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

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

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

Biotech of the Year: Spark Therapeutics

The biotech's gene therapy Luxturna, priced at $850,000 per patient, is testing a healthcare system not designed for transformative, one-time treatments.

By Jacob Bell • Dec. 3, 2018

ASH18: Bluebird's follow-on CAR-T finds initial success, but durability will be key test

Phase 1 data on a dozen patients hint that bb21217 may deliver the efficacy and persistence that Bluebird and partner Celgene have been vying for.

By Jacob Bell • Dec. 2, 2018

Gene-edited babies born in China, Chinese researcher claims

News of the achievement, which has yet to be verified, will likely spur substantial controversy over scientific ethics.

By Ned Pagliarulo • Updated Nov. 26, 2018

3 hemophilia patients lift uniQure's value more than $450M

After six weeks, hemophilia B patients who received the biotech's gene therapy had Factor IX levels thought to significantly reduce the risk of bleeding events.

By Jacob Bell • Nov. 15, 2018

Abeona inks deal with Regenxbio for gene therapy vector supply

Viral vectors are a key component of gene therapy development. Securing rights to Regenxbio's AAV9 vector could help accelerate Abeona's R&D work. 

By Suzanne Elvidge • Nov. 6, 2018

Lonza ups stake in Octane, furthering cell therapy bet

Octane's technology, which Lonza has collaborated on since 2015, is designed to manufacture cell therapies through a closed, automated system.

By Suzanne Elvidge • Nov. 1, 2018

Cryoport, in pursuit of smoother cell therapy logistics, opens new facility

Located in Livingston, New Jersey, the center is in close proximity to major cell therapy players like Novartis and Celgene.

By Jacob Bell • Oct. 24, 2018

Biogen talks threats to Spinraza, but mum on BAN2401

The spinal muscular atrophy drug was the big biotech's main growth driver in the quarter. Yet attention remains on Alzheimer's data due Thursday.

By Jacob Bell • Oct. 23, 2018

Sarepta builds out gene therapy manufacturing with latest CDMO deal

The biotech is thinking ahead, inking partnerships in recent months with Paragon Bioservices and Brammer Bio to support its gene therapy pipeline.​

By Suzanne Elvidge • Oct. 9, 2018

Gilead reaches deal for Yescarta coverage in England

For now, the CAR-T therapy will be provided through NHS' Cancer Drugs Fund, after a government agency judged it too pricey for routine coverage.

By Ned Pagliarulo • Oct. 5, 2018

Sarepta gene therapy for DMD takes another step forward

Updated results, including data from a fourth boy with Duchenne muscular dystrophy, help to firm up the prospects for the biotech's gene therapy.

By Ned Pagliarulo • Oct. 4, 2018