Unblinded: 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

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

Explore the Trendline➔

Gene 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

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

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

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

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

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

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

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

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

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