Rznomics Inc.

Rznomics gets approval from Clinical Trial Plan for Genetic Retinopathy Rznomics announced on the 8th that it has received a notification from the Australian Federal Drug Administration (TGA) on the completion of the approval process for its phase 1 clinical trial plan for RZ-004, which is being developed as a treatment for hereditary retinal pigment degeneration. In order to conduct clinical trials of gene therapy drugs in Australia, it is necessary to undergo preliminary deliberation from the Office of the Gene Technology Regulator (OGTR) as a preliminary stage for approval of clinical plans. Rznomics completed the procedure in January of this year. This means that all deliberation and administrative procedures for clinical initiation in Australia have been completed. Retinitis Pigmentosa is a disease in which visual cells are damaged and the visual field becomes narrower, resulting in loss of vision. As pigment builds up in the retina, retinal function disappears, which is well known as a rare and incurable disease. It occurs in one out of every 3,500 to 4,000 people worldwide. RZ-004 removes the mutated Rhodopsin ribonucleic acid (RNA) and replaces it with a normal rhodopsin gene using the RNA trans-splicing ribozyme Editing Technology owned by RZ-004 to induce vision damage suppression and vision recovery. In particular, RZ-004 has a groundbreaking feature that can correct more than 150 different rhodopsin mutations known so far with a single treatment. Australia has excellent medical system and medical staff capabilities, the number of target patients, patient compliance, and support from regulatory agencies, said Sung-woo Hong, head of the development division of Rznomics. "We will expand our clinical development countries to the United States and Europe, starting with Australia." The approval of the clinical plan means that it has entered the clinical stage for genetic diseases that can make good use of the characteristics of the company's platform technology following the clinical entry of several anticancer drug pipelines, said Seong-wook Lee, CEO of Rznomics. "Since the target disease of RZ-004 is a disease with very high unmet medical demand without any approved treatment, we will do our best to provide treatment opportunities to suffering patients and improve their quality of life." https://lnkd.in/gzeVwZH3

Rznomics selected for the ‘Deep Tech Incubator Project for Startup’ Development of RNA trans-splicing ribozyme Editing Technology "Will grow into an Innovative Therapeutics Development Company" Rznomics, a new drug development company based on RNA trans-splicing ribozyme editing technology, announced on the 24th that it has been selected as a new company for the 'Deep tech Incubator Project for Startup (DIPS) 1000+'. The 'Deep tech Incubator Project for Startup 1000+' project is led by the Korea Institute of Startup & Entrepreneurship Development under the Ministry of SMEs and Startups. From last year until 2027, the project aims to select and support startups with unique technological superiority in ten key future-leading fields such a bio, healthcare, system semiconductors, and next-generation nuclear power, to foster them into global unicorns. Through this project selection, Rznomics will receive direct business funding of up to 600 million KRW over the next three years, along with linked support for policy funds, guarantees, exports, and specialized support tailored to industry characteristics. Seong-wook Lee, CEO of Rznomics, said, "We are delighted that the selection for the DIPS 1000+ project has once again recognized the innovation and growth potential of Rznomics’ technology. With the support from this selection, we will focus on ongoing clinical trials and technology commercialization to grow into a leading company in developing innovative therapeutics for various rate and intractable diseases. https://lnkd.in/g7emEnEj

