Codiak BioSciences, Inc. announced new preclinical data on the Company's engEx-AAV™ discovery program, a novel strategy that aims to leverage exosomes to improve adeno-associated virus (AAV) vector gene therapy. The data, which are being presented at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), demonstrate that exosome engineering generates significant increases in AAV yield compared to unmodified exosomes, while retaining the functionality to transduce cells with AAV and resist neutralizing antibodies that impair gene therapy efficacy. These data show the power of engineering platform to enable exosome delivery of AAV as a therapeutic and the potential of this approach to address key limitations associated with AAV gene therapy currently, said Sriram Sathyanarayanan, Ph.D., Chief Scientific Officer, Codiak.

The company has identified the engineering methods and multiple exosome constructs that effectively catch' AAV capsids and release' them to cells, resulting in highly efficient transduction By increasing the AAV yield with engEx-AAV, The company has successfully cleared a significant hurdle, and as the company advances this program, The company will employ a variety of models to examine regional, systemic and repeat dosing. AAV is a commonly used gene therapy vector whose once and done clinical potential faces challenges including the presence or subsequent development of neutralizing antibodies (nAbs) preventing treatment or re-dosing, waning transgene expression, and difficulties in producing sufficient quantities of AAV. Encapsulation of AAV within exosomes (engEx-AAV) represents a strategy to protect AAV from antibody-mediated neutralization and efficiently deliver AAV to specific cell types, leading to higher potency and lower systemic exposure.

In an retinal model, engEx-AAV transduced cell types that were not affected in AAV-injected eyes, indicating engineered engEx-AAV exhibits a preferential tropism that could offer important utility in ocular disease indications.