Gene Therapy
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Gene editingLilly 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 -
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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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 -
Danielle Ternes/BioPharma Dive
JPM26: Paying cash for obesity drugs, renewed IPO optimism and pharma’s cell therapy view
Drugmaker CEOs see online sales channels as a budding opportunity, while some biotech investors told BioPharma Dive to expect a rebound in new stock offerings.
By BioPharma Dive staff • Jan. 14, 2026 -
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Startup launchesAddition emerges with $100M to make gene therapies for chronic and rare diseases
Backed by a handful of prominent investors including the Gates Foundation, the startup is pursuing an approach it believes can sidestep issues seen with traditional gene therapy technology.
By Gwendolyn Wu • Dec. 17, 2025 -
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Biotech zombies‘Zombie’ biotech buyer Xoma to acquire Generation Bio
The gene therapy developer has lost most of its value since going public in 2020 and saw a long road ahead for a delivery technology that had shown promise in preclinical tests.
By Gwendolyn Wu • Dec. 16, 2025 -
Permission granted by Kyverna Therapeutics
Immune resetKyverna to seek first clearance of a CAR-T therapy for autoimmune disease
The company intends to file a U.S. application in the first half of 2026 following positive study results in a condition called stiff person syndrome that has no approved therapies.
By Ben Fidler • Dec. 15, 2025 -
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Hemophilia gene therapies are struggling on the market, even as innovation soars
The business case for hemophilia gene therapy still isn’t adding up due to persistent market barriers.
By Kelly Bilodeau • Dec. 5, 2025 -
Courtesy of UniQure
UniQure slides further on outlook for Huntington’s gene therapy
Finalized minutes from a late-October meeting with the FDA confirm the agency doesn't see the data collected so far as enough to support an approval filing.
By Jacob Bell • Dec. 4, 2025 -
Courtesy of Regeneron Pharmaceuticals
Gene editingRegeneron inks gene editing deal with startup Tessera
The big biotech, which has made genetic medicine a focus in recent years, is paying Tessera $150 million for rights to a potential one-and-done treatment for alpha-1 antitrypsin deficiency.
By Delilah Alvarado • Dec. 1, 2025 -
Permission granted by Novartis
Novartis wins approval to use SMA gene therapy in older patients
Itvisma, an intrathecal version of Zolgensma, can be used in children, teens and adults to stabilize or improve motor function.
By Jonathan Gardner • Nov. 25, 2025 -
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Sponsored by PearsonAPOE4 and CRISPR are reframing Alzheimer’s Risk
New research on APOE4 and CRISPR brings hope for slowing Alzheimer’s progression.
Nov. 24, 2025 -
Permission granted by MilliporeSigma
Sponsored by MilliporeSigmaSecuring quality in every dose: 5 essential capabilities for CGT developers to look for in a fill-finish CDMO
With their promise to transform disease trajectories, cell and gene therapies (CGTs) are bringing new hope to patients with an ever-expanding array of high-burden and chronic conditions as well as rare genetic disorders.
Nov. 17, 2025 -
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Gene editingKorro to cut staff, shift strategy as RNA editing drug misses mark in early testing
Korro’s lead program for alpha-1 antitrypsin deficiency wasn’t as potent as anticipated, leading the company to change course and lay off a third of its workforce.
By Kristin Jensen • Nov. 13, 2025 -
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Gene editingFDA unveils new regulatory roadmap for bespoke drug therapies
The “plausible mechanism” pathway, outlined by Martin Makary and Vinay Prasad, is designed to help accelerate treatments custom-made for individuals with rare and serious diseases.
By Ben Fidler • Updated Nov. 13, 2025 -
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Lilly picks up an eye gene therapy in deal with MeiraGTx
The alliance extends a deal streak in genetic medicine for Lilly and hands the company rights to an experimental treatment for an ultra-rare form of childhood blindness.
By Gwendolyn Wu • Nov. 10, 2025 -
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Caribou results suggest renewed promise for ‘off-the-shelf’ cancer cell therapy
While early, the data support Caribou’s plan to boost its therapies’ effectiveness by using donor cells that are carefully matched to a patient’s immune system.
By Jonathan Gardner • Nov. 3, 2025 -
Permission granted by Intellia Therapeutics
Gene editingFDA places formal hold on two Intellia CRISPR trials
Two days after Intellia voluntarily paused dosing due to safety concerns, the regulator stepped in with an official hold that makes a longer halt more likely.
By Delilah Alvarado • Oct. 30, 2025 -
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BioMarin, following sluggish sales, to offload hemophilia gene therapy
The company intends to divest Roctavian and “remove it from our portfolio,” CEO Alexander Hardy said, after nearly three years of slow uptake for a medicine once viewed as a future blockbuster.
By Ben Fidler • Oct. 27, 2025 -
Courtesy of Intellia Therapeutics
Gene editingIntellia pauses two CRISPR drug studies after safety scare
A study volunteer receiving Intellia’s transthyretin amyloidosis treatment was hospitalized due to severe signs of liver stress, prompting the company to pause enrollment and dosing while evaluating a new safety protocol.
By Jonathan Gardner • Oct. 27, 2025 -
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Adverum, hammered by gene therapy downturn, sells to Lilly in unusual deal
Lilly’s upfront offer for Adverum, the developer of a gene therapy for age-related macular degeneration, is less than the company’s previous closing share price.
By Jacob Bell • Oct. 24, 2025 -
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Regeneron, with ‘game-changing’ new data, to seek approval of hearing loss gene therapy
Study results published in The New England Journal of Medicine show the therapy significantly improved hearing in nearly a dozen children with a rare, genetic form of deafness.
By Jacob Bell • Oct. 12, 2025 -
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Lexeo says FDA open to speedier approval of rare disease gene therapy
The agency will consider a submission that includes pooled data from ongoing studies, a decision analysts viewed as a notable, additional sign of regulatory flexibility for gene therapies.
By Ben Fidler • Oct. 7, 2025
