Year: 2011-12

Company: AVI Biopharma

Liaison(s): Brian Leppert

AVI BioPharma is a Seattle-based biotechnology company focused on developing RNA-based therapeutics against rare genetic diseases and certain viruses. With their versatile RNA technology, AVI BioPharma has successfully been able to direct alternative splicing of certain pre-mRNAs and inhibit translation of target mRNAs, to eliminate or limit the extent of the disease phenotype. This technology, known as Phosphorodiamidate Morpholino Oligomers (PMOs), can be used to create Splice Switching Oligomers (SSO®s) and Translation Suppressing Oligomers (TSO®s), which regulate target genes at the transcriptional and translational levels, respectively. The goal of the AVI BioPharma TMP is to identify future targets for drug development within the rare genetic disease space by creating a comprehensive and interactive database with a set of defined criteria and parameters. To accomplish this goal, the AVI BioPharma TMP team defined two specific deliverables; a comprehensive database (designed using Microsoft® Access ™) and a short list of 100-150 top diseases that would subscribe to the needs of AVI BioPharma’s drug discovery group. The short list would also be queriable through a Python script, which would allow AVI BioPharma to modify their target search by changing certain query variables. To accomplish these goals, the AVI BioPharma TMP team examined and retrieved data pertaining to rare genetic diseases from several pre-existing publicly available databases and developed specific prioritization criteria to subsequently filter and thus generate a short-list of genetic diseases targetable by AVI’s technologies. The diseases on the short list were then scored using critical parameters such research feasibility and market potential. The scoring criteria were then combined with subjective assessment to provide a numerical value guided by requirements set by AVI. Weights assigned to each parameter were designed to be readily modified within the interactive program created using Python script. Upon completion of this project, AVI BioPharma will be able to use the databases to generate prioritized lists of diseases to best meet their future research and business development goals.