Gene Therapy: Page 28
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Pfizer muscular dystrophy gene therapy stumbles on safety
Study results presented Friday, which were the first significant look at Pfizer's DMD candidate, spurred shares in rival Sarepta to climb higher.
By Jonathan Gardner • June 28, 2019 -
Courtesy of Sanofi
Sanofi to cut 466 workers in Europe as it seeks to refocus R&D
The French big pharma is limiting investment in cardiovascular disease and diabetes, focusing instead on immuno-oncology, biologics and gene therapy.
By Jonathan Gardner • June 20, 2019 -
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 -
Getty / Edited by BioPharma Dive
Axovant signs Yposkesi as manufacturing partner
The small gene therapy developer, with no manufacturing capacity of its own, looks to Yposkesi for large-scale production.
By Kristin Jensen • June 20, 2019 -
UniQure shares hit record high after news report of potential sale
Wall Street analysts see the biotech benefiting from big pharma's interest in gene therapies.
By Andrew Dunn • June 17, 2019 -
Bluebird sets $1.8M price tag for blood-disease gene therapy
The European price excludes all offsets from health system costs and only measures gains in patient health, according to the biotech.
By Jonathan Gardner • June 14, 2019 -
Getty / Edited by BioPharma Dive
Gene therapy costs, manufacturing keeping CBER head 'up at night'
Lower costs and improved reproducibility would help advance the field, said FDA's Peter Marks in an interview with BioPharma Dive.
By Andrew Dunn • June 13, 2019 -
Vertex sets sights on DMD gene therapy, joining crowded field
Cash-rich deals with CRISPR Therapeutics and Exonics could make Vertex a challenger to companies already in the clinic with muscular dystrophy treatments.
By Ned Pagliarulo • June 7, 2019 -
Getty / Edited by BioPharma Dive
Deep DiveThe gene therapy era has arrived. So have the challenges.
With multiple gene therapies nearing market, the U.S. systems of reimbursement, regulation and research are being put to the test.
By Andrew Dunn • June 7, 2019 -
Fresh off being bought, Apceth preps to supply Bluebird's gene therapy
Apceth, now a part of Hitachi Chemical, will serve as the commercial manufacturer in Europe for Bluebird's just-approved gene therapy, Zynteglo.
By Jacob Bell • June 6, 2019 -
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Bluebird gene therapy approved in Europe in first for severe blood disorder
Zynteglo is intended as a one-time, lifelong treatment for patients with transfusion-dependent beta thalassemia.
By Ned Pagliarulo • June 3, 2019 -
Elizabeth Regan/BioPharma Dive
As ASCO begins, Iovance shows progress with cancer cell therapy
The biotech's therapy, which uses tumor-infiltrating lymphocytes, has spurred responses in some patients with advanced melanoma and cervical cancer.
By Andrew Dunn • May 31, 2019 -
Amicus widens UPenn gene therapy collaboration
The Galafold maker is looking beyond protein engineering as it tries to treat genetically driven metabolic disorders.
By Jonathan Gardner • May 29, 2019 -
Kendall Davis/BioPharma Dive
Iovance to invest $75M in cell therapy plant
A new site in Philadelphia, when complete, will be used to produce Iovance's tumor-infiltrating lymphocyte therapies.
By Kristin Jensen • May 29, 2019 -
BioMarin readies hemophilia gene therapy for FDA submission
But the debate over valrox's durability has not dissipated, as factor expression continued to decline in clinical trial patients.
By Jonathan Gardner • May 28, 2019 -
Novartis gene therapy approved, but will come at cost of more than $2M
Zolgensma is designed to be a cure for spinal muscular atrophy, but its steep price will pose difficulties for a system designed for chronic therapy rather than one-time treatments.
By Ned Pagliarulo • Updated May 24, 2019 -
Novartis looking earlier for deals as biotech valuations stay high
The Swiss pharma, which bought AveXis and its gene therapy Zolgensma last year, expects to spend about $10 billion a year on acquisitions.
By Ned Pagliarulo • May 23, 2019 -
Yujin Kim/BioPharma Dive
Could Novartis' gene therapy have more than one price?
Indication-based pricing may be possible for less severe patients, who would receive a smaller dose. Lower manufacturing costs would lessen the blow to Novartis too.
By Jonathan Gardner • May 23, 2019 -
UniQure snags patents on Huntington's gene therapy as it preps for the clinic
The biotech aims to start a Phase 1/2 study in the second half of 2019 for the experimental gene therapy.
By Andrew Dunn • May 22, 2019 -
Bayer pumping $150M more into biologics
A new facility will focus on large-molecule therapies for oncology, cardiology and additional specialty care areas.
By Jacob Bell • May 13, 2019 -
Nearing gene therapy launch, Bluebird cautions a slower start
Zynteglo, Bluebird's experimental therapy for beta-thalassemia, could soon be cleared in Europe. Reworking payment models to adjust will take time.
By Ned Pagliarulo • May 9, 2019 -
Roche risdiplam data heats up SMA rivalry with Novartis
The oral agent looks like it could give gene therapy Zolgensma a run for its money.
By Jonathan Gardner • May 7, 2019 -
Novartis makes final Zolgensma pitch before FDA decision
The most severe spinal muscular atrophy patients showed sustained benefit, the company said in a data update at a neurology meeting.
By Jonathan Gardner • May 6, 2019 -
Gilead's first move under new chief is to untether Kite
Kite is now its own unit within the big biotech, a move that newly minted CEO Daniel O'Day says will improve cell therapy development.
By Jacob Bell • May 3, 2019 -
Yujin Kim/BioPharma Dive
At gene therapy meeting, insurance execs grapple with expected cost
As more gene therapies move closer to market, questions around pricing and reimbursement have taken on greater urgency for regulators, payers and drugmakers.
By Ned Pagliarulo • April 30, 2019 -
Roche's takeout of Spark not working like a Swiss watch
Antitrust review drags on, forcing the two parties to withdraw and refile paperwork and extend the tender offer by another month.
By Jonathan Gardner • April 26, 2019
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