Replicor

Replicor is a clinical-stage biopharmaceutical company developing nucleic acid polymers (NAPs). These compounds are oligonucleotides whose antiviral activity strictly depends on the sequence-independent activity of phosphorothioate oligonucleotides as biocompatible polymers interacting with hydrophobic surfaces exposed in proteins. This exposure of hydrophobic characteristics is rare in mammals but common in the life cycles of many different pathogens. The high-affinity "annealing" of NAPs along these hydrophobic surfaces is insensitive to the development of resistance (as seen with small molecules and siRNA) and effectively blocks the structural rearrangements necessary for viral replication.Unlike all other classes of oligonucleotide-based drugs (e.g., antisense oligonucleotides, synthetic interfering RNA (siRNA), or microRNA (miRNA)), the activity of NAPs does not depend on the sequence of nucleotide building blocks. Due to this unique property, NAPs can be engineered with nucleotide sequences that retain antiviral activity but eliminate immune responses or off-target effects commonly associated with other oligonucleotide-based drugs. Additionally, naturally occurring nucleotide modifications that identify themselves as "host" molecules do not interfere with the activity of NAPs. Through all these optimizations, the current leading NAP candidate, REP 2139, has demonstrated superior safety and tolerability in preclinical and clinical studies, unmatched within its class.