Press Releases / Notices

KYOTO, Japan, June 26, 2026 – Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Headquarters, Kyoto; President, Toru Nakai) and Elixirgen Therapeutics, Inc. (Elixirgen; Headquarters: Baltimore, U.S.A.; CEO: Aki Ko) have entered into an option agreement under which Nippon Shinyaku may obtain exclusive worldwide rights to develop and commercialize EXG-7001 for the treatment of Duchenne muscular dystrophy (DMD). 

DMD is a progressive muscular dystrophy caused by a deficiency of the dystrophin protein, which supports muscle cells, leading to muscle weakness in skeletal, cardiac, and respiratory muscles; various genetic mutations are known to cause this condition.

EXG-7001 is a locally administered, mRNA-based therapeutic drug that expresses full-length human dystrophin protein. Regardless of genetic mutation, it suppresses local muscle dysfunction by expressing the full-length human dystrophin protein, which is deficient in DMD, within muscle cells. Elixirgen is currently preparing for clinical trials in the United States.

Following the exercise of the option rights by Nippon Shinyaku and regulatory approval in the United States, NS Pharma, Inc. (New Jersey, USA, President: Yukiteru Sugiyama), a wholly owned subsidiary of Nippon Shinyaku, will market EXG-7001.

Nippon Shinyaku is working to develop new treatments for intractable and rare diseases and aims to be a company that is trusted by society through the creation of unique drugs.

Based on Nippon Shinyaku’s business philosophy, “Helping people lead healthier, happier lives,” we aim to be an organization trusted by the community through creating unique medicines that will bring hope to patients and families suffering from illness. Please visit our website (https://www.nippon-shinyaku.co.jp/english/) for products or detailed information.

Elixirgen is a clinical stage biotechnology company focused on the treatment of rare and aging-associated diseases using its cutting-edge technologies. For more information, visit ElixirgenTx.com and follow us on LinkedIn.