Gene Therapy
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J&J pushes further into eye gene therapy
The pharma licensed a treatment for a severe type of age-related vision loss, building on past deals that gave it a foothold in gene therapy.
By Jacob Bell • Dec. 02, 2020 -
Regeneron
Regeneron, UPenn to use gene therapy tools to deliver a COVID-19 antibody drug
Working with University of Pennsylvania researcher Jim Wilson, Regeneron aims to develop a nasal spray version of the coronavirus treatment recently cleared by the Food and Drug Administration.
By Kristin Jensen • Dec. 01, 2020 -
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, reserachers and drugmakers still face major challenges.
By BioPharma Dive staff -
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Lilly dives into gene editing
Through a deal with Precision Biosciences, the pharma aims to develop therapies for genetic disorders, including Duchenne muscular dystrophy.
By Jacob Bell • Nov. 20, 2020 -
UniQure's gene therapy for hemophilia B meets goal in key study
The results are first to emerge from a late-stage test of a hemophilia B gene therapy, and show UniQure's treatment can restore clotting protein levels to mild or even near normal levels.
By Ned Pagliarulo • Nov. 19, 2020 -
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UCB strikes 2 gene therapy deals to build its pipeline
With patent expirations nearing for a top-selling drug, the Belgian pharma will buy one company and partner with another to add several early-stage projects in neurological diseases.
By Jonathan Gardner • Nov. 12, 2020 -
Courtesy of Intellia Therapeutics
Intellia, beginning treatment in CRISPR study, secures Gates Foundation backing
The biotech won a grant from the foundation two days after dosing the first patient in a study of an in vivo CRISPR medicine, a milestone for the company and the technology.
By Kristin Jensen • Nov. 11, 2020 -
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Pfizer invests in Homology, adding to gene therapy ambitions
Along with an equity stake, Pfizer will get right of first refusal on any deal involving Homology's most advanced therapies, which the biotech is developing for a rare metabolic disorder.
By Kristin Jensen • Nov. 10, 2020 -
FDA gene therapy holdups suggest closer scrutiny by agency
Requests for more data on manufacturing, clinical holds and a surprise rejection have led to questions of a tougher standard at the agency. Officials, however, point to a large and fast-advancing pipeline.
By Jonathan Gardner • Nov. 05, 2020 -
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Gene therapy for Duchenne muscular dystrophy: nearing final tests
Sarepta and Pfizer are advancing their experimental treatments into late-stage trials, while others are accelerating research in what's become one of gene therapy's most active fields.
By Ben Fidler • Nov. 05, 2020 -
Bluebird faces delay for sickle cell gene therapy as rivals inch closer
The setback for Bluebird's LentiGlobin comes amid a string of manufacturing-related delays for gene therapy developers.
By Kristin Jensen • Nov. 05, 2020 -
Axovant shares sink on manufacturing delay for Parkinson's gene therapy
A longer-than-expected timeline for developing a new manufacturing process means a key study for Axovant's top gene therapy likely won't begin until 2022.
By Ned Pagliarulo • Oct. 30, 2020 -
Novartis
Novartis eyes its next move in gene therapy
Acquiring Atlas Venture-backed startup Vedere Bio gives Novartis a technology it believes will be valuable for developing treatments for many causes of vision loss and blindness.
By Jacob Bell • Oct. 29, 2020 -
Bayer AG
Bayer to buy AskBio for $2B in bid to become a gene therapy player
Founded by gene therapy pioneer Jude Samulski, AskBio boasts a pipeline of experimental gene therapies as well as its own manufacturing capabilities.
By Ben Fidler • UPDATED: Oct. 26, 2020 at 11:55 a.m. -
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Ultragenyx expands into Duchenne gene therapy with Solid Bio deal
The rare disease drug developer is betting it can create a better DMD treatment by combining Solid's research with its manufacturing and gene delivery technology.
By Ned Pagliarulo • Oct. 23, 2020 -
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Sarepta, continuing its gene therapy push, helps launch a startup
The biotech has joined a group of investors to form a new gene therapy developer led by one of its longtime executives and backed with $107 million.
By Jacob Bell • Oct. 22, 2020 -
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Intellia cleared to start key CRISPR drug study in UK
The trial would be the first test of a systemically administered gene editing treatment that uses the Nobel Prize-winning technology to alter DNA.
By Ned Pagliarulo • Oct. 19, 2020 -
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Roche, aiming to make better gene therapies, turns to an emerging startup
The Swiss pharma is the third large drugmaker to partner with Harvard spinout Dyno Therapeutics, a small biotech aiming to improve gene therapy technology.
By Ben Fidler • Oct. 14, 2020 -
FDA puts Voyager study on hold in latest gene therapy speed bump
The biotech won't be able to start human testing of an experimental Huntington's disease treatment until regulators see more manufacturing data.
By Jonathan Gardner • Oct. 13, 2020 -
Pfizer and Sangamo, after rival's setback, kick off late-stage gene therapy trial
The FDA's recent rejection of a hemophilia gene therapy from BioMarin sets a high standard for approval. Will Pfizer fare any better?
By Jonathan Gardner • Oct. 07, 2020 -
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Solid gets all-clear from FDA to restart gene therapy trial
Worrisome immune responses had led the FDA to halt Solid's study. Now, the agency will permit Solid to continue after the biotech made adjustments to its manufacturing and patient enrollment procedures.
By Jonathan Gardner • Oct. 01, 2020 -
Romolo Tavani via Getty Images
Sponsored by BiomerePreclinical done right: The importance of using relevant animal models in gene therapy studies
Gene therapy is no longer an approach for the future. It's a technique used now.
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Permission granted by Emergent BioSolutions
Sponsored by Emergent BiosolutionsGene therapy solution: The value of a CDMO as your end-to-end partner
As gene therapy research continues to expand, innovators in this space will need CDMOs with highly specific expertise, facilities, and equipment.
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New data bolsters Sarepta gene therapies for two deadly muscle diseases
The results disclosed at a medical meeting on Monday show that all patients tested have seen their motor skills stabilize or improve when history suggests they wouldn't.
By Ben Fidler • Sept. 28, 2020 -
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Gene therapy company Taysha completes sprint from first funding to IPO
Led by alumni of Zolgensma developer AveXis, the university spinout could have four experimental therapies in the clinic by the end of 2021.
By Jonathan Gardner • UPDATED: Sept. 24, 2020 at 4:23 p.m. -
Permission granted by Takeda Pharmaceutical
Takeda adds to cell therapy push with new manufacturing site
The Japanese drugmaker has made cell therapy a focus, advancing three experimental cancer treatments into clinical testing with plans for two more.
By Ned Pagliarulo • Sept. 15, 2020
