Gene Therapy
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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 -
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 -
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 -
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UniQure falls further on Makary comments
Remarks the FDA commissioner made during a CNBC appearance seemed to stoke investor fears that UniQure's gene therapy for Huntington's won't get approved.
By Jacob Bell • Feb. 27, 2026 -
Courtesy of Sarepta
Sarepta CEO Doug Ingram to retire, with company at a crossroads
Ingram, who steered the company through multiple drug approvals and controversies, will step aside as Sarepta faces dwindling Elevidys sales and emerging competitors to its core business.
By Ben Fidler • Feb. 26, 2026 -
Permission granted by Gilead Sciences
Gilead to buy Arcellx in $7.8B wager on multiple myeloma cell therapy
The acquisition bolsters Gilead’s sputtering cell therapy business with a medication the company expects to become a “foundational treatment” for the blood cancer, its CEO said.
By Ben Fidler • Feb. 23, 2026 -
Permission granted by Catalent
Sponsored by CatalentAll about cell therapy process characterization
Why process characterization is the key to consistent, safe, and scalable cell therapies.
Feb. 23, 2026 -
Permission granted by Catalent
Sponsored by CatalentSelecting the optimal cell therapy manufacturing platform
Navigating modular vs. integrated platforms—find the best path for your cell therapy program.
Feb. 17, 2026 -
Permission granted by Vertex
Vertex’s CRISPR therapy rebounds in latest earnings
Sales of Casgevy, a gene-edited treatment developed with CRISPR Therapeutics, more than tripled compared to the third quarter, a performance analysts saw as a bright spot in Vertex's report.
By Gwendolyn Wu • Feb. 13, 2026 -
Sarah Silbiger via Getty Images
FDA rejects Regenxbio treatment in another blow to gene therapy
The decision comes two weeks after the agency halted testing due to safety concerns and represents the latest regulatory setback for a gene therapy maker.
By Gwendolyn Wu • Feb. 10, 2026 -
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Immune resetLilly buys ‘in vivo’ CAR-T maker Orna, extending streak of genetic medicine deals
Worth up to $2.4 billion, the acquisition is also the latest in a series of buyouts involving startups with technologies that modify immune cells inside the body.
By Jonathan Gardner • Feb. 9, 2026 -
Permission granted by Catalent
Sponsored by CatalentNavigating the complexities of Cell and Gene Therapy supply chains
Turning complex CGT logistics into certainty—keeping therapies moving to patients.
Feb. 9, 2026 -
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Gene editing
Lilly bets on Seamless, delving further into genetic medicine for hearing loss
The potentially $1 billion alliance centered around a flexible gene editing technology adds to a portfolio of cutting-edge treatments the company is developing for hearing disorders.
By Delilah Alvarado • Jan. 28, 2026 -
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Regenxbio gene therapy trials suspended by FDA over safety worries
The development of a brain tumor in a study participant led regulators to suspend a Hurler syndrome therapy in early testing and a treatment for Hunter syndrome nearing an FDA decision.
By Kristin Jensen • Jan. 28, 2026 -
Kevin Frayer via Getty Images
China competitionChina’s edge in early-stage drugmaking ‘likely to persist,’ Pitchbook says
The ongoing surge in licensing deals for early drug prospects should continue this year and involve more cell and gene therapies, which have had trouble securing funding in the U.S., the firm said in a new report.
By Ben Fidler • Jan. 26, 2026 -
Jacob Bell/BioPharma Dive
Sarepta, battling slowing sales, claims Duchenne gene therapy’s impact grows with time
On a Monday conference call, Sarepta CEO Doug Ingram said new long-term data should help “rebalance the discussion” surrounding Elevidys.
By Jonathan Gardner • Jan. 26, 2026 -
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Oxford Biomedica confirms takeover talks with biotech investor EQT
Oxford, major contract manufacturer to cell and gene therapy companies, has already rejected multiple, unsolicited take-private bids from EQT that it felt “undervalued” the company and its prospects.
By Kristin Jensen • Jan. 15, 2026
