A New Partnership will Advance Exosome-Based Gene Therapy for Neuromuscular Diseases

By engineering exosomes, Sarepta Therapeutics believes they can utilize gene therapies, RNA therapies, and gene editing without triggering an immune response in the body. They have just announced a new agreement with Codiak to develop engineered exosomes, specifically for neuromuscular conditions such as forms of muscular dystrophy like Duchenne muscular dystrophy (DMD).

How It Works

Exosomes in the body work to facilitate communication between cells. They help to deliver molecules from one cell to another. Since they are a naturally occurring part of the body’s processes, using them as a method for gene therapy means the body won’t initiate an immune response against them. Most gene therapies use a viral approach which often triggers an immune response in the body.

Codiak has a technology called the engEx Platform which works to give exosomes different molecular cargos and direct them to specific cells. This process has proved to be exceptionally difficult for diseases involving the muscle, and researchers are hopeful that this new modality will provide great benefit to these patient populations.

The Agreement

Codiak has the technology, and Sarepta has the expertise in genetic medicine. The agreement between the two will last for 2 years and will evaluate up to 5 different neuromuscular targets. They will collaborate in the design of the exosomes to ensure the best potential outcomes for neuromuscular indications.

At its start, Codiak will be provided up to 72.5 million dollars for their research and for license payments. Other payments will be available for reaching certain development as well as regulatory milestones. For all future sales, they will be given tiered royalties. They will be responsible for all preclinical development as well as the submission of the investigative new drug application.

Sarepta has been awarded the exclusive opportunity to license and commercialize the technology. Sarepta will be in charge of all clinical development as well as the commercial activities.

You can read more about this new approach here.

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