After launching RaNA Therapeutics Inc. in 2011 to exploit the therapeutic potential of long-noncoding RNA (lncRNA), the company has gone where the science led it, and added a second platform that uses RNA oligonucleotides to stabilize mRNA. With much of the mechanistic work behind it, RaNA is now lining up a lead compound from each platform to take to the clinic next year.
The core platform was based on the work of Jeannie Lee, professor of genetics and pathology at Harvard Medical School, who uncovered the importance of lncRNAs - transcripts derived from the so-called "junk DNA" that doesn't encode proteins. Lee is also on the scientific advisory board at RaNA and is the recipient of this year's Foundation for the National Institutes of Health (FNIH) Lurie Prize in Biomedical Sciences.
RaNA CEO Ronald Renaud told BioCentury that Lee identified the role of lncRNAs as transcriptional repressors when she questioned the prevailing wisdom that held they had no function.
"What she stumbled on was lncRNA's activity in recruiting PRC2 to certain areas in the genome, where PRC2 would place repressive epigenetic marks on specific genes," he said. PRC2 is an enzyme commonly involved in lncRNA activity that adds methyl groups to histone proteins, which silences transcription of surrounding genes.
The idea was to block the interaction of the lncRNA with the PRC2 protein by delivering short, single-stranded RNA oligonucleotides that would sterically interfere with the