Gene Therapy: Page 14
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State of PlayEpigenetic 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 Ben Fidler • July 26, 2022 -
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Sponsored by Bio-RadNext-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 -
Explore the Trendline➔
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TrendlineGene 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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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 Ned Pagliarulo • July 21, 2022 -
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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 Jonathan Gardner • July 20, 2022 -
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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 Ned Pagliarulo • July 19, 2022 -
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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 Christopher Newman • Updated July 18, 2022 -
Seth Babin/BioPharma Dive
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 Ned Pagliarulo • July 12, 2022 -
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Sponsored by Ferring PharmaceuticalsC. 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 Ben Fidler • July 7, 2022 -
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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 Ned Pagliarulo • 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 Ben Fidler • June 27, 2022 -
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Sponsored by Ferring PharmaceuticalsC. 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 Jacob Bell • 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 Jonathan Gardner • June 23, 2022 -
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 Ned Pagliarulo • 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 Delilah Alvarado • June 21, 2022 -
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Sponsored by Ferring PharmaceuticalsBreaking 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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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 Jacob Bell • 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 Ned Pagliarulo • Updated June 10, 2022 -
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 Ben Fidler • June 10, 2022 -
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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 Ned Pagliarulo • Updated June 9, 2022 -
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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 Ned Pagliarulo • 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 Jacob Bell • 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 Ben Fidler • June 7, 2022 -
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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 Ned Pagliarulo • May 31, 2022
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