Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

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 ...

PITTSBURGH, Oct. 14, 2025 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (KRYS) (the “Company”) (NASDAQ: KRYS) announced today that the United States Food and Drug Administration (FDA) granted platform ...

After completing short-term follow-up studies, 14 patients were enrolled in the current study, which was designed to extend patient monitoring to ensure a total duration of 15 years, in compliance ...

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 ...

Co announces that a patent has been awarded titled "Diminishing Viral Gene Expression by Promoter Replacement" by the U.S. Patent and Trademark Office focusing on adenoviral vector development, which ...

Nine-year follow-up of gene therapy in haemophilia B confirms long-term safety and reduced bleeding, though full capsid ...