Gene Therapy: Page 30
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Yujin Kim/BioPharma Dive
In gene editing milestone, first patient treated in Vertex, CRISPR Therapeutics study
The drugmakers believe it to be the first ever company-sponsored use of a CRISPR-based therapeutic in a clinical trial.
By Ned Pagliarulo • Feb. 25, 2019 -
Roche acquires Spark in $4.8B gene therapy play
It's the latest evidence of pharma's growing appetite to expand in a field where Spark has been a leader with development of its approved treatment Luxturna.
By Jacob Bell • Feb. 25, 2019 -
Explore the Trendlineâž”
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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 -
Allogene inks lease for new cell therapy manufacturing facility
The 118,000-square-foot space should help the biotech be less reliant on its contract manufacturer and its partner Pfizer.
By Jacob Bell • Feb. 21, 2019 -
AveXis adds new jobs in gene therapy manufacturing expansion
More jobs and money are on the table for an AveXis plant in North Carolina, showing acquirer Novartis' interest in building out its gene therapy capabilities.
By Suzanne Elvidge • Feb. 21, 2019 -
Elizabeth Regan/BioPharma Dive
UniQure update bolsters outlook for hemophilia B gene therapy
While data came from only three patients, treatment with uniQure's experimental gene therapy led to Factor IX activity levels high enough to reduce bleeding risk.
By Suzanne Elvidge • Feb. 11, 2019 -
Solid's early DMD data comes up short, sinking shares
Preliminary biopsy data showed underwhelming results from the first three patients dosed. Company execs plan to carry on with higher dosing levels.
By Andrew Dunn • Feb. 7, 2019 -
J&J inks deal with MeiraGTx for retinal disease gene therapies
The early-stage deal is a sign of growing interest from pharma in advancing gene therapy development, analysts said.
By Suzanne Elvidge • Feb. 1, 2019 -
Novartis finds stability in Cosentyx amid company shake-ups
The drugmaker expects Cosentyx to grow further, providing a reliable boost as it deals with a large-scale restructuring and generic threats.
By Jacob Bell • Jan. 30, 2019 -
Voyager vaults higher on Neurocrine deal for Parkinson's gene therapy
Shares in Voyager rose by 40% after news Neurocrine will pay $165 million upfront in a collaboration centered on two experimental gene therapies.
By Andrew Dunn • Jan. 29, 2019 -
Elizabeth Regan/BioPharma Dive
7 biopharma trends to watch in 2019
Intensifying political pressure, surging cancer drug investment and the rise of pharma chief digital officers are among the factors which look set to shape the industry's next 12 months.
By Ned Pagliarulo • Jan. 17, 2019 -
FDA to bulk up cell and gene therapy staff, growing with the field
Two FDA leaders said the development of cell and gene therapies has reached "a turning point," leading to new agency plans to keep pace with research.
By Andrew Dunn • Jan. 16, 2019 -
Deep Dive
Hunting productivity, drugmakers pitch new 'pillars' of R&D
By some measures, returns on R&D have fallen to decade lows. Pharmas and biotechs, however, argue new technologies could open faster paths to market.
By Ned Pagliarulo • Jan. 11, 2019 -
Yujin Kim/BioPharma Dive
Q&A
Spark looks at scale, manufacturing as gene therapy matures
Though Luxturna only recently hit the market, Spark is already at work validating new ways of scaling up manufacturing.
By Jacob Bell • Jan. 10, 2019 -
Bluebird proposes installment plan for LentiGlobin gene therapy
The biotech company would receive further payments only if LentiGlobin keeps working, according to a payment idea the company rolled out Tuesday.
By Kristin Jensen • Jan. 9, 2019 -
Moderna's defense, biosimilar battles and other updates
Amid takeouts and readouts, the first two days of the J.P. Morgan Healthcare Conference featured drugmaker updates that figure to play a role in 2019.
By Ned Pagliarulo • Jan. 9, 2019 -
Novartis' cell therapy ambitions outpace early returns
Vas Narasimhan, the Swiss pharma's CEO, sees cell therapy's potential stretching beyond oncology, while acknowledging struggles with scaling manufacturing.
By Ned Pagliarulo • Jan. 7, 2019 -
Sarepta inks deal to bolster gene therapy manufacturing
An agreement with Aldevron may help the biotech avoid setbacks like the short-lived clinical hold it received on a gene therapy candidate last year.
By Suzanne Elvidge • Jan. 3, 2019 -
Merck KGaA trades rights for equity in amended CAR-T pact
New deal terms signal that German Merck has lost interest in developing Intrexon's technology in-house.
By Suzanne Elvidge • Dec. 21, 2018 -
Elizabeth Regan/BioPharma Dive
Gene therapy could be cost effective in SMA, but not at $4M
A report from ICER found Novartis' experimental Zolgensma could be more cost-effective than Biogen's Spinraza in treating spinal muscular atrophy.
By Ned Pagliarulo • Dec. 21, 2018 -
Q&A
Alnylam CEO talks politics, gene therapy and not becoming Pfizer
John Maraganore, who also serves as chair of BIO, spoke with BioPharma Dive in a wide-ranging interview on some of the industry's most pressing issues.
By Andrew Dunn • Dec. 14, 2018 -
Kendall Davis/BioPharma Dive
American leaders' distrust in science a 'dangerous situation,' BIO chair says
John Maraganore, the chair of biotech's largest trade group, said he's concerned the recent controversy over gene-edited babies could trigger knee-jerk reactions.
By Andrew Dunn • Dec. 14, 2018 -
Orchard to build gene therapy manufacturing plant in California
The planned investment comes months after Orchard struck a deal with GSK to take over development of the British pharma's gene therapy drugs.
By Kristin Jensen • Dec. 13, 2018 -
In CAR-T, manufacturing a hurdle Novartis has yet to clear
In an interview, Novartis' cell and gene therapy head said the pharma's made "significant progress" addressing issues that have hampered Kymriah's roll-out.
By Ned Pagliarulo • Dec. 6, 2018 -
Hospital docs weigh in on CAR-T reimbursement: 'It's very complicated'
Looming over clinical victories seen at ASH was a problematic question: How exactly will health systems pay for these powerful yet pricey therapies?
By Jacob Bell • Dec. 5, 2018 -
ASH18: Bluebird's gene therapy progress shadowed by MDS case
Changes made by the biotech to its Lentiglobin treatment look to be bearing fruit, but a new case of myelodysplasia syndrome has raised old concerns.
By Ned Pagliarulo • Dec. 4, 2018
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