Gene Therapy
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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 -
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News roundupUltragenyx 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 -
Explore the Trendline➔
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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 -
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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 -
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VaccinesmRNA 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 -
Courtesy of Seattle Children's Hospital
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 -
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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 -
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Immune resetAutoimmune 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 -
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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 -
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Immune resetNovartis, 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 -
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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 -
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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 -
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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 -
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Q&A // Gene editingScribe’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 -
Jacob Bell/BioPharma Dive
VaccinesFDA 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 -
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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 -
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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 -
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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 -
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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 -
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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 -
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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 -
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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 -
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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 -
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Emerging biotechLilly, Ascidian link up in RNA exon editing pact
Worth as much as $1.9 billion, the deal directs Ascidian to help Lilly find potential drugs for genetically driven kidney diseases.
By Gwendolyn Wu • June 3, 2026 -
"Eli Lilly Corporate Center, Indianapolis, Indiana, USA" by Momoneymoproblemz is licensed under CC BY-SA 4.0
Gene editingEarly data for heart drug affirm Lilly’s billion-dollar bet on Verve
The base editing medicine lowered “bad” cholesterol levels along with a key protein, clearing the way for a Phase 2 study.
By Jonathan Gardner • May 26, 2026 -
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With new data, Regenxbio to seek FDA approval of Duchenne gene therapy
Though two serious side effects muddied the results, CEO Curran Simpson expressed optimism about a clearance and claimed FDA leadership will have a “mandate on rare disease flexibility.”
By Ben Fidler • May 14, 2026
