FDA designations underscore the potential clinical impact of a pioneering RNA-based gene therapy
Rznomics, a South Korea-based biopharma, recently announced two significant advancements on their pathway to develop a novel anticancer therapy. Their lead clinical candidate, RZ-001, a viral vector engineered to deliver a proprietary RNA-based trans-splicing ribozyme, is produced leveraging Charles River’s adenoviral vector contract development and manufacturing (CDMO) experience.
RZ-001 has the potential to treat several cancer types due to its multi-faceted mechanism of action. The expressed ribozyme is directly cytotoxic against cancer cells by downregulating expression of hTERT and replacing it with an HSV-TK sequence. Additionally, Rznomics has preclinical data indicating that treatment with RZ-001 can provoke an antitumor immune response in animal models.
Both the master virus bank and RZ-001 clinical product have been manufactured at Charles River’s viral vector production site in Rockville, MD. “The needs of the program have changed over time, but we’ve been able to adapt the manufacturing process accordingly,” notes James Cody, PhD, Associate Director, Technical Sales and Evaluations. “We will continue to adapt as needed in order to meet the increased clinical need in the future”.
Previously approved for Phase I/IIa clinical trials for hepatocellular carcinoma (HCC) in both the US and South Korea, RZ-001 was granted Orphan Drug designation by the FDA, providing access to regulatory assistance from the Office of Orphan Products Development, and has also shown promise against other cancer types.
On the Fast Track
RZ-001 was also recently granted FDA Fast Track designation for the treatment of a type of brain tumor known as glioblastoma multiforme (GBM), the most common malignant brain and central nervous system (CNS) tumors accounting for around half of all cases.
The gene therapy has been approved for Phase I/IIa trials for GBM treatment in both the U.S. and South Korea, and this significant designation allows for an expedited review process to improve access to patients in critical need.
"We are proud to receive Fast Track designation from the FDA. This signifies an important milestone in developing a safe and effective treatment for patients with GBM who require new therapeutic options," commented Dr. Seong-Wook Lee, Chief Executive Officer, Rznomics.
This latest announcement highlights the opportunity for RZ-001 to make a clinical impact for patients with HCC, GBM, and potentially other types of cancer in the future. For more information on our advanced therapy development and manufacturing services, visit Cell and Gene Therapy CDMO Solutions.
