Messenger RNA (mRNA) therapeutics can treat diseases caused by a lack of protein, or by defective proteins, such as Duchenne muscular dystrophy and cystic fibrosis.
Each gene in our DNA contains the code needed to make one or more proteins. mRNA carries that code from the cell’s nucleus to the machinery that translates it into proteins. Once the proteins are made, the mRNA is cut up and recycled. If a gene has a mutation that stops it from producing protein or causes it to produce defective proteins, mRNA medicine can be used to ensure that a healthy version of the missing protein is produced.
Arcturus uses its proprietary UNA Oligomer chemistry and LUNAR™ nanoparticle delivery platform to develop mRNA therapeutics for rare disease targets. Rare diseases affect 350 million people worldwide. Over 80% of rare diseases are caused by gene mutations and 95% of rare diseases do not yet have an approved treatment.