Creation of New Nucleic Acid Therapeutics Realized by the Third Platform Technology
Expectations for Nucleic Acid Therapeutics
In the pharmaceutical market, nucleic acid therapeutics are attracting increasing attention in response to unmet medical needs, including cancer and intractable diseases.
This is due to the following advantages of nucleic acid therapeutics.
- Enables discovery of intracellular targets that cannot be targeted by conventional pharmaceuticals.
- Discovery research is possible for any gene as long as the technology to deliver the nucleic acid to the expression site of the target gene is available.
- More specific than low molecular weight compounds and less likely to cause side effects.
- Unlike antibody drugs, nucleic acid therapeutics can be produced relatively inexpensively through chemical synthesis
The challenges of Nucleic Acid Therapeutics
However, existing nucleic acid therapeutics have been regarded as having limited potential for practical use because of the following issues.
- Challenges in delivery: Difficulty in getting drugs to the site of disease.
- Challenges of Toxicity: Mainly side effects such as hepatotoxicity.
- Challenges in blood stability: degradation in blood due to enzymes and other actions after administration.
Solution by heterogeneous Nucleic Acid technology
Hetero-duplex oligonucleotide (HDO), developed by Professor Takanori Yokota and his colleagues at Tokyo Medical and Dental University, has the potential to radically solve these problems. It is our goal to establish the third platform technology after Ionis Pharmaceuticals, Inc (ASO) and Alnylam Pharmaceuticals, Inc (siRNA) to contribute to the creation of new nucleic acid therapeutics, thereby bringing therapeutics to patients as soon as possible.