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Elixirgen Therapeutics, Inc. is a clinical-stage biotechnology company focused on the discovery, development, and commercialization of therapies using its controllable RNA platform.

The company’s experienced team of researchers has a wide variety of specialties, enabling it to use both basic and translational research approaches to developing solutions for genetic diseases, cancer immunotherapy, and vaccines. Elixirgen Therapeutics is located in the Science + Technology Park at Johns Hopkins in Baltimore, an ideal research environment that allows Elixirgen Therapeutics' scientists to conduct research at state-of-the-art facilities.

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A Versatile Delivery Platform

in vivo controllable RNA
Immunotherapies and Vaccines

Controllable self-replicating RNA (c-srRNA) is a temperature-controllable RNA platform that is optimized for strong expression at 30°C-35°C, and inactivated at 37°C.

This function is used as a versatile, safety-focused expression-switch for the mRNA, and is currently being applied towards in vivo immunotherapies and vaccines.

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ex vivo controllable RNA for Telomere Biology Disorders

Controllable RNA is also currently being applied towards ex vivo cell therapy. Its temperature-controllable feature allows us to tune the expression of the therapy gene.

Our lead ex vivo candidate, EXG-34217, is an autologous cell therapy for telomere biology disorders with bone marrow failure that uses ex vivo c-srRNA with Elixirgen Therapeutics’ proprietary ZSCAN4 technology to extend the telomeres of patients.

A Phase I/II clinical trial is in progress to assess the safety and tolerability of EXG-34217 at Cincinnati Children's Hospital.


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