RNA interference is a Nobel prize-winning discovery that allows for targeted silencing of disease-causing genes.
A central limitation in using RNA interference (RNAi) to treat neurological diseases has been the difficulty of achieving distribution throughout the central nervous system.
Many of the most pressing medical challenges in neurodegeneration require RNAi technologies to thoroughly reach all affected brain areas.
Our proprietary di-siRNA for the first time enables RNA interference to be deployed as a therapeutic approach throughout the brain and spinal cord.
Within human cells, genes are constantly being expressed by way of RNA. RNA is the message that conveys genetic directions into action in the body.
RNA interference is a naturally occurring process in cells that can be harnessed for gene silencing therapeutics in the following way.
Short pieces of synthetic RNA are designed to target the message of the disease-causing gene. These are called small interfering RNA,
or siRNA
These pieces of RNA join with the Argonaute proteins to form the enzymatic structure called the RNA-induced silencing complex, or RISC
One strand of the siRNA is discarded, and the RISC is activated for searching
The RISC scans for any and all matches within the cell’s gene messages
When it finds a match, the RISC destroys it, preventing the gene’s message from being translated into a functional protein, thus effectively modulating gene expression
The RISC is then free to search again
We have developed a novel oligonucleotide structure called di-siRNA, which for the first time can perform RNA interference throughout the brain and spinal cord.
Di-siRNA is a type of “informational drug.” Informational drugs share a common scaffold that supports their delivery and distribution, combined with a variable nucleic acid sequence that defines the drug’s target.
The common scaffold for our therapeutics enables effective delivery to the central nervous system and a predictable safety profile.
This scaffold can be customized to target different genetic messages. Scientific research is rapidly expanding our understanding of genetic contributors to CNS diseases. The programmable nature of di-siRNA allows for rapid generation of new therapeutic candidates based on the latest research.
Our novel di-siRNAs show unparalleled distribution throughout the brain and spinal cord as well as substantial persistence in the brain, which has been attributed to their slow clearance profile. Additionally, di-siRNAs show prolonged duration of effect. In our preclinical studies, a single dose of di-siRNA has shown potential to perform gene silencing for six months or more, possibly allowing for once or twice yearly dosing in a clinical setting.
Our initial areas of therapeutic focus will include Huntington’s disease, Parkinson’s disease, and Alzheimer’s disease, alongside other ongoing preclinical research work in other CNS diseases.
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