ADVANCED GENETIC SYSTEMS, INC. — Department of Health and Human Services STTR Phase I: 100

Description

DESCRIPTION provided by applicant There is a significant need for novel HIV therapies given the emergence of viruses resistant to existing drug regimens The Rev RRE protein RNA interaction in HIV plays an essential role in the transport of viral mRNA from the nucleus to the cytoplasm where it can be translated or packaged Previously we identified the thienopyridine scaffold that inhibited HIV replication and by targeting HIV Rev We carried out extensive structure activity SAR studies producing patentable new analogs that are fold more potent than our original screening hits and with therapeutic indices andgt exceeding our original goals Having successfully completed key milestones towards submission of an Investigational New Drug IND we propose to carry out a detailed study of the mechanism of action of the inhibitors This study is a key scientific milestone that will open many corporate and venture opportunities as well as significantly strengthen our opportunity to access SBIR STTR Phase II funding The work described in this proposal has a high likelihood for success given that in preliminary studies we have shown that a mutant in the RRE confers resistance to the compound PUBLIC HEALTH RELEVANCE We previously carried out a structure activity study of a promising Rev inhibitor and identified the key structural elements necessary for activity We propose to carry out experiments to establish the molecular mechanism of the molecule