Gene Therapy


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    Novartis
    Gene therapy safety

    Novartis reports deaths of two patients treated with Zolgensma gene therapy

    The deaths were due to acute liver injury, a known risk of Zolgensma and a concern for gene therapies like it.

    By Aug. 11, 2022
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    Permission granted by Bristol-Myers Squibb

    GentiBio partners with Bristol Myers as cell therapy for immune disease gains momentum

    The deal, worth as much as $2 billion, is the latest sign of industry interest in treatments that harness regulatory T cells, an approach behind several recent biotech startups.

    By Aug. 10, 2022
  • 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
  • 3D rendering of T cells attacking a cancer cell.
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    Meletios Verras via Getty Images

    Roche digs into off-the-shelf cell therapy with Poseida deal

    The pharma has now signed two alliances with allogeneic drug developers since September, signalling its interest in CAR-T treatment alternatives.

    By Aug. 3, 2022
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    JuSun via Getty Images
    CRISPR gene editing

    FDA halts testing of Beam’s base editing cancer therapy

    The regulator has put on hold human testing of a blood cancer treatment called BEAM-201, one of two the biotech aims to bring into clinical trials this year.

    By Aug. 1, 2022
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    Courtesy of Sarepta

    Sarepta to ask FDA for accelerated approval of Duchenne gene therapy

    After discussions with the FDA, the biotech aims to submit an application this fall — sooner than expected and ahead of a Phase 3 study that’s now ongoing.

    By July 29, 2022
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    Permission granted by Editas Medicine

    Editas treats first patient in sickle cell trial as FDA lifts partial hold

    The sickle cell therapy being studied is the second CRISPR-based medicine that Editas has advanced into clinical testing. Initial trial results could come as soon as the end of this year. 

    By July 28, 2022
  • A photograph of an infusion bag of Abecma, a CAR-T therapy from Bristol Myers Squibb and Bluebird bio
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    Courtesy of Bristol Myers Squibb and Bluebird bio

    Bristol Myers CAR-T sales rise despite production problems

    Demand has increased faster than Bristol Myers can make Abecma, although the company said supply is improving. A manufacturing issue for Breyanzi, meanwhile, impacted sales. 

    By July 27, 2022
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    libre de droit via Getty Images
    Emerging biotech

    Epigenetic editing: a tunable CRISPR alternative

    Three startups have recently emerged with plans to edit the epigenome rather than DNA directly. Here’s why that matters and what they aim to do. 

    By July 26, 2022
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    Janiecbros via Getty Images
    Sponsored by Bio-Rad

    Next-level quality control in cell and gene therapy

    Discover examples of how Droplet Digital™ PCR (ddPCR™) is enabling cell and gene therapies to reach further than ever before. 

    July 25, 2022
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    ismagilov via Getty Images

    Freeline gives detailed look at hemophilia gene therapy results

    The London-based drugmaker is currently seeking a partner to continue development of the hemophilia B treatment, which has shown promise in testing but trails a rival therapy from CSL Behring and UniQure.

    By July 21, 2022
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    DKosig via Getty Images

    Roche digs deeper into gene therapy for the eye

    The Swiss drugmaker, which already owns rights to a marketed gene therapy for inherited vision loss, will work with startup Avista Therapeutics to develop better delivery tools for the complex treatments. 

    By July 20, 2022
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    Image courtesy of Ultragenyx.

    Ultragenyx buys gene therapy partner after new study results

    The California biotech is exercising its option to acquire GeneTx BioTherapeutics for $75 million after seeing data from the companies’ study of an Angelman syndrome treatment.

    By July 19, 2022
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    Getty / Edited by BioPharma Dive

    Editas names new chief medical officer in latest executive change-up

    Baisong Mei, a veteran of Sanofi and Biogen, comes to Editas half a year after the gene editing company fired his predecessor and one month after new CEO Gilmore O'Neill started on the job.

    By Updated July 18, 2022
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    Seth Babin/BioPharma Dive
    CRISPR gene editing

    Verve starts first human test of gene editing treatment for heart disease

    A patient in New Zealand became the first person to receive the biotech’s medicine, which uses base editing to turn off a specific gene in the liver and thereby lower cholesterol.

    By July 12, 2022
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    milan2099 via Getty Images
    Sponsored by Ferring Pharmaceuticals

    C. difficile infection: Current treatment options and challenges

    Clostridioides difficile infection is a highly contagious disease that affects 500,000 people in the U.S. and results in nearly 30,000 deaths per year.

    By Dr. Teena Chopra • July 11, 2022
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    Permission granted by Catalent

    Adverum cuts jobs, restructures to give eye gene therapy another shot

    A year after reporting side effects “not seen before in ocular gene therapy” in a clinical trial, the biotech is eliminating 38% of its workforce to save cash while it runs a new study. 

    By July 7, 2022
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    Mario Tama via Getty Images

    J&J-backed gene therapy for the eye clears early study test

    Positive results for the treatment, which was developed by U.K. biotech MeiraGTx, could rebuild expectations after another gene therapy from Biogen failed in the retinal disease.

    By June 28, 2022
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    DKosig via Getty Images
    Gene therapy safety

    Another Astellas gene therapy trial paused by FDA after side effect report

    The regulator suspended testing of Astellas’ Pompe disease treatment after one patient experienced peripheral nerve damage. Both of the pharma’s clinical-stage gene therapies are now on hold.

    By June 27, 2022
  • Close up of orange micro-organisms, C. difficile infection
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    Kateryna Kon/Shutterstock.com

    Sponsored by Ferring Pharmaceuticals

    C. difficile infection: A close-up on an urgent public health threat

    As hospitals have dealt with surging admissions and extended stays with COVID-19, staff and patients alike have still had to contend with another potentially deadly infection from a bacterium called Clostridioides difficile.

    By Dr. Dennis Deruelle • June 27, 2022
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    Getty / Edited by BioPharma Dive

    BioMarin's hemophilia gene therapy recommended for approval in Europe

    The decision brings what could be the first approved hemophilia gene therapy, Roctavian, closer to market, after a series of regulatory setbacks that have delayed its arrival. 

    By June 24, 2022
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    libre de droit via Getty Images

    UniQure buoyed by early data for Huntington's gene therapy

    After one year, researchers detected important protein changes in patients who received a low dose of the experimental treatment. Further testing and functional data are needed to assess its potential, however.

    By June 23, 2022
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    Novartis

    Novartis hunting for sickle cell cure with Precision deal

    The pharma will pay $75 million to access Precision’s gene editing technology, adding another program to the lengthening list of drug development efforts targeting the inherited blood condition.

    By June 22, 2022
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    Getty / Edited by BioPharma Dive

    A biotech startup launches with $38M to develop a cystic fibrosis gene therapy

    Carbon Biosciences believes its technology, which is based on parvoviruses, could allow for larger gene delivery and repeat dosing.

    By June 21, 2022
  • Ogilvy Health - Medicine
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    Getty Images

    Sponsored by Ferring Pharmaceuticals

    Breaking down the role of the gut microbiome

    When it comes to the human body, we are never truly alone. In fact, we share our bodies with a diverse and complex colony of microorganisms, called the microbiome.

    By Dr. Carl Crawford (GI) • June 21, 2022
  • A 3D rendering of the CRISPR-cas9 gene editing system.
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    Meletios Verras via Getty Images
    CRISPR gene editing

    Vertex, CRISPR strengthen case for pioneering gene-editing treatment

    Positive data from 75 patients with either sickle cell disease or beta thalassemia keep the companies’ therapy on track to become the first CRISPR-based treatment submitted to drug regulators, possibly by the end of the year.

    By June 11, 2022