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506,714 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
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
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
Regulatory
FDA Imposes Boxed Warning, Narrows Patient Pool for Sarepta’s Duchenne Gene Therapy Elevidys
Sarepta must also run a post-marketing study for Elevidys to better assess the risk of serious liver injury in patients dosed with the gene therapy.
November 17, 2025
·
1 min read
·
Tristan Manalac
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
·
3 min read
·
Heather McKenzie
Earnings
BioMarin Abandons Gene Therapy Roctavian, Cuts Revenue Guidance as Voxzogo Faces Competitors
BioMarin no longer expects to hit its $4 billion revenue target by 2027, citing various market factors such as impending competition for achondroplasia therapy Voxzogo and the divestment of the gene therapy Roctavian.
October 28, 2025
·
4 min read
·
Tristan Manalac
Gene editing
FDA Eyes Accelerated Reviews for Gene Editing Therapies
Vinay Prasad, chief of the FDA’s Center for Biologics Evaluation and Research, is planning to publish a paper this month to outline his office’s thinking on accelerating gene editing reviews.
November 3, 2025
·
2 min read
·
Tristan Manalac
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