CSO: Dr. Amir Mor
RNAble is a preclinical biotechnology company developing a potent small-molecule inhibitor targeting FTO, a key driver of oncogenic RNA biology. Mechanistic studies have led to the identification of a specific biomarker that predicts pan-cancer sensitivity to the inhibitor across both hematological and solid malignancies. RNAble’s lead candidate has demonstrated superior anti-tumor activity compared to standard therapies in AML models, and efficacy studies in multiple solid tumor models are ongoing and expected to be completed by end of Q3, 2025.
SCIENTIFIC BACKGROUND
Our FTO m⁶A RNA demethylase inhibitor selectively induces cytotoxicity in biomarker-selected cancers while sparing normal cells. In these sensitive cancer types, FTO inhibition triggers dose-dependent chromosome misalignment during mitosis, leading to apoptotic cell death.
INDICATIONS
RNAble’s FTO inhibitor has demonstrated strong preclinical activity in biomarker-defined subsets of acute myeloid leukemia (AML) and selected solid tumors, including lung, cervical, hepatocellular, clear cell renal (ccRCC), bladder, and ovarian cancers. These indications were prioritized based on biomarker expression, FTO dependency, and observed sensitivity to FTO inhibition, positioning RNAble’s lead compound as a targeted therapeutic candidate across both hematological and solid malignancies.
Co-Founder and CSO
Dr. Amir Mor: Dr. Mor holds a Ph.D. in molecular and cell biology from Bar-Ilan University, Israel, where he researched mRNA processing and nucleo-cytoplasmic transport. He subsequently pursued postdoctoral training at UT Southwestern, Dallas, TX, working on host pathogen interactions essential for influenza virus RNA processing. Prior to initiating RNAble Dr. Mor served as a senior scientist at LungLife AI, Thousand Oaks, CA, developing assays for analysis of circulating tumor cells and at Anima Biotech, Tel-Aviv, as the assay development team leader focusing on RNA translation.
INVENTOR AND ADVISOR
Prof. Rotem Karni: Prof. Karni recently joined the Perelman School of Medicine at the University of Pennsylvania as an Associate Professor of Genetics. Before that, he was the Chair of the Biochemistry and Molecular Biology Department at the Hebrew University-Hadassah Medical School. Prof. Karni holds a Ph.D. in Biological Chemistry from the Hebrew University, Israel, and completed postdoctoral training in RNA splicing regulation in the lab of Prof. Adrian Krainer at Cold Spring Harbor Laboratory, NY. His lab focuses on the role of RNA processing in human diseases, particularly cancer initiation and progression, and on developing technologies to therapeutically modulate RNA processing in genetic diseases and cancer.