Transcript Therapies are exciting alternatives to gene therapies and protein-based drugs. Messenger RNA is a single-strand of nucleotides that transcribes genetic information found in DNA into a message that can be read by the machinery of a cell and translated into proteins that the body needs to function.
Historically, mRNA has not been usable as a therapeutic agent because when delivered into the body it activates the immune system and is highly unstable. In addition, in order to be functional, mRNA must enter the target cells of interest by crossing the cell membrane, which requires a carrier system to transport it into the cells.
Ethris has developed two technology platforms to enable the use of messenger RNA as a therapeutic agent: The Stabilized Non-Immunogenic mRNA (SNIM®RNA) for transcript therapy, as well as proprietary delivery technologies for SNIM® RNA delivery.
Our SNIM® RNA Technology is a novel nucleic acid therapy platform based on stabilized non-immunogenic mRNA. SNIM® RNAs overcome the innate instability and immunity of mRNA. They evade the innate immune system due to chemical modifications in their building blocks. SNIM® RNAs can be administered repeatedly, leading to sustained production of therapeutically active proteins within the human body. This can be used in the therapy of hereditary diseases, as an alternative to protein therapy, and in regenerative medicine.