Gene Therapy: Page 2
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Sarepta, in shadow of FDA setback, marks gene therapy progress
The biotech released the first functional data from a trial of an experimental gene therapy treating a different type of muscular dystrophy than Exondys 51.
By Jonathan Gardner • Oct. 04, 2019 -
Gene therapy stocks have taken a beating. Their recovery may be slow
Share prices of at least 26 gene therapy biotechs are down from last October, a sign that investors may want more clarity on how the drugs will be sold.
By Jacob Bell • Oct. 03, 2019 -
Novartis to speed AveXis integration in wake of data manipulation
AveXis will be folded into Novartis' quality organization as a result of a scandal involving altered testing data for the company's gene therapy Zolgensma.
By Ned Pagliarulo • Sept. 24, 2019 -
Novartis, shadowed by data scandal, renews case for Zolgensma
Presymptomatic infants treated with the gene therapy reached motor milestones consistent with normal development, updated study results showed.
By Ned Pagliarulo • Sept. 19, 2019 -
Adverum sinks on first gene therapy data for eye disease
A decline in eyesight post-treatment reflects poorly on the therapy's potential, lengthening the company's odds of catching rival Regenxbio.
By Jonathan Gardner • Sept. 12, 2019 -
BioMarin talks down worries on gene therapy data differences
BioMarin's R&D head said timing of steroid treatment impacted interim Phase 3 data, which had spurred questions on valrox's durability.
By Andrew Dunn • Sept. 10, 2019 -
After scandal, Novartis pledges faster action on data integrity
CEO Vas Narasimhan committed to informing the FDA within five business days of any credible data integrity issues tied to a pending drug application.
By Ned Pagliarulo • Sept. 09, 2019 -
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. 05, 2019 -
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. 05, 2019 -
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. 04, 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. 03, 2019 -
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 -
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 at 12:43 p.m. -
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 -
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 at 3:18 p.m. -
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. 09, 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. 08, 2019 -
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. 08, 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 • UPDATED: Aug. 6, 2019 at 6:33 p.m. -
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. 01, 2019 -
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. 01, 2019 -
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 -
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
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