Recently, the first patient dosing of NXL-004, an AAV-NeuroD1 gene therapy product developed by NeuExcell Therapeutics, was successfully completed at the Fourth Affiliated Hospital of Soochow University (Suzhou Dushu Lake Hospital). The procedure was carried out by...
Here’s the easy game Li Xincheng has been playing at home. Her mother says a few words. Then the six-year-old, nicknamed Yiyi, repeats what she heard. “Clouds, one by one, blossomed in the mountains,” says her mother, Qin Lixue, while covering her mouth so Yiyi can’t...
Boston-based Decibel Therapeutics, in collaboration with Regeneron Pharmaceuticals, has been granted orphan drug designation by the European Medicines Agency (EMA) Committee on Orphan Medicinal Products (COMP) for its lead gene therapy product candidate, DB-OTO. This...
At the 2023 Muscular Dystrophy Association Clinical and Scientific Conference, Novartis presented new data highlighting the continued efficacy and durability of its gene therapy treatment, Zolgensma, in treating spinal muscular atrophy (SMA). Zolgensma has been shown...
M281 treatment prevents human NAb-mediated reduction of liver-targeted gene delivery after intravenous AAV administration in humanized FcRn transgenic mice. (A) Scheme of the in-mouse study assessing M281 effects on hIgG and anti-AAV NAb levels and subsequent systemic...
