South Korean biopharmaceutical company Rznomics is set to begin a Phase I/IIa clinical trial of RNA editing therapy, RZ-004, for retinitis pigmentosa.
The move comes after the company obtained a clinical trial notification (CTN) from the Australian Therapeutic Goods Administration (TGA).
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The trial will evaluate RZ-004, a ribozyme-based RNA editing therapy for the treatment of autosomal dominant retinitis pigmentosa with rhodopsin mutation.
RZ-004 utilises an adeno-associated virus (AAV) vector encoding a rhodopsin RNA-targeting trans-splicing ribozyme.
This therapy is designed to target and reprogramme mutant Rhodopsin mRNA into its normal form, potentially offering a therapeutic effect for patients with this genetic disorder.
The therapy’s targeting mechanism is particularly noteworthy as it addresses the conservative upstream region of rhodopsin mutations. This approach could enable the treatment of various mutations within each patient using a single therapeutic strategy.
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By GlobalDataRznomics CEO Dr Seong-Wook Lee said: “The Clinical Trial Notification (CTN) marks an important milestone in addressing the high unmet medical need of autosomal dominant retinitis pigmentosa with rhodopsin mutation.
“Rznomics is committed to developing innovative RNA-based biopharmaceuticals for the treatment of various human rare and intractable diseases, and we look forward to the initiation of the clinical trial to evaluate the safety and efficacy profile of RZ-004 for providing a potential treatment option for patients suffering from the disease.”
Rznomics is engaged in developing RNA-based gene therapeutics for cancer and other incurable diseases.
The company’s core platform technology is based on RNA replacement enzyme, which is known as trans-splicing ribozyme.
In February this year, the company received an orphan drug designation from the US Food and Drug Administration for RZ-001 to treat patients with hepatocellular carcinoma.
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