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507,391 Results for "sio gene therapies".
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Press Releases
RenovoRx Announces Clinical Data Supporting the TAMP™ Therapy Platform will be Presented at the SIO 2026 Annual Scientific Meeting
February 5, 2026
·
10 min read
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
·
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
·
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
·
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
·
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
·
1 min read
·
Jef Akst
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
·
1 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
·
8 min read
·
Tim Hunt
Press Releases
Autonomix Medical to Present Final Analysis on Pain Mitigation in Pancreatic Cancer at SIO 2026 Annual Scientific Meeting
January 29, 2026
·
3 min read
Gene editing
Lilly Leans Into Hearing Loss in $1.12B+ Gene Editing Pact With Seamless
The deal will help bolster Eli Lilly’s growing hearing loss portfolio, which is anchored by the gene therapy AK-OTOF.
January 28, 2026
·
2 min read
·
Tristan Manalac
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