost of $2.8 million in the U.S. 

    By • Updated Aug. 17, 2022