Elixirgen Therapeutics

About Elixirgen Therapeutics

Elixirgen Therapeutics, Inc. is a clinical-stage biotechnology company located in Baltimore, Maryland, focused on developing therapies for rare genetic diseases and aging-related disorders. Co-founded in May 2017 by Chief Executive Officer Akihiro Ko and his father, Chief Scientific Officer Dr. Minoru Ko, the company leverages its proprietary RNA platform technologies to address unmet medical needs. Akihiro Ko earned a Bachelor of Science from the University of Virginia and previously served as COO for the parent company, Elixirgen, LLC, and President of Elixirgen Scientific, LLC. Dr. Minoru Ko brings over 30 years of experience in stem cell biology and genetics, having formerly worked as a Senior Investigator at the National Institute on Aging (NIA), a division of the National Institutes of Health (NIH), where his lab made key discoveries that form the basis of the company's technology.

The company is advancing three core technologies: ZSCAN4, Bobcat mRNA™, and controllable self-replicating RNA (c-srRNA™). The ZSCAN4 technology is the basis for the company's lead candidate, EXG-34217, an ex-vivo autologous cell therapy for Telomere Biology Disorders (TBDs). This therapy, which has received Orphan Drug, Rare Pediatric Disease, and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA, aims to elongate telomeres in a patient's own hematopoietic stem cells to treat conditions like bone marrow failure. An ongoing Phase 1/2 clinical trial has shown sustained telomere elongation in patients without treatment-related adverse events.

The Bobcat mRNA™ technology is a linear mRNA platform designed to deliver large protein payloads, such as the full-length dystrophin protein, which is mutated in Duchenne muscular dystrophy (DMD). Preclinical data in mouse models have demonstrated the restoration of muscular function. This technology is engineered to be a redosable and cost-effective alternative to other gene therapies.

The controllable self-replicating RNA (c-srRNA™) platform is a temperature-sensitive RNA designed for intradermal injection, which allows it to function at skin temperature (33°C) but become inactivated at core body temperature (37°C), a feature intended to enhance safety. This technology was initially developed for a COVID-19 vaccine candidate, EXG-5003, and is being explored for other applications like cancer immunotherapy.