Gene Therapy: Page 2


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    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
  • 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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    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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    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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    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
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    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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    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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    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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    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
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    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
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    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
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    FDA advisers offer unanimous support to second Bluebird gene therapy

    By a 13-0 vote, the panel agreed the benefits of Bluebird’s beta thalassemia treatment outweigh its risks, one day after reaching a similar conclusion for another of the company’s drugs.

    By Updated June 10, 2022
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    del Aguila III, Ernesto. (2018). "CRISPR Cas9" [Illustration]. Retrieved from Flickr.

    Latest Caribou data add to 'off-the-shelf' cell therapy's durability questions

    Three of the six lymphoma patients who received Caribou’s gene editing treatment have relapsed in the latest sign that so-called allogeneic drugs could have trouble matching their CAR-T counterparts. 

    By June 10, 2022
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    Sarah Silbiger via Getty Images

    FDA panel backs Bluebird gene therapy despite safety risks

    In a major win for Bluebird, advisers to the agency voted 15-0 in support of its rare disease treatment eli-cel, downplaying concerns raised by FDA staff.

    By Updated June 9, 2022
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    Sarah Silbiger via Getty Images

    Bluebird's future in balance as FDA weighs gene therapy approvals

    Once among biotech’s most valuable companies, Bluebird is running out of cash. Its fate could rest on the FDA’s review of two rare disease treatments, which are being discussed at a two-day meeting that continues Friday. 

    By June 9, 2022
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    Astellas, despite recent setbacks, opens $100M gene therapy plant

    The opening of the plant, which could create more than 200 new jobs, comes on the heels of several significant issues with the company’s most advanced gene therapy program.

    By June 8, 2022
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    Immatics and Editas join up to bring CRISPR to 'gamma delta' cell therapy

    The two biotechs will use gene editing to make prospective treatments more potent, adding to a recent flurry of activity involving the fast-emerging form of cellular immunotherapy.

    By June 7, 2022
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    Sarah Silbiger via Getty Images

    BioMarin delays planned FDA filing for hemophilia gene therapy

    The California biotech now expects to resubmit its approval application to the FDA by the end of September, the latest regulatory setback for its closely watched treatment. 

    By May 31, 2022
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    FDA starts clock on review of CSL, UniQure gene therapy

    The regulator agreed to assess the companies' data under priority review, setting up a decision on approval of the hemophilia B treatment in six months.

    By May 25, 2022
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    PTC's first gene therapy gains European regulator's backing

    The brain-infused treatment for a rare neurological disease would be one of only three gene therapies commercially available in the EU. PTC plans to ask for FDA approval later this year.

    By May 20, 2022
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    Gene therapy safety

    Scientists home in on cause of Duchenne gene therapy side effect

    An unusual collaboration among gene therapy developers suggests certain mutations could be behind "peculiar" side effects experienced by several patients treated in clinical trials.

    By Updated May 18, 2022
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    Image courtesy of Ultragenyx.

    Ultragenyx strikes deal with struggling Abeona for rare disease gene therapy

    The treatment, which is designed to treat Sanfilippo syndrome, could become Ultragenyx's first gene therapy to reach market in the U.S., according to the biotech's CEO.

    By May 17, 2022
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    libre de droit via Getty Images

    Pfizer cleared to restart hemophilia gene therapy trial

    While a voluntary pause in dosing new patients will remain in place, the FDA's decision puts the study back on track to deliver data in 2023.

    By May 3, 2022