Gene Therapy
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Courtesy of BioMarin Pharmaceutical
BioMarin tries to get hemophilia gene therapy back on track with positive data
Full one-year results from a Phase 3 study show the therapy, known as Roctavian, sharply reduced bleeds and use of blood-clotting treatments.
By Ned Pagliarulo • Jan. 11, 2021 -
Yujin Kim / BioPharma Dive
5 questions facing gene therapy in 2021
Gene therapy developers will need to address a series of setbacks that have shaded optimism in the sector's fast growth. Dealmaking, however, appears to be continuing apace.
By Ned Pagliarulo • Jan. 08, 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, reserachers and drugmakers still face major challenges.
By BioPharma Dive staff -
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Sarepta gene therapy misses goal in key muscular dystrophy study
Company executives, however, blamed "improbably bad luck" for study enrollment that resulted in more older patients with milder disease receiving placebo, and pledged to press on.
By Jonathan Gardner • Jan. 07, 2021 -
Pfizer beats Sarepta to start of first late-stage Duchenne gene therapy trial
Sarepta, though, may try to seek accelerated approval using Phase 2 data that's due imminently, along with results from another, smaller trial.
By Jonathan Gardner • Jan. 07, 2021 -
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Biogen pushes further into eye gene therapy with new deal
Partnering with German biotech ViGeneron bolsters Biogen's pipeline of drugs for eye diseases, which includes assets acquired through its buyout of Nightstar Therapeutics.
By Kristin Jensen • Jan. 06, 2021 -
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Cancer case puts UniQure's hemophilia gene therapy on hold, raising alarms
The FDA halted the AMT-061 program, which could be the first approved gene therapy for hemophilia B, because a patient who received the treatment appears to have developed liver cancer.
By Jacob Bell • Dec. 21, 2020 -
Lilly pays $880M for Prevail, pushing further into genetic medicine
The acquisition marks a significant bet by Lilly on gene therapy's potential to address tough-to-treat neurological disorders like Parkinson's disease.
By Jonathan Gardner • Dec. 15, 2020 -
UniQure offers a closer look at the leading hemophilia B gene therapy
Doctors on Tuesday got a more detailed idea of how the therapy works and who might be eligible to take it, as late-stage results were presented at ASH.
By Jacob Bell • Dec. 08, 2020 -
Bayer AG
Bayer strikes a deal with Atara, wading deeper into cell therapy
A pact for two Atara programs continues the German pharma's efforts to use dealmaking to build a gene and cell therapy division.
By Ben Fidler • Dec. 07, 2020 -
Jonathan Gardner / BioPharma Dive
4 storylines to track at the year's biggest meeting on blood diseases
A closely watched gene editing therapy, a slate of new multiple myeloma drugs and much more are set to highlight the American Society of Hematology conference this weekend.
By Ben Fidler , Ned Pagliarulo , Jacob Bell • Dec. 03, 2020 -
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 -
Getty / Edited by BioPharma Dive
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 -
Getty / Edited by BioPharma Dive
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
