Gene Therapy

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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 reset

    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 • Sept. 2, 2026
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    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
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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 reset

    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 • 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 • 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 editing

    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 • Aug. 10, 2026
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    Vaccines

    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 • 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 • 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 • 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 • 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 • 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 • 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 • 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 biotech

    Lilly, 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 • June 3, 2026
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    Gene editing

    Early 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 • 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 • May 14, 2026
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    Sarepta tumbles as its gene therapy sales decline further

    Even though Elevidys beat Wall Street expectations, analysts expect investor focus to shift toward Sarepta's early-stage RNA drugs.

    By • May 7, 2026
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    With $97M, Latus pursues a different kind of Huntington’s gene therapy

    The therapy is part of a plan to develop gene therapies that can be efficiently delivered at lower doses, enabling them to be used against more than just “ultra-rare” conditions.

    By • May 4, 2026
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    Gene editing

    Intellia CRISPR drug succeeds in late-stage study against rare swelling disorder

    The findings position Intellia to bring to market the first “in vivo” gene editing medicine, though the therapy’s commercial potential remains the source of intense investor debate.

    By • April 27, 2026
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    FDA approves Regeneron’s hearing loss gene therapy

    Otarmeni, now cleared to treat a rare, inherited kind of hearing loss, is the first gene therapy cleared under the FDA’s “national priority” voucher program and will be offered to eligible patients at no cost.

    By • April 23, 2026
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    Roche to start new Elevidys study following setback in Europe

    The Swiss drugmaker, which owns rights to the Duchenne gene therapy outside the U.S., hopes the trial will yield additional approvals in Europe and elsewhere.

    By • April 16, 2026