Sarepta and Codiak Biosciences Join Hands to Tackle Rare Diseases Using Exosome-Based Therapeutics

By Pavel Ryzhov, Ph.D.

Partnerships between biopharma facilitate the acceleration of the research and development process and expedite the timeline of drug delivery to patients. Often, they leverage each other’s areas of expertise whether it is the proprietary technology or the clinical research experience. This “divide and conquer” approach is crucial to the faster advancement of cutting-edge therapeutics, like precision genetic medicines, because of the costs, risks, and longer return on investment. The recent announcement of an agreement between Sarepta Therapeutics and Codiak BioSciences is a great example of such an approach.

According to the press release, the two companies are entering into 2-year global research and option agreement with the aim to develop and commercialize new biologic therapies for up to five neuromuscular targets. The lynchpin of the approach is in the Codiak’s expertise in engineering exosomes, naturally occurring nanoparticles, typically used in cell-to-cell communication. Upon binding to their target cells, they can elicit a cellular response, based on the contents of the exosome.

Specifically, the engEx Platform, developed by Codiak, would be used to engineer exosomes as targeted delivery systems for efficiently delivering gene therapies, gene editing, and RNA technologies in a tissue-specific manner. It is made possible via two main technologies: the proprietary scaffold proteins, embedded in the exosome membrane, that can bind specific therapeutic molecules and proprietary manufacturing process for the production of highly pure exosomes and incorporation of nucleic acids or small molecules.

As Doug Ingram, President and CEO of Sarepta Therapeutics mentioned, another key advantage of the platform is in the “non-viral delivery approach with no immunogenic potential” which allows them to have more freedom with dosing regimens.

Codiak will receive up to $72.5 million in upfront payments, near-term license payments, and additional research funding, while Sarepta has an option for any co-developed candidate therapies. Depending on the success of the partnership, Codiak would also be entitled to obtain additional development and regulatory payments and royalties on any future sales of the medicines.

The successful implementation of this strategy would involve the collaboration between the companies to advance the engEx platform for neuromuscular disease indications, i.e. to target reengineered exosomes to muscle cells, potentially overcoming the current delivery difficulties and complementing the existing therapeutic modalities developed by Sarepta. In addition, the alliance would allow Sarepta to exclusively commercialize engineered exosome medicines. The “divide and conquer” approach would see Codiak being responsible for the preclinical research and developments leading to investigational new drug (IND) application, while Sarepta taking over the clinical trials and the subsequent path to market.

While the current approaches for gene therapy, editing, or RNA technologies involve using viral delivery, the acute immune response in the patient is one of the most important safety concerns. The strategic partnership between Codiak BioSciences and Sarepta Therapeutics signals their confidence that using engineered exosomes for targeted delivery of these biologics to the specific sites in the body, like muscle cells in patients with neuromuscular rare diseases, this next-generation approach may prove to be more efficient and limit the risk of adverse immunological complications.

Related Article: Mochida Pharma, Gene Techno Science Collaborate to Develop Regenerative Medicine for Rare Disease

References

1. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-and-codiak-biosciences-collaborate-research

Author

Pavel Ryzhov