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491,875 Results for "novartis gene therapies".
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Gene therapy
Vertex Abandons AAV as Gene Therapy Space Weathers Difficulties
The cell and gene therapy space in recent months has hit several speedbumps, including layoffs, dropped drugs and discontinued partnerships.
May 5, 2025
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2 min read
·
Tristan Manalac
Spinal muscular atrophy
Novartis’ Intrathecal Zolgensma Effective in Older Children
After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with an intrathecal formulation intended for older patients.
March 19, 2025
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6 min read
·
Heather McKenzie
FDA
Cell and Gene Therapy Sector Enters ‘Critical’ Period After Marks’ FDA Exit
As tariffs, HHS workforce cuts and the ouster of CBER Director Peter Marks threaten the “lifeblood” of the cell and gene therapy space, experts express wariness over the unknowns and optimism that Marks’ legacy will carry on.
April 28, 2025
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7 min read
·
Heather McKenzie
Gene therapy
CRISPR’s Casgevy on the Rise With More Gene Therapy Proof of Concept To Come in 2025
CRISPR Therapeutics’ partner Vertex reported that more than 65 treatment centers have been activated for the gene therapy Casgevy. While Vertex handles the market, CRISPR has been focused on its clinical program.
May 7, 2025
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4 min read
·
Annalee Armstrong
In partnership with GenScript
Beyond the Breakthrough: Industry Leaders Address Key Hurdles Facing Cell and Gene Therapies
At the GenScript Biotech Global Forum 2025, industry leaders celebrated CAR T cell therapy achievements while discussing ongoing challenges in manufacturing, distribution, treatment center capacity, and global payment structures for cell and gene therapies.
April 14, 2025
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6 min read
·
BioSpace Insights
Layoffs
Tenaya Slashes a Third of Workforce To Fund Development of Cardio Gene Therapies
The layoffs will take place throughout 2025 and will mostly affect Tenaya’s research and manufacturing operations. The company is continuing to test its hypertrophic cardiomyopathy gene therapy.
April 1, 2025
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2 min read
·
Tristan Manalac
IN PARTNERSHIP WITH CHARLES RIVER LABORATORIES
Cell and Gene Therapy Sector Sees 30% Investment Surge Despite Market Challenges
Industry updates and investment insights—including investor behavior and fundraising advice—were among the topics experts explored at Charles River Laboratories’ 2025 Cell & Gene Therapy Summit.
March 31, 2025
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5 min read
·
BioSpace Insights
Rare diseases
Novartis’ Fabhalta Approved as First Therapy for Rare Kidney Disease
This is the third indication for Fabhalta after Novartis won FDA approval of the small molecule in paroxysmal nocturnal hemoglobinuria and primary immunoglobulin A nephropathy.
March 21, 2025
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2 min read
·
Dan Samorodnitsky
Gene Therapy
Sarepta Shares Crash 22% After Patient on DMD Gene Therapy Elevidys Dies
After a patient taking the Duchenne muscular dystrophy gene therapy Elevydis died of liver injury, Sarepta will update the label to reflect the safety signal.
March 18, 2025
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2 min read
·
Tristan Manalac
Gene therapy
Vertex Severs Liver Gene Therapies Partnership With Verve
The companies were two years into a four-year, $400 million agreement aimed at developing and marketing gene therapies together.
February 27, 2025
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2 min read
·
Dan Samorodnitsky
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