Specific gene targets
Dicerna’s gene-targeting intellectual property provides us with a precise and long-lasting way to knock down specific disease-causing protein production. First recognized over 20 years ago, the concept of RNA interference targeting came from studies on the nematode Caenorhabditis elegans. Today our structural approach utilizes Dicer substrate short interfering RNA, or DsiRNA, to achieve our therapeutic goals. Our gene targets in this space include our proprietary primary hyperoxaluria (PH) program and its focus on the lactate dehydrogenase gene, our alpha-1 antitrypsin (A1AT) liver-associated disease program and numerous other gene targets with our collaborators.
GalXC technology platform
Along with targeting specific genes, Dicerna invented the GalXC RNAi technology platform, which enhances the Company’s DsiRNA constructs and improves their ability to safely deliver RNAi therapies that are specific and potent in the way they target disease. We continue to expand the utility of our GalXC platform through a variety of modifications and chemistries. Given their structure, our RNAs provide a long duration of action, have a high target specificity and have a high therapeutic index.
These compounds include DsiRNAs that have a chemically attached component for targeting, making them:
- Highly efficient in accessing the desired tissue.
- Suitable for administration via a simple injection under the skin.
The delivery/targeting vehicle is directly attached, or conjugated, to the RNAi molecule created with our GalXC technology.
We are using our proprietary GalXC platform to advance the development of next-generation RNAi-based therapies designed to silence disease-driving genes in the liver and other organ systems, including neural tissues, to address a broad range of other diseases.