#South Korea #Rznomics #RZ-001 #Glioblastoma #Seongnam

Rznomics Initiates Expanded Access Program for RZ-001

Rznomics, Inc. has recently secured the Expanded Access Program (EAP) approval from the United States Food and Drug Administration (FDA) for its groundbreaking RNA editing gene therapy, RZ-001. This innovative treatment is aimed at patients aged 18 and older diagnosed with Glioblastoma (GBM), a highly aggressive form of brain cancer.

Understanding the Expanded Access Program

The FDA's EAP, also known as 'compassionate use', serves as a vital pathway that provides patients facing serious or life-threatening conditions with access to investigational medical products, such as drugs, biologics, or medical devices, outside of the constraints of clinical trials. This program is crucial for those patients who have exhausted all standard treatment options and are in dire need of alternatives.

GBM is notorious for being one of the deadliest tumors within the central nervous system, marked by a notably high mortality rate, and unfortunately, it is often resistant to existing treatments. Statistically, TERT promoter mutations, which lead to an upregulation of TERT expression, are present in up to 80% of GBM cases. This overexpression correlates with a poor prognosis, emphasizing the urgent need for effective therapeutic solutions.

RZ-001's Mechanism of Action

RZ-001 operates as an innovative RNA replacement enzyme-based cancer gene therapy. It specifically targets and degrades hTERT mRNA, subsequently replacing it with a therapeutic RNA. This mechanism not only reduces hTERT expression, which is critical for the tumor's survival and proliferation but also initiates an anti-cancer response that can lead to cytotoxic effects against tumor cells. The therapy simultaneously integrates a sequence encoding HSVtk into the reprogrammed hTERT mRNA, further enhancing its therapeutic potential.

Dr. Chiocca, a Professor at Harvard Medical School and the Executive Director of the Center for Tumors of the Nervous System at Mass General Brigham Cancer Institute, expressed optimism regarding the launch of RZ-001 through the EAP, highlighting that it provides a promising new treatment avenue for GBM patients who often have limited options.

Commitment to Patients

Seong-Wook Lee, PhD, CEO of Rznomics, echoed Dr. Chiocca's sentiments, emphasizing the company's commitment to patients who have struggled with existing treatments. He reassured that the Rznomics team is dedicated to accelerating the clinical development process to secure timely market authorization for RZ-001.

In their prior announcements, Rznomics revealed that RZ-001 has already received Fast Track designations and Phase I/IIa IND approval from both the FDA and the South Korean Ministry of Food and Drug Safety (MFDS) specifically for GBM. A clinical trial is currently underway, marking the beginning of RZ-001’s administration to patients enrolled in the study.

About Rznomics

Rznomics is a biopharmaceutical company rooted in innovative research from the laboratory of Professor Seong-Wook Lee at Dankook University’s Department of Bioconvergence Engineering. The company’s mission revolves around the development of novel RNA-based gene therapeutic bio-drugs aimed at tackling cancer and other incurable diseases. Rznomics specializes in a core platform technology based on an RNA replacement enzyme known as trans-splicing ribozyme. This technology allows for targeted RNA editing through simultaneous degradation and repair, effectively yielding the desired therapeutic RNA and selectively activating therapeutic gene activity within relevant cells.

For further information about Rznomics and its promising research initiatives, visit Rznomics' website.