Gene Therapy
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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 -
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 -
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 -
Jacob Bell/BioPharma Dive
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 -
Courtesy of Sarepta
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 Delilah Alvarado • 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 Ben Fidler • May 4, 2026 -
Permission granted by Intellia Therapeutics
Gene editingIntellia 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 Ben Fidler • 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 Jacob Bell • April 23, 2026 -
Permission granted by Roche
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 Ben Fidler • April 16, 2026 -
Permission granted by Rocket Pharmaceuticals
Rocket gene therapy cleared by FDA for rare immune disorder
Though the therapy, Kresladi, isn’t seen as a big seller, its clearance is a step forward for a company that’s lost most of its value since 2021 amid several setbacks.
By Ben Fidler • March 27, 2026 -
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China competitionAstraZeneca to boost cell therapy capabilities with new China investment
The planned Shanghai facility extends a yearslong push into the field and will support CAR-T therapies AstraZeneca is making for autoimmune disease and cancer.
By Ben Fidler • March 19, 2026 -
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News roundupSarepta tests new Elevidys safeguards; Sana advances diabetes cell therapy
Sarepta has begun evaluating a regimen meant to lower the risk of liver damage in Elevidys recipients. Elsewhere, Inovio laid off staff and a Bayer kidney drug notched another trial victory.
By Ben Fidler • March 16, 2026 -
Courtesy of UniQure
UniQure leads genetic medicine biotech rally after news of Prasad’s exit
Vinay Prasad contributed to an uncertain regulatory climate for gene therapy makers focused on rare conditions. Shares of many developers rose on the chance of a more industry-friendly successor.
By Jacob Bell • March 9, 2026 -
Permission granted by MilliporeSigma
Sponsored by MilliporeSigmaNavigating the PPQ process: Proven strategies to safeguard quality for cell and gene therapies
An often overlooked but critically important step along the path from bench to bedside, PPQ often results in delays and potential non-compliance issues without proper planning.
March 9, 2026 -
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Gene editingPrime to test FDA flexibility with 2-patient gene editing submission
The planned filing comes amid heightened scrutiny of the FDA’s rare disease stance and a year after Prime deprioritized the program for economic reasons.
By Kristin Jensen • March 4, 2026
