Gene Therapy: Page 4
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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
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 -
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 -
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 -
MadamLead via Getty Images
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 -
Alexander Ford via Getty Images
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 -
Love Employee via Getty Images
Immune resetKyverna 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 -
Dr_Microbe via Getty Images
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 -
JuSun via Getty Images
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 -
Meletios Verras via Getty Images
First-of-its-kind cell therapy approved by FDA for rare soft tissue cancer
Adaptimmune’s Tecelra is the first TCR cell therapy to reach market and, at $727,000, is also the priciest cellular medicine for cancer in the U.S.
By Ben Fidler • Aug. 2, 2024 -
Courtesy of UniQure
UniQure restructuring to claim 300 jobs
The job cuts affect 65% of UniQure’s workforce and include the gene therapy developer’s recent sale of a manufacturing plant to Genezen.
By Delilah Alvarado • Aug. 1, 2024 -
Permission granted by University of Pennsylvania
Jim Wilson, prominent gene therapy researcher, to depart UPenn
Wilson, who founded UPenn’s gene therapy program three decades ago, will step down to start two new spinouts, Gemma Bio and Franklin Biolabs.
By Ned Pagliarulo • Aug. 1, 2024 -
Permission granted by AIRNA
Biotech startup Airna raises $60M for RNA editing medicines
Launched last September, the company is working on a treatment for alpha-1 antitrypsin deficiency, which it said could enter the clinic in 2025.
By Gwendolyn Wu • July 31, 2024 -
George Clerk via Getty Images
Pfizer quits Duchenne gene therapy, lays off staff following study setback
The company is letting go of 150 staffers alongside a decision to officially terminate the high-profile program, which was acquired in 2016.
By Ben Fidler • July 30, 2024 -
Michael M. Santiago via Getty Images
Pfizer says hemophilia gene therapy meets late-stage study goal
While the study results were positive, questions remain about the longer-term potential of hemophilia treatments like Pfizer’s.
By Kristin Jensen • July 24, 2024 -
Permission granted by Bluebird bio
US government declines to clear Bluebird fertility support for Zynteglo patients
The Health and Human Services' inspector general issued a "negative opinion" on Bluebird's request one week after Vertex sued the federal government over access to fertility preservation services.
By Ned Pagliarulo • July 22, 2024 -
Permission granted by InterVenn Biosciences
Sponsored by InterVenn BiosciencesBreakthroughs in immunotherapy: glycoproteins as predictive biomarkers for therapeutic response
Serum-based glycoproteins demonstrate success in predicting immune checkpoint inhibitor therapy outcomes.
July 22, 2024 -
Marcin Klapczynski via Getty Images
Immune resetArtiva prices $167M IPO, riding optimism for autoimmune cell therapy
The offering comes days after the publication of a paper showing what analysts said was early proof that “off-the-shelf” cell therapies can treat inflammatory diseases.
By Ben Fidler • July 18, 2024 -
Courtesy of Vertex Pharmaceuticals
Vertex taps Orum to hunt for better ‘preconditioning’ drugs
The deal gives Vertex rights to use Orum’s ADC-like technology to discover gentler preparatory regimens for patients receiving intensive treatments like Casgevy.
By Gwendolyn Wu • July 16, 2024 -
Courtesy of Vertex Pharmaceuticals
Vertex sues US over limits on providing fertility services to Casgevy patients
In the suit, Vertex argues HHS’ stance forces a “Hobson’s choice” on people with sickle cell seeking to undergo the potentially curative treatment.
By Ned Pagliarulo • Updated July 16, 2024 -
iStock via Getty Images
Lexeo gene therapy shows signs of heart benefit in small study
The biotech, which went public late last year, argued the results justify exploring an accelerated approval. But shares fell by double digits amid investor skepticism.
By Ben Fidler • July 15, 2024
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