Gene Therapy: Page 6

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    Switch Therapeutics launches with $52M to capitalize on RNA medicine ‘era’

    The company’s name comes from its goal of developing drugs that only “switch” on in target cells, potentially enabling more precise therapies.

    By • March 14, 2023
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    BioMarin, as expected, sees delay to FDA review of hemophilia gene therapy

    The agency is considering three-year follow-up data in its review of Roctavian, pushing back the potential U.S. arrival of BioMarin’s drug once again.

    By Kristin Jensen • March 7, 2023
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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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    Courtesy of Business Wire
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    Voyager licenses gene therapy tools to Novartis

    The Swiss drugmaker's decision is the latest in a string of partnership announcements for the gene therapy maker, which aims to bounce back from past research failures.

    By • March 6, 2023
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    FDA clears Intellia to start US tests of ‘in vivo’ gene editing drug

    The announcement, which follows regulatory setbacks for some of Intellia’s peers, is a “big win” for the gene editing field, according to one analyst.

    By • March 2, 2023
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    Amid industry turbulence, Chroma raises fresh funding to edit the epigenome

    As many drug startups struggle to gain cash, Chroma has raised another $135 million to back its research, a new twist on genetic medicine.

    By • Updated March 1, 2023
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    Sickle cell pipeline narrows as gene therapy developers rethink research plans

    Graphite Bio and Sangamo are stopping work on their respective sickle cell gene therapies, while Intellia revealed partner Novartis ended development of its genetic treatment for the blood disease.

    By • Feb. 23, 2023
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    Medicaid, with planned payment pilot, girds for influx of pricey gene therapies

    The proposed model could help state Medicaid agencies explore different kinds of outcomes-based payment schemes, but may come too late to prepare for the first sickle cell gene therapy.

    By • Feb. 21, 2023
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    Talaris restructures, ending effort to develop a cell therapy for kidney transplants

    Following a patient death and difficulty enrolling study participants, the company is laying off a third of its staff and ending a pair of studies for a two-decade-old program once owned by Novartis.

    By • Feb. 16, 2023
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    With Aera, CRISPR pioneer Feng Zhang goes after gene therapy’s delivery problem

    The company’s platform technology is built on research from Zhang’s lab at the Broad Institute, which focuses on proteins that could be used to package and deliver nucleic acids.

    By • Feb. 16, 2023
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    Astellas switches CEOs amid plans to pursue ‘aggressive’ growth

    Naoki Okamura, currently Astellas’ chief strategy officer, will replace current CEO Kenji Yasukawa as the company continues a strategic reboot that’s seen it invest more heavily in gene therapy.

    By • Feb. 6, 2023
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    Courtesy of Roche
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    Roche writes off $3B on lower sales forecasts for gene therapies, cancer drugs

    Among the drugs Roche recorded impairment charges on are four gene therapies acquired in the company's 2019 deal for Spark Therapeutics. 

    By • Feb. 3, 2023
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    Pixelimage via Getty Images
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    Magenta to explore sale, merger after study setback

    The biotech recently stopped a trial of its experimental conditioning regimen over safety concerns. Now it’s halting further development work as it undertakes a strategic review.

    By • Feb. 3, 2023
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    Head of FDA gene therapy office set to retire in March

    Wilson Bryan, a key decision maker in the agency’s oversight of genetic medicines, is expected to leave next month amid an organizational revamp of his office, the FDA confirmed.

    By Kristin Jensen • Feb. 1, 2023
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    UniQure nabs another gene therapy for ALS

    For $10 million upfront, UniQure has licensed rights to its second experimental treatment for the condition, a medicine from startup Apic Bio that should enter human testing later this year.

    By • Jan. 31, 2023
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    J&J, Legend’s cancer cell therapy has early success in key clinical trial

    The trial is an important test of Carvykti’s potential for earlier use in multiple myeloma and found treatment helped extend progression-free survival over standard regimens.

    By • Jan. 27, 2023
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    Magenta halts study of targeted conditioning drug after participant’s death

    The biotech said one patient died after receiving an experimental antibody drug designed to deplete certain blood and bone marrow cells before a transplant.

    By • Updated Jan. 26, 2023
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    Sales of J&J, Legend cell therapy plateau amid production challenges

    Carvykti, which was approved a year ago for hard-to-treat multiple myeloma, generated $55 million in fourth-quarter sales, on par with the previous quarter.

    By • Jan. 25, 2023
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    FDA lifts hold on Astellas gene therapy for Pompe disease

    The regulator’s decision ends a seven-month study pause that followed a report of one participant experiencing mild symptoms of peripheral neuropathy.

    By • Jan. 20, 2023
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    Courtesy of Business Wire
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    Q&A

    Al Sandrock on his short retirement and taking on a biotech turnaround project

    In a conversation at the J.P. Morgan Healthcare conference, the longtime Biogen executive discussed his hesitance to jump back into an executive role and why an opportunity to run Voyager Therapeutics drew him in.

    By • Jan. 17, 2023
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    Novartis
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    Gene therapy safety

    EMA weighs new safety guidance for Novartis’ gene therapy Zolgensma

    The discussions come months after two patients died from acute liver injury following treatment with the spinal muscular atrophy therapy.

    By • Jan. 13, 2023
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    Danielle Ternes/BioPharma Dive
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    Gene editing

    5 questions facing gene therapy in 2023

    Buoyed by recent approvals, the field faces a pivotal year that’s likely to bring new treatments as well as more challenges.

    By • Jan. 12, 2023
  • A sign for Pfizer is seen outside the Pfizer headquarters on November 9, 2020 in New York City.
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    Pfizer pares back early-stage research in rare disease, cancer

    The drugmaker said it is considering licensing out some assets to shorten development timelines, and will stop work on early viral-based gene therapies. 

    By • Jan. 6, 2023
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    Pfizer says hemophilia B gene therapy controlled bleeding in key study

    The Phase 3 results showed the one-time treatment outperformed standard factor replacement in adults with the bleeding disorder, but suggest it may be less potent than CSL and UniQure’s rival therapy. 

    By • Dec. 29, 2022
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    Ferring wins FDA approval for bladder cancer gene therapy

    The OK caps a long development journey for the treatment, which was previously turned back by the agency. Ferring doesn’t expect it to be available until the second half of next year, however.

    By • Dec. 19, 2022
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    Gene therapy safety

    FDA lifts hold on Bluebird’s sickle cell gene therapy

    Removal of the partial study suspension, which the agency imposed last December after a case of persistent anemia, helps clear the way for Bluebird to submit an approval application early next year. 

    By • Dec. 19, 2022