Gene Therapy: Page 26
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Novartis replaced AveXis chief scientist, R&D head amid Zolgensma uproar
Neither Brian Kaspar nor his brother Allan has been involved in AveXis operations since early May, when the Swiss pharma says it confirmed inaccuracies in the gene therapy's data.
By Ned Pagliarulo • Updated Aug. 14, 2019 -
Lawmakers up pressure on Novartis in wake of data manipulation
Republican Senator Chuck Grassley demanded the Swiss pharma give a full account of its handling of faulty data used in winning approval for the gene therapy Zolgensma.
By Ned Pagliarulo • Aug. 13, 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 -
In rebuke of Novartis, FDA making 'statement for the world'
"This is a rapidly evolving field where there are going to be accelerated approvals," former FDA chief Robert Califf told BioPharma Dive. "The quality of the data is critical."
By Ned Pagliarulo • Aug. 9, 2019 -
Hospitalized gene therapy patient triggers Sarepta sell off
An erroneous report of rhabdomyolysis in a patient enrolled in a Duchenne trial was the latest safety worry to send shares in a gene therapy biotech spiraling.
By Jonathan Gardner • Aug. 8, 2019 -
Courtesy of Gilead Sciences
Medicare boosts CAR-T cancer therapies with expanded coverage
A long-awaited decision from CMS marks a step forward in addressing reimbursement hurdles for the expensive drugs.
By Ned Pagliarulo • Aug. 8, 2019 -
Novartis used manipulated data in winning Zolgensma approval, FDA says
AveXis, the Novartis unit which developed Zolgensma, knew of the data issue more than two months before the therapy's landmark OK, the FDA said.
By Ned Pagliarulo • Aug. 6, 2019 -
Regenxbio inks viral vector rights deal with Pfizer
Pfizer gains non-exclusive rights to AAV9 vector technology from Regenxbio, a collaboration that supports one of the pharma's early gene therapy programs.
By Kristin Jensen • Aug. 1, 2019 -
Getty / Edited by BioPharma Dive
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 -
Getty / Edited by BioPharma Dive
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 -
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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 -
Getty / Edited by BioPharma Dive
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
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