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503,524 Results for "sio gene therapies".
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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
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
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
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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
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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
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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
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2 min read
·
Tristan Manalac
Gene therapy
AviadoBio Looks to China for Eye-Based Gene Therapy in $413M UgeneX Pact
AviadoBio will have the option to exclusively license UGX-202, a vision-restoring gene therapy for the rare eye condition retinitis pigmentosa.
October 10, 2025
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2 min read
·
Tristan Manalac
FDA
FDA Details Thinking for Streamlining Development of Cell, Gene, Regenerative Therapies
Three draft recommendation documents published on Wednesday are intended to guide drug sponsors and accelerate the development of cell and gene therapies.
September 25, 2025
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2 min read
·
Tristan Manalac
Gene therapy
Capsida Reports Patient Death in Gene Therapy Trial
Capsida has yet to disclose the exact cause of death. The patient had received the gene therapy CAP-002 for a type of epilepsy.
September 11, 2025
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2 min read
·
Tristan Manalac
Deals
Lilly Dives Deeper Into Gene Therapy With up to $262M Adverum Buy
The cornerstone of the deal is Ixo-vec, an intravitreal gene therapy currently in Phase III development for wet age-related macular degeneration. Eli Lilly made another foray into genetic medicine in June, picking up Verve Therapeutics for up to $1.3 billion.
October 24, 2025
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2 min read
·
Heather McKenzie
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