RZNOMICS Inc. received FDA approval to initiate clinical development of trans-splicing ribozyme-based RNA editing technology in Glioblastoma Multiforme Patients

More specifically, RZ-001 engenders effective anti-GBM activity by suppressing hTERT expression selectively in cancer cells, which over-express hTERT, and simultaneously inducing a cytotoxic effect by trans ligating an HSVtk-encoding sequence into the reprogrammed hTERT mRNA. Moreover, the result of such editing efficiently induces immune cell infiltrations into GBM tumors and hampers angiogenesis in the tumor tissues in preclinical animal models. (http://www.rznomics.com/pipeline/RZ-001.php). The Phase 1/2a clinical trial will be a dose escalation/expansion study to assess the safety and tolerability of RZ-001 and to determine the most effective dose with the least toxicities of RZ-001 in recurrent GBM patients without extracranial metastases. 

More specifically, RZ-001 engenders effective anti-GBM activity by suppressing hTERT expression selectively in cancer cells, which over-express hTERT, and simultaneously inducing a cytotoxic effect by trans ligating an HSVtk-encoding sequence into the reprogrammed hTERT mRNA. Moreover, the result of such editing efficiently induces immune cell infiltrations into GBM tumors and hampers angiogenesis in the tumor tissues in preclinical animal models. (http://www.rznomics.com/pipeline/RZ-001.php). The Phase 1/2a clinical trial will be a dose escalation/expansion study to assess the safety and tolerability of RZ-001 and to determine the most effective dose with the least toxicities of RZ-001 in recurrent GBM patients without extracranial metastases. 

"It's a monumental achievement of Rznomics that RZ-001, the first trans-splicing ribozyme therapy at the front of our therapeutic pipeline, has successfully received another IND approval in the United States with the indication for the GBM. I am really grateful that RZ-001 earned the opportunity to potentially fulfill the unmet needs of GBM patients. Through the advanced development phase, I hope Rznomics can provide more new therapeutic options to patients suffering from intractable diseases. Rznomics will further expand our pipeline by targeting indications with highly unmet medical needs for which the unique characteristics of our platform technology may be the most competitively applied," said Dr. Seong-Wook Lee, Ph.D., CEO, and founder of Rznomics.

In addition to the HCC & GBM, Rznomics is developing ribozyme-based RNA editing treatments for Alzheimer's disease (RZ-003) and inherited retinal dystrophies, called Retinitis pigmentosa (RZ-004). 

Contact
rznomics_bd@rznomics.com

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