Elixirgen Therapeutics, Inc

Elixirgen Therapeutics is a clinical-stage RNA therapeutics company focused on the discovery and development of innovative therapies, including cell and gene therapies. The Company is advancing three technologies to develop therapeutics for a broad spectrum of diseases including rare and genetic diseases, and aging-related disorders. Bobcat mRNA™ technology uses a proprietary mRNA design to deliver large payloads such as full-length dystrophin, which is mutated in Duchenne muscular dystrophy. Preclinical data have demonstrated restoration of muscular function in DMD model mice with no safety findings associated with administration or treatment. Controllable self-replicating (c-srRNA™) technology controls the powerful gene expression of self-replicating RNA to reduce off-target effects and improve efficacy. ZSCAN4 technology, delivered via the RNA-based Sendai virus, is currently in a Phase 1/2 clinical trial to extend telomeres in patients with telomere biology disorders (TBDs), a rare disease with significant unmet need. Initial data have demonstrated ex vivo telomere elongation in TBD patients for the first time ever and suggest the potential emergence of a cell population with longer telomeres in vivo. Elixirgen Therapeutics is located in the Science + Technology Park at Johns Hopkins in Baltimore.