Gene Therapy
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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 -
Explore the Trendline➔
iStock via Getty Images
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 -
Dr_Microbe via Getty Images
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 -
Selvanegra via Getty Images
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 -
wildpixel via Getty Images
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 -
Jose Luis Calvo Martin, Jose Enrique Garcia-Maurino Muzquiz via Getty Images
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 -
Olena_T via Getty Images
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 -
Leonid Sorokin via Getty Images
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 -
Jacob Bell/BioPharma Dive
Sarepta scraps a Duchenne drug as gene therapy sales rise
Elevidys sales have increased since the FDA made a controversial choice to expand the therapy's use. Meanwhile, Sarepta is abandoning a successor to its drug Exondys 51, citing an “evolving" treatment landscape.
By Kristin Jensen • Nov. 7, 2024 -
Love Employee via Getty Images
Sana to lay off staff, deepen autoimmune focus in latest retrenchment
Just three years removed from a lucrative IPO, the company will cut jobs for a third time and invest more heavily in cell therapies for diabetes and lupus.
By Ben Fidler • Nov. 5, 2024 -
Dr_Microbe via Getty Images
Gene editingSickle cell patient dies in Beam study of base editing therapy
The death, which investigators linked to a preparatory chemo treatment rather than Beam's medicine, highlights the risks of using decades-old transplant drugs alongside cutting-edge CRISPR medicines.
By Ned Pagliarulo • Nov. 5, 2024 -
Maximusnd via Getty Images
Neurogene secures $200M ahead of anticipated study readout
The news led to a stock surge on expectations that forthcoming data for the company’s experimental gene therapy for Rett syndrome may turn out positive.
By Delilah Alvarado • Nov. 4, 2024 -
ShutterStock/Chepko Danil Vitalevich
Sponsored by Bio-RadEmpowering versatile applications of digital PCR with standardized, validated assays
Bio-Rad’s extensive ddPCR assay offerings help researchers leverage the full power of digital PCR.
Nov. 4, 2024 -
del Aguila III, Ernesto. (2018). "CRISPR Cas9" [Illustration]. Retrieved from Flickr.
Intellia data spark debate about CRISPR drug’s potential
Newly published data show the therapy reduced the rate of swelling attacks in people with hereditary angioedema, but didn't silence questions about its commercial outlook.
By Kristin Jensen • Oct. 24, 2024 -
Permission granted by Bluebird bio
New data underline cancer risk of Bluebird therapy for brain disease
Seven young boys given Bluebird's Skysona later developed blood cancers, findings that could shape how doctors balance the gene therapy’s risks against its benefit.
By Ned Pagliarulo • Updated Oct. 10, 2024 -
Jian Fan via Getty Images
Purespring raises $105M to advance gene therapy for kidney disease
The Series B round will fund a Phase 1/2 trial of the company’s experimental treatment for IgA nephropathy.
By Delilah Alvarado • Oct. 9, 2024 -
Permission granted by Basecamp Research
AI startup Basecamp allies with the Broad to dream up ‘programmable’ genetic medicines
The company, which is building a database of biological interactions found in nature, revealed $60 million in funding and a collaboration with David Liu’s lab.
By Gwendolyn Wu • Oct. 9, 2024 -
Permission granted by Intellia Therapeutics
Intellia starts late-stage test of CRISPR therapy for rare swelling disease
The study, of Intellia’s treatment for hereditary angioedema, is the second Phase 3 trial the biotech has begun testing its “in vivo” gene editing medicines.
By Ned Pagliarulo • Oct. 7, 2024 -
Courtesy of Editas Medicine
Editas trades Vertex fees for upfront cash in DRI deal
The gene editing company is selling to DRI Healthcare Trust future license fees that are owed to it under an agreement with Vertex last year.
By Delilah Alvarado • Oct. 4, 2024 -
JuSun via Getty Images
Gene editingPrime to narrow gene editing research as it strikes deal with Bristol Myers
The high-profile biotech is zeroing in on programs that serve as proof points for its technology, while seeking partners for other assets.
By Ben Fidler • Sept. 30, 2024
