Isis Pharmaceuticals have an impressive patent portfolio built upon th
eir ability to synthesise modified synthetic oligonucleotides in large
amounts. The use of these compounds to inhibit gene expression by blo
cking translation or activating RNase H has been demonstrated in vitro
and one product is in Phase III clinical trials. Much depends upon th
e Company's ability to use antisense oligonucleotides designed to inhi
bit ras, raf, ICAM-1 and protein kinase C in disease tissues where the
se proteins are valid drug targets. Having established this, the quest
ion of drug delivery in whole animals will need to be addressed.