Gene Therapy: Page 27
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Amicus gives early look at Batten disease gene therapy
"It's important to treat these children as soon as we can and to halt the progression of the disease," Amicus CEO John Crowley told BioPharma Dive.
By Andrew Dunn • Aug. 1, 2019 -
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Editas, Allergan launch first in vivo study of CRISPR-based medicine
The Phase 1/2 trial will test Editas' genome editing approach in a form of inherited blindness known as Leber congenital amaurosis.
By Ned Pagliarulo • July 25, 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 -
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Ultragenyx close to learning whether gene therapy gamble will pay off
Two therapies acquired in a 2017 buyout of Dimension are poised to enter late-stage testing, if results due soon look good.
By Jonathan Gardner • July 19, 2019 -
Catalent to help make Novartis' Zolgensma at Maryland site
The gene therapy is currently produced at an AveXis facility in Illinois, but Novartis is expanding its capacity in anticipation of growing demand.
By Ned Pagliarulo • July 18, 2019 -
Permission granted by Novartis
Novartis gives first glimpse at Zolgensma launch
Despite some signs of insurer pushback, the Swiss pharma indicated steady progress in rolling out the pricey gene therapy commercially.
By Ned Pagliarulo • July 18, 2019 -
Jacob Bell/BioPharma Dive
Kite building out manufacturing as next cell therapy inches to market
A new facility will span 67,000 square feet and be located in Oceanside, California, tucked into an existing Gilead site for biologics.
By Jacob Bell • July 16, 2019 -
Roche acknowledges the obvious, extends Spark buyout deadline
"Good things almost always take time," Roche CEO Severin Schwan wrote in a note to Spark employees.
By Jonathan Gardner • July 8, 2019 -
Sangamo backs up early success for hemophilia gene therapy
Updated results show the highest dose of Sangamo's gene therapy boosted blood-clotting protein levels for hemophilia A patients to normal levels.
By Andrew Dunn • July 5, 2019 -
Zolgensma set a new drug pricing bar. Insurers show some signs of pushback
One month after approval, a handful of payers have adopted policies taking a more cautious stance than what the FDA set out in the gene therapy's label.
By Ned Pagliarulo • Updated July 5, 2019 -
Bayer backs cell therapy startup with $215M in funding
Created by Versant Ventures, Century Therapeutics aims to develop allogeneic cancer therapies using pluripotent stem cells rather than donor T cells.
By Ned Pagliarulo • July 1, 2019 -
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 -
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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 -
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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 -
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Deep Dive
The 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
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