Codiak Receives $72.5 Million Up Front from Sarepta in Neuromuscular Disease Research Pact

Sarepta Therapeutics entered into a research alliance with Codiak BioSciences to design and develop engineered exosome therapeutics for neuromuscular diseases. The engineered exosome technology is believed to be a method for improving delivery of gene therapies, gene editing and RNA technologies by avoiding the adaptive immune response.

Under the terms of the deal, which will last two years, Codiak is eligible for up to $72.5 million in upfront and near-term license fees as well as research funding. Sarepta gains an option to any of the drug candidates.

“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,” said Doug Ingram, president and chief executive officer of Sarepta. “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 redosing.”

Exosome act as a communication network between cells, transferring a broad range of molecules between cells. Because they are natural nanoparticles, they offer a significant advantage as a delivery system because they don’t trigger the body’s immune system. Codiak’s engEx platform allows them to engineer exosomes with drugs or therapies and guide them to cells of interest.

The partnership will leverage Codiak’s exosome engineering technology with Sarepta’s precision genetic medicine expertise. The focus will be on neuromuscular diseases.

On June 1, Codiak announced it had entered into two strategic deals with the Ragon Institute of Massachusetts General Hospital, MIT and Harvard to study the potential of its exoVACC vaccine platform in SARS-CoV-2, the virus that causes COVID-19 and in HIV. As part of that deal, Codiak will work with Bruce Walker and Gaurav Gaiha of the Ragon Institute to develop integrated exosome-based vaccines.

The exoVACC platform uses exosomes to deliver antigens and adjuvants simultaneously and selectively to the same antigen presenting cells (APCs), which stimulate an integrated, innate, cellular and antibody-mediated immune response. The engEx platform helps design the single exosome multiple complex antigens and adjuvants.

Sarepta is best known for its approved drugs for Duchenne muscular dystrophy (DMD), Exondys 51 and Vyondys 53. Both treat DMD, but specific for DMD that is affected by different exons, 51 and 53, respectively.

DMD is a muscle wasting disease caused by mutations in the dystrophin gene. It is a progressive disease that usually causes death in early adulthood, with serious complications that include heart or respiratory-related problems. It mostly affects boys, about 1 in every 3,500 or 5,000 male children.

The current deal will focus on five neuromuscular targets, with exosomes that can deliver and functionally release different payloads, such as nucleic acids and gene therapy or gene editing constructs. No specific diseases were mentioned.

If Sarepta decides to exercise its option on one of the targets, it will handle clinical development and commercialization. In addition to the upfront fees, research funding and license payments, Codiak is eligible for development and regulatory milestones and tiered royalties on future sales.

“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 chief executive officer of Codiak. “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.”