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

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

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

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

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

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

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

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