Sarepta Therapeutics, Inc. and Codiak BioSciences, Inc. announced a global research and option agreement to design and develop engineered exosome therapeutics to deliver gene therapy, gene editing and RNA technologies for neuromuscular diseases. The engineered exosome approach offers the potential to effectively deliver genetic therapeutics without triggering the adaptive immune response.
The two-year agreement includes up to five neuromuscular targets. Codiak is eligible to receive up to $72.5 million in upfront and near-term license payments plus research funding. Sarepta is granted an option to any of the candidates developed pursuant to the research alliance.
Exosomes are natural nanoparticles that serve as the body’s intercellular communication system, facilitating the transfer of a wide variety molecular payloads between cells. As they are derived from human cells, exosomes provide a unique advantage as a targeted delivery system for genetic medicines because they are inherently non-immunogenic. Through its proprietary, engEx Platform, Codiak can engineer exosomes with specific cargos and guide tropism to cell types of interest. The collaboration will leverage Codiak’s exosome engineering capabilities with Sarepta’s expertise in precision genetic medicine to develop next generation therapeutics for patients with neuromuscular diseases that have few or no treatment options.
“As Sarepta expands its leadership position in precision genetic medicine, this alliance with Codiak furthers our goal to deliver the most advanced therapies to patients. Codiak’s engEx technology could potentially address some of the limitations of current treatment approaches and offers broad utility across Sarepta’s therapeutic modalities — gene therapies, gene editing and RNA. Codiak’s exosomes are engineered for precise tissue targeting and offer a non-viral delivery approach with non-immunogenic potential, thus opening up avenues for more efficient delivery and potential re-dosing,” said Doug Ingram, president and chief executive officer, Sarepta Therapeutics.
“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.