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511,583 Results for "novartis gene therapies".
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Approvals
Novartis Expands Spinal Muscular Atrophy Gene Therapy Market With FDA’s Itvisma Greenlight
The FDA approved an intrathecal form of Novartis’ spinal muscular atrophy gene therapy Zolgensma on Monday, broadening access to patients two years and older in what one Stanford Medicine professor called a “game changing advance” for the field.
November 25, 2025
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3 min read
·
Heather McKenzie
Sickle cell disease
Sickle Cell Gene Therapies Casgevy and Lyfgenia Still Lacking Traction 2 Years In
The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates.
February 23, 2026
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6 min read
·
Heather McKenzie
Gene therapy
In Vivo Is Having a Moment as Cell and Gene Therapy Sector Gathers in San Diego
A recurring theme Tuesday morning at Phacilitate’s Advanced Therapies Week was the quickly emerging potential of in vivo approaches to cell and gene therapy—a trend also reflected in recent investments by Eli Lilly and Regeneron.
February 11, 2026
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4 min read
·
Heather McKenzie
Complete response letters
REGENXBIO’s Hunter Syndrome Gene Therapy Fails To Win FDA Nod
The FDA recommended that REGENXBIO run a new study, treat more patients and include a placebo arm to support a resubmission for the gene therapy RGX-121.
February 10, 2026
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2 min read
·
Tristan Manalac
Gene therapy
Vertex, CRISPR Set Lofty Goal for Casgevy Gene Therapy as Patient Starts Ramp
Following over a year of slow uptake, Vertex Pharmaceuticals and CRISPR Therapeutics expect Casgevy revenues to nearly triple in 2026, as patient access to the sickle cell disease and beta thalassemia gene therapy grows.
February 13, 2026
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3 min read
·
Dan Samorodnitsky
Duchenne muscular dystrophy
Sarepta’s DMD Gene Therapy Staves Off Disease Three Years After Treatment
After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing.
January 26, 2026
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2 min read
·
Dan Samorodnitsky
Podcast
Weight Loss Drama Compounds on Heels of Novo, Lilly Earnings, Plus Gene Therapy News
A lawsuit and FDA warning ensued after Hims & Hers launched a compounded version of Novo Nordisk’s new obesity pill, more Big Pharma report earnings—including from weight loss rivals Novo and Eli Lilly—and the gene therapy space sees another rejection.
February 11, 2026
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1 min read
·
Jef Akst
Collaboration
Novartis Bets Up to $1.7B in Cardio-Focused Peptide Partnership
Novartis and Unnatural Products did not specify which disease targets they’re going after, only noting that the latter’s macrocyclic platform can generate potentially next-generation therapies that could apply to cardiovascular conditions.
February 19, 2026
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2 min read
·
Tristan Manalac
Opinion
Cell and Gene Therapy Will Transform Lives—If We Let It
Mixed headlines have plagued the cell and gene therapy space of late. We believe that a renewed case of optimism is not only warranted but essential if these therapies are to reach their full potential.
November 24, 2025
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8 min read
·
Tim Hunt
Rare diseases
FDA Greenlights First Gene Therapy for Rare Pediatric Disease
Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a non-profit sponsor to win FDA approval.
December 10, 2025
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1 min read
·
Tristan Manalac
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