Elixirgen Therapeutics has signed an option agreement with Nippon Shinyaku to advance and potentially commercialise its preclinical Duchenne muscular dystrophy therapy EXG-7001.

Elixirgen Therapeutics has entered into an option agreement with Nippon Shinyaku to develop and potentially commercialise EXG-7001, an investigational mRNA therapy for Duchenne muscular dystrophy (DMD).

Under the agreement, Elixirgen will continue development of the preclinical therapy while Nippon Shinyaku will fund development costs. If the option is exercised, Nippon Shinyaku will obtain exclusive worldwide commercialisation rights to EXG-7001.

The agreement includes an upfront payment to Elixirgen, with the company also eligible to receive development and sales milestone payments. Financial terms were not disclosed.

Following regulatory approval in the US, commercialisation would be led by NS Pharma, Nippon Shinyaku’s wholly owned US subsidiary.

EXG-7001 is being developed as a locally administered messenger RNA therapy designed to deliver full-length dystrophin protein. Unlike many existing approaches that focus on restoring truncated forms of dystrophin for specific genetic mutations, the therapy is intended to deliver the complete dystrophin protein regardless of the patient’s underlying mutation.

Duchenne muscular dystrophy is a rare, progressive neuromuscular disease caused by mutations in the DMD gene, resulting in the absence or dysfunction of dystrophin, a protein essential for maintaining healthy muscle function. Despite recent therapeutic advances, significant unmet need remains for treatments capable of addressing the underlying cause of the disease across a broad patient population.

Aki Ko, chief executive officer of Elixirgen Therapeutics, said: “Current approaches for treating DMD focus on delivering or restoring an incomplete dystrophin protein, and there still remains a significant unmet need for a therapy that can successfully deliver a full-length dystrophin protein.

“By design, EXG-7001 has the potential to deliver the full-length, complete dystrophin protein that is missing in DMD patients, regardless of their genetic mutation. We look forward to combining Elixirgen’s clinical and regulatory excellence with Nippon Shinyaku’s expertise and mission to improve the lives of those living with rare diseases.”

The collaboration combines Elixirgen’s development programme with Nippon Shinyaku’s established presence in rare diseases, providing funding to support continued advancement of EXG-7001 while creating a pathway towards potential global commercialisation.

Although EXG-7001 remains in preclinical development, the agreement reflects continued investment in next-generation genetic medicines targeting Duchenne muscular dystrophy, an area where researchers continue to seek therapies capable of restoring functional dystrophin across a wider range of patients than current mutation-specific approaches.