CSO: Dr. Amir Mor
RNAble is developing novel compounds that alter RNA stability through modulation of RNA epitranscriptomics for the treatment of specific cancers and rare genetic diseases with nonsense mutations.
RNA transcripts undergo modifications that are recognized by specific RNA binding proteins dictating their stability. Prof. Rotem Karni, RNAble inventor, discovered that targeting components of the RNA epitranscriptomic machinery with small molecules resulted in stabilization of several RNAs, including unstable nonsense mutated transcripts which are degraded by the nonsense mediated decay machinery (NMD). This opens the possibility to restore the expression of mutated genes essential in rare diseases and to suppress the growth of specific cancers.
NMD dependent cancers: Several malignancies, including specific AML and lung adenocarcinoma are dependent on NMD function to eliminate aberrant transcripts. Thus, blocking mRNA degradation by our molecules enhance the accumulation of cytotoxic mRNA transcripts in these tumor cells, resulting in their destruction.
Rare Diseases: The global prevalence of single-gene disorders is 10/1000 births, affecting more than 75,000,000 people world-wide. Approximately 11% of the mutations causing diseases are nonsense mutations which cause degradation of the mutant mRNA by the NMD machinery. RNAble is developing molecules that enhance the stability of mRNAs containing nonsense mutations with the goal of enhancing the production of functional, or semi-functional, proteins essential in specific rare diseases.
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 is the chair of the Biochemistry and Molecular Biology Department at the Hebrew University-Hadassah Medical School. He 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. Prof. Karni’s lab focuses on the role of RNA processing in human diseases, especially on cancer initiation and progression, and the development of technologies to modulate RNA processing as therapy for genetic diseases and cancer.