Elixirgen Therapeutics is at the forefront of developing innovative RNA-based therapies aimed at addressing significant medical needs. Their proprietary Bobcat mRNA™ technology allows for the delivery of large genetic payloads, such as full-length dystrophin, which is crucial for treating Duchenne muscular dystrophy (DMD). This technology overcomes the limitations of traditional gene therapy vectors, which can only carry smaller payloads, by enabling the transport of over 13 kb of genetic material. This capability not only enhances the potential for effective treatment of DMD but also opens avenues for addressing other genetic disorders involving large genes, such as Ehlers-Danlos syndrome and Hemophilia A.

In addition to Bobcat mRNA™, Elixirgen has developed controllable self-replicating RNA (c-srRNA™) technology, which allows for regulated gene expression, minimizing off-target effects while maintaining elevated and prolonged expression levels. This technology is being explored for various therapeutic applications. Furthermore, their ZSCAN4 technology, which is currently in clinical trials, aims to extend telomeres in patients with telomere biology disorders, potentially offering new treatment options for conditions like bone marrow failure. Together, these advanced RNA technologies position Elixirgen Therapeutics as a leader in the development of next-generation therapies for rare and complex diseases.

United States

Elixirgen Therapeutics is a biotechnology company advancing its controllable self-replicating RNA technology to develop therapies for a broad spectrum of diseases, including rare diseases, aging-related disorders, and cancer. Elixirgen Therapeutics is located in the Science + Technology Park at Johns Hopkins in Baltimore.

Elixirgen Therapeutics

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