Re: Antisense and RNAi Citing the relative advantages of oligonucleotide molecules, Viaud remarked, “Intermediately sized bioactive molecules, peptides, small RNAs, and DNA oligonucleotides may offer the advantages of both small molecule-based drugs and biologicals while avoiding many of their shortcomings.” From the point of view of a drug developer, these molecules have multiple advantages including simplified rational drug design; simple, scalable, and inexpensive synthesis; and the possibility of extensive chemical modifications to enhance their properties, such as pharmacokinetic and clearance profiles, he added.
GEN asked Viaud what challenges remain for oligonucleotide development. “Since not many antisense oligonucleotides have made it to regulatory approval, our biggest challenge has been for our community to recognize the potential of an oligonucleotide.” But given the advantages of oligonucleotide drugs and some recent clinical successes, large pharma companies may reconsider their development pipeline options.
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