A phase 1/2 trial of a novel viral vector gene therapy in adults with type 1 diabetes is scheduled to begin this year.
UF scientist develops safer gene therapy restoring vision in children with rare inherited eye diseases; pivotal trials begin ...
A groundbreaking gene therapy tested through a UCLA-led clinical trial has received approval from the U.S. Food and Drug ...
A groundbreaking gene therapy trial has successfully restored hearing in individuals with a rare form of congenital deafness.
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Base-Editing Gene Therapy Shows Promise in Sickle Cell Disease
Durable expression of anti-sickling fetal hemoglobin and reduction in sickle hemoglobin ob ...
The milestone reflects KFSH’s ongoing efforts to advance its gene therapy program, bridging clinical care with translational ...
Modifying hematopoietic stem cells to correct the underlying genetic defect, followed by reinfusion of the modified cells ...
After years of hospital stays, these kids are thriving thanks to a gene therapy that just earned accelerated FDA approval.
If approved, UX111 will be the first approved therapy for the treatment of Sanfilippo syndrome Type A, a rare disease ...
CRISPR Cas9 gene therapy explained with DNA scissors, hereditary diseases treatment, and designer babies ethical dilemmas ...
Early biomarker and safety data show encouraging signals with plans to present additional data in 2H 2026 AviadoBio secures ...
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