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

Rare Daily Staff

Sarepta Therapeutics and Codiak BioSciences 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 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. It is granted Sarepta 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 variety of 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.

“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 Williams, president and CEO of Codiak Biosciences. “Engineered exosomes may offer a solution through their potential to selectively target muscle cells.”

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.

Under the terms of their 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.

“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,” said Doug Ingram, president and CEO of Sarepta Therapeutics. “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.”

Photo: Doug Ingram, president and CEO of Sarepta Therapeutics

The photo for this story was updated July 22, 2020. The original photo used was incorrectly identifies as Dough Ingram.