Sarepta Therapeutics and Codiak BioSciences Collaborate to Research and Develop Exosome-Based Therapeutics for Rare Diseases - Seite 2

“The development of targeted delivery systems that enable repeat-dosing to select cell types has been a challenge for the emerging field of genetic medicine, especially in diseases of the muscle,” said Douglas E. Williams, President and CEO of Codiak BioSciences. “Engineered exosomes may offer a solution through their potential to selectively target muscle cells. We are excited to work with the world-class team at Sarepta to further build the engEx Platform and evaluate the potential of exosomes as next generation constructs incorporating gene therapy, gene editing and other nucleic acid payload modalities.”

Under the terms of the agreement, Sarepta has the exclusive option to license Codiak’s technology to develop and commercialize engineered exosome therapeutics for up to five neuromuscular targets. Sarepta and Codiak will collaborate to design exosomes that can deliver and functionally release select payloads, such as nucleic acids and gene therapy and gene editing constructs, in neuromuscular indications. If Sarepta elects to exercise its option on a target, Codiak will be responsible for research and preclinical development through IND preparation, and Sarepta will be responsible for clinical development and commercial activities. In addition to upfront, research funding and license payments, Codiak will be eligible to receive significant development and regulatory milestone payments and tiered royalties on future sales.

About engEx Platform
The engEx Platform is Codiak’s proprietary exosome therapeutic engine for engineering and manufacturing novel exosome product candidates designed to target multiple pathways throughout the body. Using this platform, Codiak can design exosomes with precisely engineered properties, incorporate various types of biologically active molecules and direct them to specific cell types and tissues. These exosomes engage targets by cellular uptake, membrane-to-membrane interaction or a combination of both mechanisms and are designed to change the biological functioning of the recipient cells in order to produce the intended biological effect. Codiak is building a broad pipeline of engEx product candidates that may have a transformative impact on the treatment of many diseases.