On January 21, 2021, Codiak BioSciences, Inc. (NASDAQ: CDAK), a clinical-stage biopharmaceutical company focused on pioneering the development of exosome-based therapeutics as a new class of medicines, announced the advance online publication of a new manuscript. The paper, entitled “A Versatile Platform for Generating Engineered Extracellular Vesicles with Defined Therapeutic Properties” by Kevin Dooley (PhD, Associate Director, Protein & Cellular Engineering, Codiak) et al., was published online on January 20, 2021 (https://www.cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(21)00020-4), and will appear in an upcoming print issue of Molecular Therapy, the journal of the American Society of Gene and Cell Therapy. The publication describes the identification and characterization of two novel exosome-associated proteins, PTGFRN and BASP1, that enable Codiak’s proprietary engEx Platform and facilitate precise design and engineering of exosomes for potential therapeutic purposes. PTGFRN and BASP1 are highly abundant, naturally-occurring proteins that, through Codiak’s research, have been found to enable high-density exosome surface display and luminal loading of a wide range of macromolecules that can be directed to specific target cells of interest. In preclinical models, the use of PTGFRN and BASP1 to display biologically active molecules on the surface of or inside exosomes, respectively, resulted in uniform incorporation and increased potency, suggesting the potential utility of these engineered exosomes in a wide variety of therapeutic settings. These two proteins can also be used in combination, which further broadens the utility and modular approach to designing engineered exosomes for targeted delivery of numerous drug payloads to specific cells.
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