Gene Therapy: Page 18
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Sarah Silbiger via Getty Images
What to expect at the FDA's two-day meeting on gene therapy safety
A group of advisers convened by the agency began the two-day meeting Thursday with a discussion of cancer risk to AAV gene therapy.
By Ned Pagliarulo , Ben Fidler • Sept. 1, 2021 -
Getty / Edited by BioPharma Dive
Astellas again hits pause on gene therapy trial
The company is holding off dosing more patients with AT132, a potential treatment for a deadly neuromuscular disease, after one recently experienced a serious adverse event in the form of unusual liver function.
By Jacob Bell • Sept. 1, 2021 -
Explore the Trendline➔
iStock via Getty Images
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 -
Novartis falls behind rivals in race to bring CAR-T to early lymphoma
The company's cancer cell therapy Kymriah failed to top standard care in patients with an aggressive form of lymphoma, a setting in which treatments from Bristol Myers Squibb and Gilead recently succeeded.
By Ben Fidler • Aug. 24, 2021 -
National Institutes of Allergy and Infectious Diseases. (2016). "Human natural killer cell" [Micrograph]. Retrieved from Flickr.
With new Fate data, same promise, questions surround 'natural killer' cell therapy
Though early results from two of the biotech's experimental lymphoma treatments have shown promise, it’s unclear whether they can match the potency and durability of T cell therapies.
By Ben Fidler • Aug. 20, 2021 -
Mario Tama via Getty Images
Cell, gene therapy company funding reaches new heights, despite setbacks
Developers of the complex treatments raised $14 billion between January and June, nearly three-quarters of last year's record-setting total, according to numbers compiled by the Alliance for Regenerative Medicine.
By Ned Pagliarulo • Aug. 19, 2021 -
FDA clears Rocket to resume testing gene therapy for a rare heart disease
The decision from the regulator ends a three-month study pause that had stalled Rocket's development of the Danon disease treatment.
By Ben Fidler • Aug. 16, 2021 -
Bluebird to wind down business in Europe amid gene therapy struggles
Shares in the biotech sunk in value as the company also announced new safety concerns for one of its leading programs.
By Jacob Bell • Aug. 9, 2021 -
Jacob Bell/BioPharma Dive
Gilead's Kite bets on early-stage biotech to advance allogeneic cell therapies
Appia Bio, which launched with over $50 million in Series A funding earlier this year, will provide preclinical research for two CAR-iNKT candidates, which Kite will pick up should they advance past early clinical testing.
By Shoshana Dubnow • Aug. 5, 2021 -
Courtesy of Sarepta
Sarepta adds another gene therapy to its ever-expanding pipeline
The biotech company followed through on an option deal it struck two years ago with Nationwide Children's, licensing an experimental treatment for the most common form of limb-girdle muscular dystrophy.
By Ned Pagliarulo • Aug. 4, 2021 -
FDA allows Novartis gene therapy trials to resume after nearly 2 year pause
After reviewing animal study data submitted by the Swiss pharma, the FDA cleared spinal injections of Zolgensma for study in older patients with SMA.
By Jonathan Gardner • Aug. 3, 2021 -
Bluebird preps for split by selling manufacturing plant to well-funded startup
The startup manufacturing specialist National Resilience, which raised $800 million when it launched last year, will pay Bluebird $110 million for the North Carolina facility, bringing its North American network up to 10.
By Kristin Jensen • July 29, 2021 -
A startup raises $117M to deliver gene therapies in a new way
Flagship Pioneering, the biotech incubator that created Moderna, is helping fund Ring Therapeutics, a startup hoping to disrupt gene therapy development through the use of a different viral vector.
By Kristin Jensen • July 28, 2021 -
Getty / Edited by BioPharma Dive
A biotech's eye gene therapy faces longer odds as serious side effects pile up
Five trial participants with diabetic macular edema who received Adverum Biotechnologies' treatment experienced side effects never before reported for an eye gene therapy.
By Ben Fidler • July 23, 2021 -
Danielle Ternes/BioPharma Dive
Gene therapy for Fabry: early stages, promising results
Three companies are testing their gene therapies in early clinical trials, with initial results due later this year. Others are in preclinical stages and aim to follow soon.
By Shoshana Dubnow • July 22, 2021 -
Getty / Edited by BioPharma Dive
Bluebird, with little fanfare, is first to bring a second gene therapy to market
The approval of Skysona for a genetic brain disease is a milestone for one of gene therapy's pioneering companies, but isn't expected to turn Bluebird's financial fortunes around.
By Ben Fidler • July 21, 2021 -
Permission granted by Prime Medicine
Prime Medicine raises $315M to fuel 'search-and-replace' gene editing work
Formed around research from the Broad Institute's David Liu and Andrew Anzalone, Prime has attracted significant funding from a wide range of high-profile biotech backers.
By Shoshana Dubnow • July 14, 2021 -
Getty / Edited by BioPharma Dive
Bluebird resumes marketing gene therapy in Europe
Zynteglo sales have been on hold since February, when a patient in a clinical trial of another, related Bluebird medicine developed leukemia.
By Jacob Bell • July 9, 2021 -
Danielle Ternes/BioPharma Dive
Gene therapy for sickle cell disease: progress and competition
Treatments from Bluebird bio and CRISPR Therapeutics have shown considerable promise and could soon be nearing regulatory review. A full pipeline is growing behind them.
By Jacob Bell • July 8, 2021 -
The 'dovetail' partnership meant to turn Boston Children's into a hub for gene therapy startups
A five-year alliance with high-profile biotech ElevateBio is designed to get around a bottleneck that has held back the institution’s gene therapy work.
By Shoshana Dubnow • July 1, 2021 -
Getty / Edited by BioPharma Dive
Beam, Apellis partner in deal to expand gene editing's reach
The companies aim to develop therapies for complement-driven diseases, a specialty of Apellis and relatively new research ground for gene editing.
By Ned Pagliarulo • Updated June 30, 2021 -
del Aguila III, Ernesto. (2018). "CRISPR Cas9" [Illustration]. Retrieved from Flickr.
Deep DiveIntellia, with first results, delivers a 'landmark' for CRISPR gene editing
Results published in The New England Journal of Medicine offer the first clinical evidence that CRISPR gene editing inside the body can be safe and effective, a culmination of years of scientific research.
By Ben Fidler • June 26, 2021 -
Getty / Edited by BioPharma Dive
A year after getting UniQure's gene therapy, hemophilia patients are still doing better
New results from a key study show the treatment's benefit is holding up well over time. But the FDA wants to see more data, delaying when UniQure and partner CSL expect to file for approval.
By Jacob Bell • June 22, 2021 -
Long-term Zolgensma data backs up gene therapy's use in youngest patients
With more and more states screening for SMA at birth, the study results are important for assessing Zolgensma's benefits when given very early on.
By Jonathan Gardner • June 21, 2021 -
Biogen gene therapy deal has yet to bear fruit
Another gene therapy from Nightstar Therapeutics, which Biogen bought for $800 million in 2019, has failed in a key clinical study.
By Jacob Bell • June 15, 2021 -
Getty / Edited by BioPharma Dive
CRISPR, with new partner, to develop gene editing therapies for ALS, nerve disorder
A deal with biotech startup Capsida, which recently raised $140 million, gives CRISPR access to a Caltech technology that aims to improve the delivery of genetic medicines.
By Jonathan Gardner • June 15, 2021
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