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...
Providing an overview of the submissions process and examples of U.S. Food and Drug Administration (FDA) applications for Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD), a new article can help developers of gene therapies for rare genetic...
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...
February 28 is Rare Disease Day, which looks to raise awareness of rare diseases and bring about change for patients and families living with them. The event also brings attention to the need for improved access to diagnosis, as well as the need to develop more...
February 28th is the 16th International Rare Disease Day with the theme of “Shine lights on your life” . Rare diseases, also known as “orphan diseases”, have a low incidence rate and multiple disease types. Most of them are chronic, lifelong,...
