Beam sues 2 biotechs, alleging theft of base editing trade secrets

Beam claimed a former scientist stole confidential information and quietly spun up a rival rare disease treatment at YolTech Therapeutics. Serapha Bio, which licensed that therapy, denies the charges. 

By Gwendolyn Wu • Sept. 29, 2026

UniQure tumbles with latest data for Huntington’s gene therapy

Analysts guessed that investors wanted to see stronger durability results at a four-year check-in of the key trial supporting a potential approval of "AMT-130."

By Jacob Bell • Sept. 29, 2026

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, Ionis RNA drug scores in kidney disease study

The results position Roche to bring a new kind of medicine to market for IgA nephropathy, a disease that’s drawn considerable attention of late from drugmakers. 

By Jonathan Gardner • Sept. 23, 2026

Beacon eye gene therapy hits mark in late-stage study

Beacon claimed the findings represent the first time a treatment for X-linked retinitis pigmentosa, a rare and potentially blinding condition, met its main goal in a pivotal trial.

By Gwendolyn Wu • Sept. 21, 2026

Ultragenyx prices Sanfilippo gene therapy at $4M; Bristol Myers shelves Orum drug

Ultragenyx’s second gene therapy approval since August gives it another sellable voucher. Elsewhere, Roche declared success in a blood cancer trial and a cell therapy maker is cutting costs.

By BioPharma Dive staff • Sept. 18, 2026

Cellectis quits CAR-T and pivots to ‘in vivo’ gene editing

Long a frontrunner in donor-derived cell therapy research, the French biotech cited a shifting landscape in changing course and choosing to offload its lead programs.

By Jonathan Gardner • Sept. 14, 2026

mRNA cancer vaccines’ biggest barriers to market

Manufacturing hurdles are just one challenge the industry must overcome to commercialize personalized mRNA cancer vaccines.

By Alexandra Pecci • Sept. 11, 2026

BrainChild hauls in $116M to make cell therapies for childhood brain cancers

Spun out of research at Seattle Children’s Hospital, the startup has brought a CAR-T treatment for a rare and deadly pediatric cancer into mid-stage testing.

By Gwendolyn Wu • Sept. 8, 2026

The hidden scale-out problem behind CAR-T’s growth

CAR-T’s commercial challenge is making thousands of patient-specific manufacturing runs behave like one reliable system. "In vivo" production techniques could be one solution.

By Sy Mukherjee • Sept. 3, 2026

Autoimmune CAR-T faces tough questions after Novartis, Bristol Myers study halts

Wall Street analysts believe that a more cautious approach to manufacturing, study enrollment and side effect management could help developers avoid future safety hazards.

By Jonathan Gardner • Sept. 2, 2026

UniQure, after setbacks, seeks FDA approval of Huntington’s gene therapy

The company is now hoping for an affirmative answer on its application after an unusually turbulent year dealing with U.S. regulators.

By Kristin Jensen • Sept. 2, 2026

Novartis, Bristol Myers pause autoimmune CAR-T trials due to safety concerns

The deaths of three study participants led Novartis to halt development of rap-cel in multiple indications, while “transient” side effects pushed Bristol Myers to voluntarily pause its zola-cel program.

By Gwendolyn Wu • Updated Sept. 1, 2026

Safety concerns spur FDA to again halt testing of Regenxbio gene therapy

Investigators detected masses on the spines of five patients who received its Hunter syndrome treatment, leading to the program’s second clinical hold this year.

By Delilah Alvarado • Aug. 24, 2026

Ultragenyx, after setbacks, nabs first gene therapy approval

The clearance of Genglycos for a rare glycogen storage disease gives Ultragenyx a sellable voucher and momentum ahead of an eagerly anticipated study readout.

By Kristin Jensen • Aug. 20, 2026

PTC takes a chance on Sangamo’s Fabry disease gene therapy

Wall Street analysts view the deal as a risky, high-reward bet on a treatment that’s nearing a possible regulatory approval and could be superior to existing medications.

By Kristin Jensen • Aug. 13, 2026

Scribe’s Benjamin Oakes on a rare IPO and bringing CRISPR to the masses

The CEO spoke with BioPharma Dive about convincing investors to buy into the first gene editing IPO in more than two years. 

By Gwendolyn Wu • Aug. 10, 2026

FDA approves Moderna’s mRNA flu vaccine

The clearance paves the way for the first preventive flu shot based on mRNA technology and completes a dramatic regulatory turnaround.

By Delilah Alvarado • Aug. 6, 2026

Capricor Duchenne cell therapy voted down by FDA panel

At a contentious meeting with possible implications for other rare disease drugmakers, outside experts ultimately determined that Capricor didn't prove deramiocel's benefits in Duchenne-related cardiomyopathy.

By Ben Fidler • July 29, 2026

Sarepta, in search of a turnaround, taps ex-AbbVie exec Severino as CEO

Most recently the leader of buzzy startup Tessera Therapeutics, Severino will look to improve the fortunes of a company facing emerging competition and declining sales for its prized gene therapy Elevidys.

By Ben Fidler • July 27, 2026

Arrowhead hits ‘best-case scenario’ with latest results for fat-lowering drug

To analysts, the drug’s apparent safety and effects on both triglyceride levels and pancreatitis cases should make it competitive with a rival Ionis therapy.

By Jacob Bell • July 22, 2026

Top FDA gene and cell therapy regulator to step down

Following Vijay Kumar’s exit, acting CBER Director Karim Mikhail will also oversee a review office that has become a battleground for debates on FDA flexibility.

By Jonathan Gardner • June 30, 2026

Remix to go public in reverse merger with once high-flying Passage Bio

The transaction marks the latest turn in the fortunes of Passage Bio, one of the companies started by gene therapy pioneer Jim Wilson.

By Kristin Jensen • June 25, 2026

Regenxbio to resubmit gene therapy as FDA backtracks on another drug rejection

The agency took another step toward clearing a backlog of Makary-era rejections that appeared to contradict earlier agreements with drugmakers.

By Jonathan Gardner • June 22, 2026

UniQure to file gene therapy for approval, reflecting major shifts at FDA

The decision marks another regulatory U-turn following the exits of Marty Makary and Vinay Prasad, suggesting to some analysts that current FDA leadership may be more flexible in certain cases. 

By Jacob Bell • June 17, 2026

Sensorion, citing Regeneron competition, shifts focus to a different hearing loss therapy

A strategic review determined that the development environment had "notably changed," leading Sensorion to end work on its gene therapy for OTOF-related hearing loss.

By Kristin Jensen • June 11, 2026