Gene Therapy: Page 4
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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 -
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
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 -
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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 -
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Sickle 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 -
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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 -
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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 -
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Prime 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 -
Permission granted by Thermo Fisher Scientific
Sponsored by Thermo Fisher ScientificAccelerating precision oncology: The value of an end-to-end partner for therapeutic access
Learn how optimal NGS solutions can help developers overcome challenges throughout targeted therapeutic development and the value of an end-to-end partner to help every step of the way.
Sept. 30, 2024 -
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An Italian biotech gets $52M to advance pediatric gene therapies
The financing will support a technology designed to make delivery of lentiviral gene therapies, which are typically administered through a complex process, more convenient.
By Gwendolyn Wu • Sept. 25, 2024 -
Courtesy of Bristol Myers Squibb
Struggling 2Seventy scraps a key cancer study
The biotech expects that stopping enrollment for KarMMa-9, a trial evaluating early use of the multiple myeloma cell therapy Abecma, should save it $80 million in the near term.
By Jacob Bell • Sept. 25, 2024 -
Permission granted by Bluebird bio
Bluebird to lay off another 25% of workforce in latest restructuring
The job cuts are the latest in a series of steps Bluebird has taken to preserve cash and break even financially amid slow uptake of its marketed gene therapies.
By Delilah Alvarado • Sept. 24, 2024 -
istockphoto.com/Anusorn Nakdee
Sponsored by Scientist.comStreamlining IND applications for cell and gene therapy innovations
Regulatory and strategic challenges can delay the delivery of life-changing cell and gene therapies.
Sept. 23, 2024 -
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4D Molecular data ‘bode well’ for eye gene therapy, but shares fall
Shares dropped by as much as one-quarter Thursday after the biotech shared longer-term results for a gene therapy it’s developing for wet AMD.
By Ned Pagliarulo • Sept. 19, 2024 -
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Immune reset
Kyverna swaps CEOs, leaning on Kite veterans to lead ‘next chapter’
Warner Biddle and Christi Shaw, two prominent former executives at Gilead’s CAR-T division, will try to turn around an autoimmune cell therapy developer that’s lost most of its value since raising $319 million in a February IPO.
By Ben Fidler • Sept. 16, 2024 -
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Gene therapy startup emerges with green light for first-of-its-kind trial
Spun out of research at Nationwide Children’s Hospital, Vironexis claims it will be the first company to start a study testing a cancer drug delivered via the adeno-associated viruses commonly used in genetic medicines.
By Kristin Jensen • Sept. 12, 2024 -
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BridgeBio trims gene therapy budget after seeing data on adrenal gland medicine
Data from an early trial of the therapy fell short of the company’s “threshold to warrant additional capital investment,” an executive said.
By Ned Pagliarulo • Sept. 11, 2024 -
Courtesy of Sarepta
Sarepta reveals lower Elevidys sales, but points to ‘massive’ opportunity ahead
Company executives claimed “unprecedented demand” for the Duchenne gene therapy will soon cause a sales surge, countering a dip in quarterly sales and financial projections that raised concerns among investors.
By Ben Fidler • Aug. 8, 2024 -
Courtesy of BioMarin Pharmaceutical
BioMarin pares Roctavian spending as it narrows sales focus
The company plans to limit sales of the hemophilia gene therapy to the U.S., Italy and Germany, while ending most clinical development work.
By Ned Pagliarulo • Aug. 6, 2024
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