790 Memorial Drive, Suite 203
Cambridge, MA 02139
RaNA Therapeutics is breaking new ground in the field of epigenetic drug discovery with a unique approach to upregulating genes in a predictable and clinically relevant manner. For the first time, the company’s proprietary epigenetic gene-specific activation platform makes it possible to increase protein expression of a wide variety of genes with exquisite selectivity. RaNA’s revolutionary approach leverages new discoveries in the science of epigenetic gene regulation, a field which previously focused only on enzyme inhibitors whose non-specific effects often regulated thousands of genes. RaNA’s goal is to generate new, highly-selective treatments in many therapeutic areas, with an initial emphasis in the fields of rare genetic disorders, oncology, metabolic diseases and neurodegenerative diseases.
Research by scientists around the world, including RaNA Scientific Founder, Dr. Jeannie Lee,1has established that non-coding RNA perform a variety of critical regulatory functions in controlling gene expression, cell differentiation, and cell development and are increasingly recognized as having diverse regulatory effects on mRNA transcription.2, 3 RaNA’s approach represents a pioneering step in the field of oligonucleotide therapeutics. Whereas other approaches silence the expression of disease-causing genes by destroying mRNA and blocking production of an unwanted gene product, RaNA uses single-stranded oligonucleotides to target the repressive interactions of long non-coding RNA (lncRNA) with specific epigenetic modulators, thereby upregulating mRNA and increasing expression of the encoded protein.
During transcription of the long non-coding RNA, a PRC2 binding domain is revealed, which recruits EZH2 and other epigenetic factors locally. PRC2 activity is then targeted to the nearby gene, suppressing gene transcription.
RaNA’s therapeutic oligonucleotide can be administered as a subcutaneous injection in saline, and is then taken up by cells in most tissues of the body, crossing the endosome membrane to enter the cytoplasm and nucleus. When transcription of the unique lncRNA target reveals the PRC2 binding domain, the RaNA antagonist therapy binds the lncRNA, which blocks PRC2 recruitment and allows for transcription to proceed resulting in mRNA upregulation.Learn More About RaNA’s Revolutionary Science
Based in Cambridge, Massachusetts, RaNA’s team leverages aptitude and experience gained through an extensive record of success.
Art brings more than 20 years of experience in oligonucleotide R&D in academia, biotech, and pharma environments.
Jim has more than 25 years’ experience in biotechnology drug discovery and development. Dr. Barsoum has generated protein, small molecule, and gene therapy drug candidates in multiple therapeutic areas.
René has worked in biopharma corporate development, M&A and corporate finance, both in-house and as a consultant, for over ten years.
RaNA received initial seed funding from Atlas Venture in 2011 and raised a Series A financing in January of 2012, which included Atlas Venture, MGH Partners Innovation, Monsanto, and SR One.
Atlas Venture is a leading early-stage venture capital firm that invests in technology and life sciences companies. Since inception in 1980, Atlas has helped build over 350 companies in more than 16 different countries.
Monsanto Company is a leading global provider of technology-based solutions and agricultural products that improve farm productivity and food quality.
SR One is the independent corporate venture capital arm of GlaxoSmithKline. The firm invests globally in emerging life science companies that are pursuing innovative science which will significantly impact medical care.