Rznomics Signs Clinical Trial Collaboration Agreement With Celltrion for Hepatocellular Carcinoma   Combination of RZ-001, Atezlizumab, and Bevaciuzumab to be evaluated for safety and efficacy in Hepatocelluar Carcinoma clinical trial. Rznomics and Celltrion announced on the 14th that they have signed a clinical collaboration agreement to evaluate the combination of RZ-001 and Bevacizumab for hepatocellular carcinoma patients. Through this partnership, Celltrion will provide Rznomics with Bevacizumab (product name Begzelma) and, in return, will receive the right of first negotiation for the Korean regional license after Rznomics’ RZ-001 Phase 1b/2a clinical trial. Rznomics plans to explore the efficacy and safety of adding its RZ-001 to the combination therapy of Atezolizumab and Bevacizumab, the first-time standard treatment for hepatocellular carcinoma, in a Phase 1b/2a clinical trial. Last November, Rznomics entered into a clinical collaboration agreement with Roche, including the free supply of Atezolizumab (product name: Ticentric). By incorporating RZ-001, which employs its proprietary trans-splicing ribozyme-based RNA reprogramming and editing technology, into the combination therapy of atezolizumab and bevacizumab, Rznomics expects to achieve stronger effects and higher response rates. RZ-001, an RNA trans-splicing enzyme-based cancer gene therapy, targets and cleaves telomerase messenger RNA (hTERT mRNA, replacing it with therapeutic gene RNA. It selectively suppresses hTERT expression in cancer cells expressing hTERT and reprograms the hTERT mRNA to encode HSVtk, inducing cytotoxic effects and anticancer activity.   Preclinical studies have shown that when used in combination with atezolizumab and bevacizumab, RZ-001 significantly enhances anticancer efficacy in a dose-dependent manner.   Sung-woo Hong, vice president and head of development at Rznomics'  expressed confidence that RZ-001 could overcome the limitations of the atezolizumab and bevacizumab combination therapy. Seong-wook Lee, CEO of Rznomics, stated, “We are delighted to collaborate with a global biotechnology company like Celltrion,” and added, “We hope this collaboration will help provide new therapeutic options for patients in need.” Sung-hyun Kim, head of medical affairs at Celltion, also emphasized their commitment to continuous efforts in new drug development through partnerships with biotech companies possessing promising technologies and platforms like Rznomics. https://lnkd.in/gbqJukGb

Rznomics registered a U.S. patent for its original ‘circular RNA' source technology Rznomics announced on the 26th that it has registered a U.S. patent for its foundational technology platform, the ‘self-circularization RNA structure’ which enables efficient and simple production of circular RNA, overcoming the limitations of the existing technologies. Rznomics has also completed applications in Europe, Japan, China, Australia, Canada, Singapore, Israel and Brazil. Circular RNA (circular RNA or circRNA), unlike linear RNA used in mRNA vaccines, has a closed structure, providing superior stability against nucleases. Rznomics has developed a new technology fundamentally different from the existing ‘PIE(Permuted Intron-Exon) method’ based on the conventional ‘Group I Intron ribozyme’ for RNA circularization. While the PIE method leaves unwanted nucleotide sequences in the final product, Rznomics’ RNA circularization technology ensures that specific nucleotide sequences are completely absent from the circular RNA after the self-circularization reaction.   Dr. Kyung-hyun Lee, a senior researcher at Rznomics, stated, "Due to the unique characteristics of our self-circularization mechanism, various designs can enhance self-circularization efficiency compared to the PIE method, resulting in significantly higher self-circularization and purifications yields." Seong-wook Lee, CEO of Rznomics, stated, "In the finally registered patent, we have been granted broad rights by describing the components of the self-circularization RNA structure based on their functions, without limiting them to specific sequences in the examples.” He continued, “With the registration of the U.S. patent, we are actively engaged in research on various applications and indications using circular RNA, in addition to establishing mass production processes through advanced self-circularization and purification efficiency improvements for rapid commercialization." https://lnkd.in/gyw5hpBn

Promising Anticancer Gene Therapy Makes Headway Promising Anticancer Gene Therapy Makes Headway 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. https://lnkd.in/gsvaRKPd

Rznomics Gene Therapy-Based Anti-Cancer Candidate Approved by FDA for New Manufacturing Process Rznomics has changed the manufacturing method of its anti-cancer candidate ‘RZ-001’, which is currently in clinical trials. It has become possible to produce a sufficient amount of clinical drug for high-concentration administration cost-effectively. Rznomics announced on the 6th that it has received approval from the U.S. Food and Drug Administration (FDA) to use the newly manufactured RZ-001 in clinical trials. Changes in the manufacturing method for drugs that have already entered clinical trials under the approval of the IND require approval from regulatory authorities. An Rznomics official said, "By implementing the new manufacturing method, we can obtain a greater quantity of finished pharmaceutical drugs at the same cost, allowing for more efficient conduct of clinical trials for safety and efficacy validation." They added, "The development of the new process was led by Rznomics rather than CDMO." Rznomics' RZ-001 is currently undergoing clinical trials for hepatocellular carcinoma (HCC) and glioblastoma (GBM). Last month, it received Orphan Drug Designation(ODD) from the FDA for the treatment of hepatocellular carcinoma and was granted Fast Track status for glioblastoma. Bo-mi Kim, head of Rznomics’ RA team, said, "From a regulatory perspective, the establishment of the new manufacturing method’s CMC(Chemistry, Manufacturing and Controls) will likely be significantly beneficial when considering Biologics License Application(BLA) submissions." Seong-wook Lee, CEO of Rznomics, also emphasized, "The CMC(Chemistry, Manufacturing and Controls) is a crucial aspect to confirm during discussions with global partners for licensing agreements and it will serve as a highly positive factor from a business perspective." https://lnkd.in/gBCGYcB5

