Gene Therapy: Page
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Seth Babin/BioPharma Dive
Gene editingVerve gets FDA green light to expand base editing trial into US
The company expects next quarter to read out initial data from its Heart-2 trial, which is testing a one-time treatment for people with genetically elevated cholesterol.
By Gwendolyn Wu • March 24, 2025 -
Courtesy of Novartis
Novartis builds case for new SMA gene therapy
To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as Zolgensma, could be approved for a broad range of spinal muscular atrophy patients.
By Jacob Bell • March 20, 2025 -
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 Prime Medicine
Gene editingPrime sets sights on liver, lung disease as next target for its gene editing tech
The biotech unveiled a new research program in alpha-1 antitrypsin deficiency, a disease that’s also a focus for rival Beam Therapeutics.
By Ned Pagliarulo • March 18, 2025 -
Jacob Bell/BioPharma Dive
Duchenne patient dies after receiving Sarepta gene therapy
A young man treated with Elevidys died of acute liver failure. The case may give doctors pause before prescribing the treatment in the future, some analysts wrote.
By Ben Fidler • Updated March 18, 2025 -
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MeiraGTx spins Parkinson’s, obesity gene therapies into AI startup
The joint venture with generative AI firm Hologen hands MeiraGTx $200 million up front as well as other financial perks — a “transformative” deal, according to the company’s CEO.
By Jacob Bell • March 13, 2025 -
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2seventy bio, Bluebird’s cell therapy spinout, sells to Bristol Myers for less than $300M
The deal ends 2Seventy’s short run as an independent company, during which it restructured, sold off research and lost nearly all of its market value.
By Ben Fidler • March 11, 2025 -
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Gene editingBeam base editing therapy gets ‘proof of concept’ in rare lung disease
While initial study results suggest Beam's technology can correct alpha-1 antitrypsin deficiency's genetic roots, shares fell by double digits.
By Ned Pagliarulo • March 10, 2025 -
Permission granted by MilliporeSigma
Sponsored by MilliporeSigma3 ways to accelerate development and de-risk cell and gene therapy manufacturing
Development strategies designed to reduce the unknowns so that cell and gene therapy innovators can develop robust processes efficiently.
March 10, 2025 -
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Regeneron gene therapy helps deaf children hear in small study
Ten of 11 children born with a rare form of congenital hearing loss experienced improvements after receiving the company’s treatment, new data show.
By Ned Pagliarulo • Feb. 25, 2025 -
Jacob Bell/BioPharma Dive
Pfizer stops selling hemophilia gene therapy, citing weak demand
Pfizer’s decision to halt further marketing of Beqvez is further sign of the sparse patient interest in gene therapies for the bleeding condition.
By Ned Pagliarulo • Feb. 21, 2025 -
Permission granted by Bluebird bio
Bluebird, at risk of default, agrees to take-private deal
The pioneering gene therapy developer will be acquired by investment firms Carlyle Group and SK Capital for a fraction of what it was once worth.
By Ben Fidler • Feb. 21, 2025 -
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Solid says early data suggest ‘differentiated’ Duchenne gene therapy
The company raised $200 million after early findings hinted its therapy could be more potent than others like it, including Sarepta Therapeutics’ Elevidys.
By Ben Fidler • Feb. 18, 2025 -
Permission granted by Cargo Therapeutics
Cargo to drop lead CAR-T therapy, lay off staff after study setback
Safety concerns and disappointing results led Cargo to stop testing the treatment, cut 50% of its workforce and evaluate strategic alternatives.
By Gwendolyn Wu • Jan. 30, 2025 -
iStockphoto.com/ZeynepKaya
Sponsored by PHC Corporation of North AmericaAre we overlooking something critical in cell and gene therapy research?
Taking control of O2 — ushering in the future of cell therapy research.
Jan. 27, 2025 -
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Regenxbio licenses gene therapies to Japan’s Nippon Shinyaku
Nippon Shinyaku will pay Regenxbio $110 million upfront for U.S. and Asia rights to treatments for Hunter and Hurler syndromes.
By Ned Pagliarulo • Jan. 14, 2025 -
Courtesy of Vertex Pharmaceuticals
Vertex, startup Orna to partner on gene editing research
The three-year partnership is another example of Vertex’s interest in improving on Casgevy, its CRISPR medicine for sickle cell and beta thalassemia.
By Delilah Alvarado • Jan. 7, 2025 -
iStockphoto.com/ZeynepKaya
Sponsored by PHC Corporation of North AmericaAre we overlooking something critical in cell and gene therapy research?
Taking control of O2 — ushering in the future of cell therapy research.
Dec. 16, 2024 -
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Chroma, Nvelop merge to marry genetic medicine ‘cargo’ to delivery
The combined company, which will pair Chroma’s epigenetic editing with Nvelop’s non-viral particles, has raised $75 million from a broad syndicate.
By Gwendolyn Wu , Ned Pagliarulo • Dec. 11, 2024 -
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Gene therapy uptake in sickle cell stays slow, despite patient interest
A lengthy treatment process, coupled with weighty risks for recipients to consider, has resulted in plodding adoption of Casgevy and Lyfgenia during their first year on market.
By Ned Pagliarulo , Gwendolyn Wu • Dec. 9, 2024 -
Permission granted by MilliporeSigma
Sponsored by MilliporeSigmaAre you using next-gen sequencing to inform AAV product and process quality? Here are 4 reasons you should
Next-generation sequencing allows for critical insights into gene therapy products, which can help streamline and accelerate everything from process development and production to regulatory approval.
Dec. 2, 2024 -
Permission granted by Intellia Therapeutics
Gene editingCRISPR therapy from Intellia may ameliorate rare heart disorder, data suggest
Phase 1 data indicate Intellia’s medicine could be a powerful treatment for a cardiac form of ATTR amyloidosis. But rival drugs are further ahead.
By Ned Pagliarulo • Nov. 18, 2024 -
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FDA endorses speedy approval path for Regenxbio Duchenne gene therapy
The agency's openness to a targeted pivotal study shows it’s still willing to consider accelerated clearance for Duchenne gene therapies despite questions about their effectiveness.
By Ben Fidler • Nov. 18, 2024 -
Christoph Burgstedt/Shutterfly
Sponsored by SyngeneReducing variability in gene expression: bottlenecks and solutions
Explore solutions to reduce variability in gene expression during cell line development.
Nov. 18, 2024 -
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PTC wins US approval of gene therapy for fatal enzyme disorder
Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review voucher for PTC.
By Kristin Jensen • Nov. 14, 2024 -
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Neurogene hits a setback in the clinic, days after fundraise
The company, which secured $200 million from investors in a private funding deal last week, learned Tuesday of a serious side effect experienced by one participant on a high dose of its Rett therapy.
By Ned Pagliarulo • Nov. 12, 2024
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