New research in cystic fibrosis rats shows that chronic Pseudomonas aeruginosa lung infection does not hinder lentiviral ...

Jessica Nance, MD, MS, discusses maximizing gene therapy efficiency by selecting vectors for gene size, tissue targeting, and low exposure, and focusing on precise promoters for expression. This is a ...

An R75W mutation in the gap junction β2 (GJB2) gene causes severe fragmentation of gap junction plaques, connecting adjacent cells and leading to syndromic hearing loss. In a new experimental study, ...

October 12, 2026) - Frost & Sullivan officially released the 2026 Global Gene Therapy Industry Development Blue Book in October 2026, in collaboration with industry partners. The report systematically ...

Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a ...

Regenxbio has a competitive advantage in the market with its proprietary NAV technology platform, based on AAV vectors, that is highly effective, efficient, and scalable. The recent Phase II trial on ...

Cell and gene therapies rely on engineered viruses to deliver them to their cellular destinations. While the progress of these types of medicines is evident in a growing number of FDA product ...

Investigators headed by a team at Brigham and Women’s Hospital have developed an adeno-associated virus (AAV) vector that studies show can far more efficiently cross the blood-brain barrier (BBB), in ...

Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a ...