Formulation Development and Feasibility of AAV5 as a Lyophilized Drug Product.

The majority of adeno-associated virus (AAV) gene therapies are currently developed as frozen formulations (e.g., ≤ - 60°C) that are challenging to maintain and distribute world-wide.

The inflammasome adapter ASC assembles into filaments with integral participation of its two Death Domains, PYD and CARD.

The inflammasome is a multiprotein complex necessary for the onset of inflammation. The adapter protein ASC assembles inflammasome components by acting as a molecular glue between danger-signal sensors and procaspase-1. The assembly is mediated by ASC self-association and protein interactions via its two Death Domains, PYD and CARD.