Gene Therapy: Page 29
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Medicare proposes raising CAR-T pay, but reimbursement solution years away
As new technologies nip at CAR-T's heels, CMS could take three years to gin up a separate reimbursement category for Novartis' Kymriah and Gilead's Yescarta.
By Jonathan Gardner • April 24, 2019 -
Novartis chief defends cell, gene therapy push
CEO Vas Narasimhan has bet heavily on complex medicines, but the pharma's first quarter earnings show that plan is a work in progress.
By Ned Pagliarulo • April 24, 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 -
'Bubble boy' gene therapy results shoot Mustang Bio's stock up 200%
Following the clinical success, BioPharma Dive spoke with Mustang's CEO on the manufacturing hurdles still standing in the therapy's way.
By Andrew Dunn • April 18, 2019 -
'This is a cure.' St. Jude's gene therapy succeeds in 'bubble boy' disease study
Treatment has rebuilt the immune systems of 10 boys with the life-threatening condition, allowing them to live normal lives so far.
By Andrew Dunn • April 17, 2019 -
Sarepta may have seen off another rival in Wave
Toxicity observed with Wave's DMD drug raises questions over whether the biotech can find a safe and effective dose.
By Jonathan Gardner • April 17, 2019 -
With FDA decision near, Novartis bolsters SMA gene therapy case
Zolgensma could be approved by the FDA within weeks. Fresh clinical data give further evidence of the therapy's benefit, although reports of two deaths invite questions.
By Ned Pagliarulo • April 16, 2019 -
Catalent to buy Paragon for $1.2B as gene therapy demand grows
The deal follows close on the heels of Thermo Fisher's buy of Brammer Bio, a peer of Paragon in making the viral vectors needed for gene therapy.
By Jacob Bell • April 15, 2019 -
Audentes lays down marker in muscular dystrophy
In a challenge to Sarepta, Solid and Pfizer, a new gene therapy approach aims to deliver a more functional muscle-repairing protein to DMD patients.
By Jonathan Gardner • April 8, 2019 -
Yujin Kim/BioPharma Dive
What to look for after Zolgensma's launch
An ICER report provides clues on how payers will hedge their reimbursement bets for a likely million-dollar neuromuscular-disease treatment.
By Jonathan Gardner • April 4, 2019 -
FDA warns of potential violations at 21 stem cell clinics, manufacturers
Agency leaders said some stem cell companies are deceiving patients by "flouting the statutes and our regulations" and "falsely promoting their benefits."
By Kristin Jensen • April 4, 2019 -
Roche must ask Spark shareholders once again to sell
The Swiss pharma has extended its tender offer for its $4.8 billion buy of the gene therapy pioneer after failing to secure a majority of shares by a deadline.
By Jonathan Gardner • April 3, 2019 -
Sangamo hemophilia A progress boosts shares
Early data suggest Sangamo's gene replacement therapy could restore near-normal levels of a needed clotting factor. Competitors in the space still maintain a sizable lead, however.
By Ned Pagliarulo • April 2, 2019 -
AveXis buys its 4th US plant in preparation of Zolgensma launch
The six-building campus in Longmont, Colorado, gives the Swiss biotech nearly 700,000 square feet of additional space for biologic drug manufacturing.
By Jacob Bell • April 2, 2019 -
Bluebird gene therapy on cusp of European market entry
A nod from an EMA expert panel means Bluebird's LentiGlobin for beta-thalassemia has just one last hurdle before it's cleared.
By Jacob Bell • March 29, 2019 -
Q&A
Thermo Fisher's pharma head talks Brammer Bio and future M&A
Michel Lagarde, who leads Thermo Fisher's pharma services unit, explained to BioPharma Dive why the life science giant chose to enter gene therapy via a $1.7 billion deal for Brammer Bio.
By Andrew Dunn • March 28, 2019 -
Yujin Kim/BioPharma Dive
Bluebird opens cell and gene therapy plant in North Carolina
The site, which Bluebird bought in November 2017, will make the lentiviral vectors needed to produce the biotech's experimental treatments.
By Suzanne Elvidge • March 26, 2019 -
Yujin Kim/BioPharma Dive
Masthercell to build cell and gene therapy plant in Belgium
Expected to become operational in 2021, the new plant should triple the CDMO's manufacturing capacity.
By Andrew Dunn • March 26, 2019 -
Thermo Fisher to buy Brammer Bio for $1.7B, expanding gene therapy offerings
The life sciences giant grows its ambitions in the pharma CDMO space with another acquisition, this time focused on viral vector production.
By Andrew Dunn • March 25, 2019 -
Pfizer makes sure it won't get left out of the gene therapy deal boom
The big pharma now holds an exclusive option to acquire all shares of a privately held gene therapy biotech based in France.
By Jacob Bell • March 20, 2019 -
Gene therapy progress spurs competition in biotech buyouts
Nightstar Therapeutics, the latest gene therapy biotech bought, fielded interest from four drugmakers before agreeing to an $800 million offer from Biogen.
By Ned Pagliarulo • March 19, 2019 -
Kendall Davis/BioPharma Dive
Deep DiveUnblinded: Scott Gottlieb on biosimilars' precarious moment and the gene therapy boom
The outgoing FDA chief spoke with BioPharma Dive a day before his surprise resignation, detailing how drugmaker "shenanigans" have evolved and weighing in on gene therapy's inflection point.
By Andrew Dunn • March 12, 2019 -
Yujin Kim/BioPharma Dive
Axovant buoyed by early gene therapy data
After a damaging Alzheimer's setback, Axovant has bet its future on gene therapy. Its programs in Parkinson's and Tay-Sachs are its most advanced.
By Jacob Bell • March 11, 2019 -
Cellectis to bring CAR-T manufacturing in-house with new plants
The cell therapy biotech, best known for its allogeneic ambitions, currently relies on contract manufacturers for its clinical trial supply.
By Suzanne Elvidge • March 8, 2019 -
Biogen avoids heavy manufacturing lift in latest gene therapy deal
Nightstar's therapies target the eye, which means delivery of the DNA-based treatments is less onerous than it would be in other tissues, Biogen argues.
By Jacob Bell • March 5, 2019 -
Biogen buys Nightstar in search of more eye gene therapies
Shelling out roughly $800 million, the big biotech will get its hands on two mid- to late-stage treatments that it claims could launch in the next few years.
By Jacob Bell • March 4, 2019
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