Topical delivery of therapeutic aptamers

PROJECT ABSTRACTTopical delivery of therapeutic agents across skin offers key benefits in improving patient compliancelowering systemic drug exposureand enabling tunable deliveryTherapeutic macromolecules such as antibodies or aptamers enables better efficacyless off target effectsand longer half life comparing to small moleculesand may unlock tremendous additional value if delivered topicallyMultiple approaches have been developed for transdermal delivery of macromoleculesbut have so far met with commercial and or clinical setbacksIn consequenceso far topical delivery is critically limited to small molecules that possess particular physicochemical properties to diffuse across the skin barrierThere is a significant unmet need for a platform technology that can effectively deliver therapeutic macromolecules across the skinAptamers are single stranded oligonucleotides that bind to molecular targets with high potency and specificitysimilar to antibodiesComparing to antibodiesaptamers are a unique fit for topical delivery thanks to its small sizesuperior stabilityand lack of immunogenicityWe propose to develop the first platform to effectively deliver therapeutic aptamers across the skinand enable safer and more efficacious treatment of skin diseasesIf successfulthis platform has the potential to enable topical delivery of aptamers targeting many targets and disease indications Therapeutic macromolecules such as aptamers enables better efficacyless off target effectsandlonger half life comparing to small moleculesand may unlock tremendous additional value ifdelivered topicallyWe propose in this project to develop the first platform to effectivelydeliver therapeutic aptamers across the skinIf successfulthis platform has the potential toenable topical delivery of aptamers targeting many targets and disease indications