Isis Pharmaceuticals received a phase 2 SBIR grant from the NIH for up to $1.5 million to design oligonucleotide therapeutics that exploit the RNAi antisense mechanism.
The phase 2 funding builds upon a successful phase 1 program that demonstrated the feasibility of using single-stranded antisense drugs to target the RNAi pathway.
“Based on our extensive work with single-stranded antisense drugs that work through an RNase H mechanism and the feasibility studies we have completed with single-stranded antisense drugs that harness the RNAi pathway, we are optimistic that the two drug families will share certain characteristics including bioavailability and tissue distribution,” points out C. Frank Bennett, Ph.D., svp of research.
The multiyear NIH backing will fund research to improve the stability and tissue distribution of RNAi drugs. Isis says that much of the work will focus on optimizing the chemical properties of single-stranded oligonucleotides that trigger the RNAi pathway.