Ribonova,

RiboNova is a privately-held biopharmaceutical company based in the Lankenau Institute for Medical Research near Philadelphia, Pennsylvania. Our product pipeline includes a Phase-2 ready small molecule drug for the treatment of all forms of genetically-confirmed mitochondrial disease. In addition to this product candidate, we have a proprietary drug discovery platform that targets transfer RNA with novel precision medicines for the treatment of mitochondrial and other diseases. Mitochondrial Disease Burden Genetic mitochondrial disease is a rare disease with high morbidity and mortality ~75,000 people affected in the US (>1 in 4,300); onset can be in childhood or as an adult (35% mortality before age 18); 10% of adults hospitalized for mitochondrial disease die within 5 years. Mitochondrial disease primarily affects the high-energy systems in the human body: Nerves, heart, liver, muscles, kidney, endocrine and respiratory systems. Genetic Diagnosis -DNA sequencing enables the rapid diagnosis of inherited mitochondrial disease - MSeqDR database: 1,628 genes associated with mitochondrial biology - 4,507 variants in these genes associated with mitochondrial disease Significant Impact: The estimated cost of care in the USA is > $10 billion per year. There are no drugs approved by the FDA for the treatment of mitochondrial disease. tRNA DRUG DISCOVERY PROGRAM We have developed a proprietary drug discovery platform for new precision medicines that target a novel family of pharmaceutical targets: defective transfer RNAs. Transfer RNA is the most abundant ribonucleic acid in the human cell, and delivers amino acids to biological factories called ribosomes, to make the proteins that drive all cellular operations. Defects in transfer RNAs are associated with several human diseases. We are applying technology to map RNA modifications in a wide range of human diseases, to identify clinically useful biomarker & powerful therapeutic targets for drug discovery.