As we continue to explore the potential of RNA, new technologies are emerging that are revolutionizing the way we approach disease treatment.
At Conscience, we’ve seen a large spike in the number and quality of RNA therapeutics companies over the last few years, generally falling into the following 3 categories:
1. Using RNA as a drug.
For example, small interfering RNA (siRNA, and sometimes known as silencing RNA) can be used to target and silence the expression of genes that are overexpressed in certain diseases. It does so by degrading the mRNA that encodes the protein, therefore preventing its translation.
Switch Therapeutics is attaching siRNAs to sensors to conditionally activate it only in certain cells, and companies such as Eleven Therapeutics is using a computational approach to design siRNAs.
2. Using RNA to encode a drug.
This can take on many forms, but more commonly, as RNA, to encode for therapeutic proteins.
Strand Therapeutics, for example, is working on mRNA-based cancer therapeutics that can also control the location (cell type), timing, and intensity of expression of therapeutic proteins within the patient’s body, enabling precise and controlled delivery.
3. Using other molecules to modulate RNA.
RNA modulation by other small molecules or proteins can be used to turn genes on or off, increase or decrease RNA stability, or even modify RNA structure to alter its function.
Skyhawk Therapeutics is working alongside some of the largest pharma players, such as Sanofi and Merck, to develop small molecules that can regulate RNA splicing.
There are also innovations operating across the intersection of these categories, eg, Laronde’s “Endless RNA” as a new class of programmable medicines to express therapeutic proteins inside the body while using non-coding regions to control expression, stability, and targeting.
At Conscience, our most recent investment is a stealth company operating at the intersection as well.
We’re excited about the next generation of RNA technologies and how they will enable more durable, longer-lasting drugs that can target previously “undruggable” genes.
If you’re a scientist, founder, or investor operating in this space, reach out to us for a chat!
