Charles River Laboratories (CRL) International and Rznomics Inc. have announced a contract development and manufacturing organization (CDMO) partnership around liver cancer. CRL has recently taken major steps, including investing about $1B, to be able to offer a comprehensive cell and gene therapy development platform.
A South Korea-based biotech, Rznomics specializes in RNA-based gene therapeutics. Its lead product is RZ-001 for hepatocellular carcinoma (HCC), the most common cause of primary liver cancer.
“We are proud to have achieved FDA approval to begin Phase I/IIa trials and excited to take this next step with Charles River,” said Seong-wook Lee, president and CEO, Rznomics.
Over the last couple of years, Charles River beefed up its CDMO offerings with key acquisitions including Cobra Biologics, Cognate BioServices, and Vigene Biosciences, which specializes in viral vectors.
At the time of the Vigene acquisition, James C. Foster, Chairman, President and Chief Executive Officer of Charles River Laboratories, said, “The addition of Vigene Biosciences’ extensive gene therapy expertise will enable us to expand our comprehensive cell and gene therapy portfolio to span each of the major CDMO platforms—cell therapy, viral vector, and plasmid DNA production.”
CRL also recently opened a plasmid production facility in the U.K., and expanded its cell therapy manufacturing facility in Memphis, Tennessee. All this investment seems to be paying off, in October of last year CRL signed an agreement around its CDMO services with Nanoscope for work on retinal degenerative diseases.
RZ-001 is the first ribozyme-based RNA reprogramming approach approved by the US FDA for evaluation in patients. The treatment was developed using Rznomics’ proprietary RNA reprogramming and editing technology and uses an adenoviral vector that expresses an hTERT targeting ribozyme to treat HCC, which accounts for 80 percent of primary liver cancer cases worldwide.
Rznomics received Phase I/IIa IND approval for RZ-001 from the FDA last October, allowing the initiation of an international clinical study in HCC patients. Early-phase trials have also commenced in Korea following IND approval for RZ-001 from the South Korean Ministry of Food and Drug Safety (MFDS), formerly the Korea Food & Drug Administration (KFDA), in June.
CRL has standardized protocols for cell culture, transfection, and downstream purification, as well as a validated platform process. CRL says it offers end-to-end support and supply chain simplification for advanced therapy medical product developers.
“This collaboration with Rznomics will tap into our industry-leading CDMO capabilities and we are thrilled that our expertise will help to bring RZ-001, a potentially curative therapy, to HCC patients,” said Birgit Girshick, Corporate Executive Vice President and Chief Operating Officer, Charles River Laboratories.
Founded in the laboratory of Professor Seong-wook Lee, Dankook University Department of Bio-convergence Engineering, Rznomics’ core platform technology uses an RNA replacement enzyme, known as a trans–splicing ribozyme. This enzyme can edit target RNA through simultaneous destruction and repair (and/or reprogramming) to yield the desired therapeutic RNA, thus selectively inducing therapeutic gene activity in cells expressing the target RNA.
Besides its lead candidate for HCC, the company also has treatments for glioblastoma, Alzheimer’s disease, and hereditary retinal dystrophy (RD) under development.