Gene Therapy
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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 -
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Getty / Edited by BioPharma Dive
TrendlineManufacturing Gene Therapy
Manufacturing is a major bottleneck in gene therapy, a challenge that large pharmaceutical companies, biotech startups and contract suppliers are all racing to meet.
By BioPharma Dive staff -
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 09, 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 08, 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 01, 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 at 5:55 p.m. -
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 -
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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 -
Novartis
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 -
Getty Images
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 07, 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 03, 2021 -
FatCamera via Getty Images
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.
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Getty / Edited by BioPharma Dive
UniQure moves Huntington's gene therapy to next phase of key trial
Safety monitors cleared the company to test a higher dose after reviewing data from 10 patients, providing some hopeful news after recent setbacks for experimental drugs targeting the genetic disease.
By Jonathan Gardner • May 27, 2021 -
Bluebird's next gene therapy gets backing from European regulator
The treatment for a progressive, often deadly brain disease could soon become Bluebird's third approved product and one of only a handful of marketed gene therapies in the world.
By Jacob Bell • May 21, 2021 -
In the midst of a 'strategic shift,' Voyager loses CEO and R&D head
The shake-ups come as the biotech prepares to invest more in its technology and also begin human testing for one of its most advanced gene therapies.
By Jacob Bell • May 20, 2021 -
Sarah Silbiger via Getty Images
FDA seeking more consistency from cell, gene therapy developers, top official says
Several drugmakers have recently faced development delays after the FDA's asked for more information on how they measure the potency of their products.
By Ned Pagliarulo • May 19, 2021 -
Courtesy of Sarepta
Sarepta's Duchenne gene therapy clears study hurdle, although questions linger
A version of the treatment made at commercial scale looks similar to what Sarepta used in early testing. But the findings are no longer the last step before an approval filing, as the company once hoped.
By Ben Fidler • May 18, 2021 -
National Institutes of Allergy and Infectious Diseases. (2016). "Human natural killer cell" [Micrograph]. Retrieved from Flickr.
Fate offers glimpse at 'natural killer' cell therapy for leukemia
The biotech's treatment is one of a wave of new treatments meant to mimic the effects of NK cell transplants in leukemia, though analysts remain skeptical.
By Ben Fidler • May 14, 2021 -
Permission granted by Biogen
Biogen gene therapy misses goal in eye disease study
The results are a setback for Biogen, which spent $800 million two years ago to acquire the treatment's developer, Nightstar Therapeutics.
By Ned Pagliarulo • May 14, 2021
