Pharma Focus Asia

Rznomics Received FDA Approval to Initiate Clinical Development of Trans-splicing ribozyme-based RNA Editing Technology

Friday, May 19, 2023

Rznomics received Phase 1/2a IND approval from the U.S FDA for its Glioblastoma Multiforme (GBM) treatment called RZ-001.

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 tumours and hampers angiogenesis in the tumour tissues in preclinical animal models.

Being the first U.S. FDA-approved ribozyme-based RNA reprogramming approach to be evaluated in patients, RZ-001, a gene therapy approach utilising the proprietary trans-splicing ribozyme-based RNA reprogramming and editing technology, is a replication-incompetent adenoviral vector that expresses an hTERT targeting ribozyme with multiple additional MoA to treat GBM patients.

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