Emerging opportunities in the viral vector manufacturing market include personalized medicine growth, expanded clinical trials, and regional manufacturing partnerships. Advances in bioprocessing and ...

A significant advance in treatment of patients with immunity to AAVs previously ineligible for clinical trials and existing ...

Lentiviral vectors have emerged as indispensable tools in gene therapy, offering the ability to integrate therapeutic genes into both dividing and non-dividing cells. Their unique capacity to achieve ...

Sarepta Therapeutics SRPT announced that the FDA has granted platform-technology designation to its viral vector rAAVrh74, used in the investigational gene therapy SRP-9003. This therapy is being ...

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 ...

Gene therapy is at the cutting edge of medicine, with exciting new products being developed and used to treat a wide variety of genetic diseases. However, one major roadblock to delivering gene ...

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 ...

Regeneron Pharmaceuticals, Inc. REGN announced updated data on its investigational gene therapy DB-OTO from the CHORD study. This gene therapy is being evaluated for profound genetic hearing loss due ...

Researchers have achieved the first demonstration in mice of using gene therapy to reverse hallmark symptoms of SYNGAP1-related disorder, a devastating condition affecting an estimated 1 million ...

Gene therapy achieved its first major breakthroughs in the early 1990s with the treatment of a rare disease called severe combined immunodeficiency. However, the field was dealt a serious setback in ...