Rare Diseases and Orphan Drugs

Disorders caused by missing or defective protein synthesis can lead to early-onset complications and the patients need a life-long treatment. Today, treatment options are often limited or ineffective and associated with high costs. Here, the in vitro transcribed (IVT) mRNA can be used as a potential drug. Synthetic mRNA has several advantages over conventional gene

therapy and protein substitution strategies. The mRNA-based therapy is founded on the induction of the transient translation with the cell own ribosomes of fully functional proteins without integration into host genome. This approach minimizes the risk of mutagen and carcinogen effects. The control of translation duration as well as immunogenicity of mRNA can be achieved though different modifications and provide for different therapeutically applications, like replace or supplement proteins. Moreover, mRNA cocktails contacting mRNAs encodes for different proteins can be applied to induce the expression of different proteins simultaneously in one cell. The use of mRNA to develop therapeutic drugs opens up new perspectives and challenges in disease treatment.