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...
A new research paper titled “AAV1.NT-3 gene therapy prevents age-related sarcopenia” has been published in Aging. Sarcopenia is progressive loss of muscle mass and strength occurring during normal aging with significant consequences on the quality of life...
Remedium Bio today announced the publication of pre-clinical data demonstrating the safety and chondrogenic activity of RMD1101, its innovative gene therapy for Osteoarthritis. These data are the first to be published from Remedium’s ongoing research...
