Gene Therapy: Page 27
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Brian Tucker/BioPharma Dive
ColumnPharmacquired: Vertex has an appetite for deals. Gene therapy is on the menu
CEO Jeffrey Leiden told BioPharma Dive that one or two gene therapy deals wouldn't be surprising as the biotech builds out what he calls its tool kit of technologies.
By Jacob Bell • Sept. 5, 2019 -
Getty / Edited by BioPharma Dive
Ultragenyx gene therapy data disappoints investors
The biotech said the current dose of DTX401 will be taken into Phase 3 testing, although a less potent dosage level showed similar efficacy.
By Jonathan Gardner • Sept. 5, 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 -
Novartis secures NICE nod for Luxturna with discount offer
The U.K. cost agency recommended Luxturna after reaching a deal with Novartis, which sells the Spark gene therapy outside the U.S.
By Ned Pagliarulo • Sept. 4, 2019 -
UniQure stays a step ahead in hemophilia B
Seeing high interest from patients and study investigators, uniQure is enrolling six more participants than planned in its Phase 3 gene therapy study.
By Ned Pagliarulo • Sept. 3, 2019 -
Jacob Bell/BioPharma Dive
Immatics shows Celgene isn't done stocking cell therapy pipeline
As its takeover by Bristol-Myers nears closing, Celgene is committing $75 million to research into T-cell redirecting technology.
By Jonathan Gardner • Aug. 28, 2019 -
Getty / Edited by BioPharma Dive
Gene editing companies oppose altering of embryos
Editing should be restricted to altering faulty genes in tissues, said Sangamo, Bluebird bio and 11 other biotechs in a statement of principles.
By Jonathan Gardner • Updated Aug. 27, 2019 -
Pfizer injects $500M more into gene therapy manufacturing
The money is going toward a North Carolina plant that will make the inactivated viruses used to deliver gene therapies and employ 300 staff.
By Jacob Bell • Aug. 21, 2019 -
Getty / Edited by BioPharma Dive
Sarepta now needs flawless progress for gene therapy
An unexpected rejection for the biotech's Vyondys 53 puts investor focus even more tightly on the biotech's earlier-stage muscular dystrophy pipeline.
By Jonathan Gardner • Aug. 20, 2019 -
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 -
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 -
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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 -
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 -
Getty / Edited by BioPharma Dive
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
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