In the past three years, gene therapy has reshaped what's possible in hemophilia treatment for patients 18 and older. But a key question remains: How soon will these advances reach children? At the ...
Wall Street Journal bestselling author, Italian-American singer-songwriter, and gene therapy entrepreneur Patrick Girondi will appear at the Oxford Exchange Book Fair in Tampa on , from ...
If approved, UX111 will be the first approved therapy for the treatment of Sanfilippo syndrome Type A, a rare disease affecting young children that leads to progressive, irreversible neurodegeneration ...
Seeking Alpha: 4D Molecular Therapeutics: This Gene Therapy Company May Soon Surprise Investors
Lowering cholesterol is one of the most effective ways to reduce your risk of heart disease, and it may soon be possible to get a one-and-done gene therapy to keep cholesterol and triglyceride levels ...
KUNC: Gene therapy for a rare type of deafness shows lasting results
Researchers say a gene therapy allowed deaf children and adults as old as 32 to hear for the first time. The benefits have persisted for more than two years for some patients.
Fierce Biotech: Eying 3 approvals in 2 years, Ocugen’s CEO wants to bring gene therapy to the masses
To Ocugen CEO Shankar Musunuri, Ph.D., most gene therapy biotechs today lack vision. | Many gene therapy outfits focus on technical advances, Ocugen CEO Shankar Musunuri, Ph.D., said, meaning they ...
Eying 3 approvals in 2 years, Ocugen’s CEO wants to bring gene therapy to the masses
Yahoo: Doctors awarded science ‘Oscar’ for curing inherited blindness with gene therapy
Three scientists have won the 2026 Breakthrough Prize for developing Luxturna — the world's first FDA-approved gene therapy for inherited blindness.
The Philadelphia Inquirer on MSN: Philly scientists win 2026 Breakthrough Prize for developing gene therapy for blindness
Three Philadelphia scientists won a $3 million Breakthrough Prize for developing the first FDA-approved gene therapy for a genetic disease, prize sponsors announced Saturday. Their work created a ...
Philly scientists win 2026 Breakthrough Prize for developing gene therapy for blindness
Labiotech.eu: The future of cell & gene therapy: Key trends to watch
Cell and gene therapies are moving towards correcting root causes of diseases. Let's take a look at future cell and gene therapy trends.
Detroit Free Press: Author, Gene Therapy Pioneer, and Film Producer Patrick Girondi at Oxford Exchange Author Fair in Tampa on April 23 ’26
Author, Gene Therapy Pioneer, and Film Producer Patrick Girondi at Oxford Exchange Author Fair in Tampa on April 23 ’26
Yahoo Finance: Ultragenyx Announces U.S. FDA Acceptance of BLA Resubmission for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)
Ultragenyx Announces U.S. FDA Acceptance of BLA Resubmission for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)
Key takeawaysResults from a UCLA-led clinical trial for sickle cell disease reveal the unique challenges of treating blood disorders with gene therapy ...
FierceBiotech: Regenxbio posts clean safety profile for DMD gene therapy as pivotal data loom
Regenxbio has guided its Duchenne muscular dystrophy (DMD) gene therapy candidate through another test, reporting a clean safety profile and functional improvements as it heads toward pivotal data.
Regenxbio posts clean safety profile for DMD gene therapy as pivotal data loom
NPR: Gene therapy for a rare type of deafness shows lasting results
An experimental gene therapy appears safe and highly effective for restoring hearing to people born with a rare form of deafness, researchers reported Wednesday. The study, the largest and longest to ...
News Medical: Advances in hemophilia gene therapy bring hope for pediatric patients
Seeking Alpha: Solid Biosciences: SGT-003's Safety Profile Illuminates DMD Gene Therapy Path (Rating Upgrade)
Solid Biosciences is approaching key catalysts in DMD gene therapy, with SGT-003 showing promising early safety and efficacy signals. The evolving DMD landscape, especially safety concerns with ...
Solid Biosciences: SGT-003's Safety Profile Illuminates DMD Gene Therapy Path (Rating Upgrade)
4D Molecular Therapeutics trades below its cash balance, offering significant upside as its late-stage gene therapy programs advance. FDMT's lead asset, 4D-150, targets wet AMD and DME, with Phase 3 ...
MSN: Biotech Breakthrough: Latus Bio Doubles Down on AI to Revolutionize Gene Therapy
Biotech Breakthrough: Latus Bio Doubles Down on AI to Revolutionize Gene Therapy
FiercePharma: FDA restricts bluebird bio gene therapy Skysona after blood cancer reports
After telegraphing an investigation in November, the FDA has moved to restrict the use of bluebird bio’s gene therapy Skysona in certain patients. The FDA has updated Skysona’s indication, allowing it ...
MSN: First-ever experimental gene therapy seeks to restore vision by rejuvenating eye neurons
Life Biosciences is pushing cellular rejuvenation into the clinic with ER-100, an experimental gene therapy designed to restore vision by reviving damaged neurons in the eye. Rather than slowing ...
First-ever experimental gene therapy seeks to restore vision by rejuvenating eye neurons
GeneCards®: The Human Gene Database GeneCards is a searchable, integrative database that provides comprehensive, user-friendly information on all annotated and predicted …
Search GeneCards Free for academic non-profit institutions. Other users need a Commercial license
Complete information for ACSL4 gene (Protein Coding), Acyl-CoA Synthetase Long Chain Family Member 4, including: function, proteins, disorders, pathways, orthologs, and …
Complete information for SQSTM1 gene (Protein Coding), Sequestosome 1, including: function, proteins, disorders, pathways, orthologs, and expression. GeneCards - The Human Gene …
CIViC Summary for SMAD3 Gene GeneCards Summary for SMAD3 Gene SMAD3 (SMAD Family Member 3) is a Protein Coding gene. Diseases associated with SMAD3 include Loeys …
Complete information for TNF gene (Protein Coding), Tumor Necrosis Factor, including: function, proteins, disorders, pathways, orthologs, and expression. GeneCards - The Human Gene …
Complete information for TNFAIP3 gene (Protein Coding), TNF Alpha Induced Protein 3, including: function, proteins, disorders, pathways, orthologs, and expression. GeneCards - The …
Complete information for G3BP1 gene (Protein Coding), G3BP Stress Granule Assembly Factor 1, including: function, proteins, disorders, pathways, orthologs, and expression. GeneCards - …
Complete information for TIMP1 gene (Protein Coding), TIMP Metallopeptidase Inhibitor 1, including: function, proteins, disorders, pathways, orthologs, and expression. GeneCards - The …
Complete information for NOTCH1 gene (Protein Coding), Notch Receptor 1, including: function, proteins, disorders, pathways, orthologs, and expression. GeneCards - The Human Gene …