Gene Therapy: Page 21
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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 -
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 -
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 -
Biogen looks to build better gene therapies through latest deal
The collaboration with Capsigen hands Biogen rights to a technology that could support the development of gene therapies for central nervous system and neuromuscular disorders.
By Jacob Bell • May 11, 2021 -
Sarah Silbiger via Getty Images
FDA unexpectedly grounds a gene therapy for a rare heart disease
Rocket Pharma's Danon disease treatment — key to the company's quiet rise over the past year — is the latest gene therapy to be put on hold by the agency. Executives predict only a short delay, however.
By Ben Fidler • May 11, 2021 -
National Institutes of Allergy and Infectious Diseases. (2016). "Human natural killer cell" [Micrograph]. Retrieved from Flickr.
Two biotechs team up to bring CRISPR to 'natural killer' cell therapy
A wide-ranging alliance between CRISPR Therapeutics and Nkarta is the latest sign of interest in a fast-emerging form of cancer immunotherapy.
By Ben Fidler • May 6, 2021 -
Permission granted by Eric Kelsic / Dyno Therapeutics
Dyno, in demand for its gene therapy work, raises $100M for fast expansion
After inking three pharma deals within a year of launching, the Harvard spinout has the backing of Andreessen Horowitz and several other top investors.
By Jonathan Gardner • May 6, 2021 -
Jacob Bell/BioPharma Dive
Avrobio changes course after FDA closes path for speedy gene therapy approval
The FDA recently converted its clearance for a standard Fabry treatment to a full approval, complicating Avrobio's plans to seek an accelerated OK for its rare disease therapy.
By Ned Pagliarulo • May 3, 2021 -
Mario Tama via Getty Images
BioMarin partners with Allen Institute to develop gene therapies for the brain
Far along in testing for a hemophilia treatment, BioMarin is expanding its gene therapy research into diseases of the central nervous system and aims to use some of the institute's technology.
By Ned Pagliarulo • April 28, 2021 -
Sarah Silbiger via Getty Images
FDA lifts hold on UniQure gene therapy study after review of cancer case
An investigation by UniQure determined the company's hemophilia gene therapy was "highly unlikely" to have caused a study volunteer's liver cancer, clearing the way for the FDA's green light.
By Jonathan Gardner • April 26, 2021 -
Getty / Edited by BioPharma Dive
Bluebird to withdraw gene therapy from Germany after dispute over price
The biotech will cut staff, primarily in Europe, after negotiations with German health authorities resulted in a price lower than Bluebird had sought.
By Ned Pagliarulo • April 20, 2021 -
Lilly, Arch join Deerfield in backing new gene therapy biotech Jaguar
Just two months after launching, Jaguar Gene Therapy has raised another $139 million for a preclinical pipeline aimed at gene-linked autism and diabetes.
By Ned Pagliarulo • Updated April 13, 2021 -
Courtesy of Bristol Myers Squibb
New CAR-T therapy from Bristol, Bluebird effective but too costly, ICER finds
The influential drug cost watchdog recommended a discount of between 37% and 54% to the $419,500 list price the companies charge for Abecma, a multiple myeloma cell therapy cleared by the FDA in March.
By Ned Pagliarulo • Updated April 6, 2021 -
Getty / Edited by BioPharma Dive
UniQure says hemophilia gene therapy likely not cause of study volunteer's cancer
The determination, made by UniQure and an independent lab, could ease safety concerns spurred by the patient's diagnosis in December.
By Ned Pagliarulo • March 29, 2021 -
Drugmakers flock to Vineti's fix for a cell, gene therapy problem
A group of drug companies, including Novartis, Takeda and Roche's Genentech, have agreed to collaborate with Vineti to usher in new identification standards for complex medicines.
By Jacob Bell • March 24, 2021 -
Yujin Kim/BioPharma Dive
With new results, Sarepta's 2nd gene therapy holds steady
Stung by a clinical trial miss in Duchenne muscular dystrophy, the biotech now has promising, if early, data to back a gene therapy for a different rare neuromuscular disease.
By Jonathan Gardner • March 19, 2021 -
Getty / Edited by BioPharma Dive
Record funding flowed into cell, gene therapy companies last year
Nearly $20 billion was invested in biotechs developing cell-, gene- and tissue-based treatments in 2020, according to a new report from industry group ARM.
By Ned Pagliarulo • March 16, 2021 -
Getty / Edited by BioPharma Dive
Solid Bio, eyeing comeback, points to 'totality' of new Duchenne gene therapy results
The data keep Solid in the running in one of gene therapy's most competitive races, but still don't make clear whether the treatment is changing the trajectory of the disease.
By Ben Fidler • March 16, 2021 -
Permission granted by Rocket Pharmaceuticals
How Rocket Pharma quietly became one of gene therapy's high flyers
An unconventional strategy is behind the rise of Rocket, a New Jersey biotech whose executives boldly talk of turning into the "Genentech of gene therapy."
By Sarah de Crescenzo • March 11, 2021 -
Bluebird says gene therapy 'very unlikely' cause of cancer case in trial
The biotech is discussing its findings with U.S. and European regulators in hopes of resuming study of the treatment as well as sales of a related product.
By Ned Pagliarulo • March 10, 2021 -
Permission granted by Biogen
Biogen to spend $200M on gene therapy plant in North Carolina
The planned factory is a strong sign of Biogen's interest in gene therapy, an area of research it has invested in through dealmaking over the past few years.
By Kristin Jensen • March 4, 2021
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