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November 6–8, 2023Munich, Germany



Rznomics has core competencies to meet a variety of unmet medical needs through development of gene therapeutic biopharmaceuticals based on  RNA platform technology for human intractable diseases including liver cancer, intractable cancers, degenerative diseases and genetic diseases. 

-Platform technology of Rznomics : RNA replacement/repair by group I intron-based trans-splicing ribozyme

-Brief description of the platform : 

A ribozyme can recognize target RNA at accessible uridine residue by base pairing to the sequence through its IGS (internal guide sequence). The ribozyme then removes the sequence downstream of the target site and replaces it with a 3’ exon that encodes a therapeutic RNA sequence.

Rznomics can select the optimal target site on target RNA through RNA mapping technology. In addition, the trans-splicing ribozyme system was optimized through modification of ribozyme and improvement of expression system.

-Key advantage: 

• Targeting of non-druggable target via specific targeting of disease-associated/related RNA

• Multi-function of one molecule; Inhibition of target RNA expression and disease-specific induction of therapeutic gene expression by one molecule

• Regulation of therapeutic gene expression in proportion to target RNA level

• Maintenance of intracellular therapeutic gene expression via DNA vector as tool

• Minimization of genome toxicity and eternal genome change through gene editing at RNA level

• Increase specificity of therapy by therapeutic gene expression only in the cells expressing the target RNA

• Increase efficacy of therapy by removing target RNAs which reduce gene activity and simultaneously triggering therapeutic RNA in targeted cells

• Can be modularly engineered → Can expand indications for various types of RNA-associated diseases.