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Anticancer Gene Therapy Alliance Aims to Advance Survival Rates
Hepatocellular carcinoma is the most prevalent type of primary liver cancer, accounting for 80% of cases globally
South Korea-based Rznomics is developing a pioneering RNA-based gene therapy, RZ-001, which targets primary liver cancer, also known as hepatocellular carcinoma (HCC).
The potentially curative treatment utilizes a proprietary trans-splicing, ribozyme-based RNA reprogramming and editing technology and takes the form of a replication-incompetent adenoviral vector that expresses an hTERT targeting ribozyme with multiple additional MoA. The aim is to produce higher treatment response rate with fewer side effects, ultimately improving survival rates for advanced liver cancer patients.
RZ-001 received IND approval from the US FDA in October, allowing Rznomics to commence an international clinical study in HCC patients. “We are proud to have achieved FDA approval to begin Phase I/IIa trials and excited to take this next step with Charles River,” commented Seong-wook Lee, President and Chief Executive Officer, Rznomics. “A reliable and experienced manufacturing partner is of utmost importance, and we are keen to continue building this relationship to enable us to bring our leading pipeline, RZ-001, into clinical development.”
Leveraging Charles River’s significant adenoviral vector contract development and manufacturing (CDMO) experience, this alliance will enable the initiation of clinical-stage development of RZ-001, the first FDA-approved ribozyme-based RNA reprogramming approach to be evaluated in patients.
Extensive Viral Vector Track Record
With over 20 years of viral vector CDMO expertise and a validated platform process, our team has established standardized protocols for cell culture, upstream production, and downstream purification. These high-yield, optimized methods increase speed to clinic, reducing process development time and costs while ensuring the highest quality production.
In a press release announcing the gene therapy manufacturing alliance, Birgit Girshick, Charles River Corporate Executive Vice President and Chief Operating Officer, also remarked: “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.”
For more information on our cell and gene therapy development and manufacturing services, please visit: Cell and Gene Therapy CDMO Solutions.
Charles River launches nAAVigation® Vector Platform
This new platform provides AAV vector gene therapy developers an expedited, cost-effective, and predictive pathway to GMP manufacturing, with the capability to reduce timelines by 55% or in fewer than 8 months compared to traditional process development.
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