Gene Therapy: Page 20
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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 -
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 -
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 -
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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 -
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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 -
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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 -
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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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Blood disease treatment from CRISPR, Vertex shows promise in more patients
The latest results from the companies' trials give support to hopes that CRISPR gene editing could functionally cure sickle cell disease and beta thalassemia.
By Ned Pagliarulo • June 11, 2021 -
Permission granted by Bristol-Myers Squibb
Bristol Myers says CAR-T therapy beat chemo, transplant in lymphoma study
The results could help support taking CAR-T therapy into earlier lines of cancer treatment, a long-held goal for the complex cellular drugs.
By Ned Pagliarulo • June 10, 2021 -
Sarah Silbiger via Getty Images
Bluebird cleared by FDA to resume studies of sickle cell gene therapy
An investigation by the company determined the treatment was "very unlikely" to be the cause of a case of leukemia reported in one of the trials.
By Ned Pagliarulo • June 7, 2021 -
Orchard abandons promising gene therapy for rare immune disorder
The U.K. biotech deprioritized the therapy last year, despite positive results in clinical testing that were recently published in a top medical journal.
By Kristin Jensen • June 3, 2021 -
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Sponsored by CatalentInnovation in cell and gene therapy: Insights from an industry specialist
Cell and gene therapies (CGTs) are poised to revolutionize the landscape of biologic drugs that treat diseases and offer hope in areas where there was previously none.
June 1, 2021
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