Arcturus Therapeutics

Messenger RNA (mRNA) can be used as protein replacement therapy to treat diseases caused by a lack of protein, or by defective proteins, such as cystic fibrosis. If a gene has a mutation that stops it from producing protein or causes it to produce defective protein, mRNA medicine can provide a healthy version of the missing protein.

We have a number of mRNA medicine development programs that leverage our proprietary LUNAR delivery system, including a partnership with Cystic Fibrosis Foundation Therapeutics, Inc. to develop novel mRNA therapeutic candidates to treat cystic fibrosis and a strategic collaboration with CureVac AG to jointly discover, develop and commercialize mRNA therapeutic candidates to treat ornithine transcarbamylase (OTC) deficiency and other diseases. We have also partnered with Ultragenyx to develop up to ten mRNA therapeutic candidates for rare disease targets. The first disclosed indication is Glycogen Storage Disease type III.

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