Rznomics "Hepatocellular Carcinoma Drug ‘RZ-001’ Designated as an Orphan Drug by FDA" Phase 1/2a clinical trial in patients with liver cancer that does not respond to transarterial chemoembolization Rznomics (CEO Seong-wook Lee) announced on the 22nd that its developmental cancer treatment, “RZ-001(developmental code name), targeting hepatocellular carcinoma, has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA). With Orphan Drug Designation, the company becomes eligible for benefits such as tax credits for research and development expenses, exemption from approval application fees, and a seven-year market exclusivity period starting from the approval date. The designation is expected to provide favorable conditions during the priority and accelerated review decisions. RZ-001 is currently undergoing Phase 1/2a clinical trials, having received approval for its clinical trial plan from the Ministry of Food and Drug Safety in Korea and the FDA. The trial focuses on patients with hepatocellular carcinoma who do not respond to transarterial chemoembolization(TACE) and aims to develop a treatment option that can be prioritized before using systemic therapies. Seong-wook Lee, CEO of Rznomics, said, "Effective treatments for hepatocellular carcinoma are urgently needed at various stages, and we will do our best to make RZ-001 a new therapeutic option." In addition to the ongoing clinical development, Rznomics has expanded the application of RZ-001 to include glioblastoma, receiving Fast Track designation from the FDA  in November last year. The company is actively engaged in global clinical development through collaborations with pharmaceutical companies, including a clinical partnership with Roche. https://lnkd.in/gYMR_F8Y

Rznomics Launches Clinical Trials in Australia for Genetic Retinal Disease Treatment 'RZ-004' Approved by OGTR in Australia... Plans to expand clinical development countries in the future Rznomics (CEO Seong-wook Lee) has announced on January 16th that it has received approval for the review of the phase 1 clinical trial for ‘RZ-004’ in Australia, a therapeutic candidate for genetic retinal diseases, including Retinitis Pigmentosa. The review is being conducted by the Office of the Gene Technology Regulator (OGTR) in Australia. Retinitis Pigmentosa is a rare and incurable genetic retinal disorder where visual cells progressively deteriorate, leading to a narrowing field of vision and eventual vision loss. It occurs globally at a rate of approximately 1 out of every 3,500 to 4000 people. ‘RZ-004’ aims to eliminate mutated Rhodopsin RNA by using ‘Trans-splcing ribozyme’, a RNA correction platform technology owned by Rznomics. Notably, Rznomics explains that its technology can correct over 150 different known mutations with a single therapeutic agent. Sung-woo Hong, the Head of Development at Rznomics, stated, “Australia possesses elements conducive to the early clinical development of genetic ocular diseases, such as an excellent healthcare system, medical expertise, patient numbers, patient compliance and regulatory support.” He further mentioned plans to expand clinical development to the United States and Europe, with Australia as the starting point.   Seong-wook Lee, the CEO of Rznomics, emphasized the significance of ‘RZ-004’ by highlighting the lack of approved treatments and the scarcity of ongoing therapeutic developments for the targeted condition. He expressed the company’s commitment to providing treatment opportunities for patients suffering from diseases and enhancing their quality of life through swift development efforts. https://lnkd.in/gJegzSRF

Rznomics’s Liver Cancer Treatment, Clinical trials in combination with immuno-oncology drugs Rznomics, a gene therapy development company, announced that its Phase 1b/2a clinical trial plan (IND) for the administration of liver cancer drug candidate “RZ-001” along with immune-oncology drugs has been recently approved by the Ministry of Food and Drug Safety. Rznomics plans to evaluate the efficacy and safety of “RZ-001” as a first-line treatment in about 50 patients diagnosed with hepatocellular carcinoma along with the immune-oncology drug ‘Ticentric’ and the targeted therapy ‘Avastin’. ‘Ticentric’ and ‘Avastin’ are Roche’s most widely used anti-cancer drugs for the first-line treatment of hepatocellular carcinoma. Roche will provide Rznomics for the ‘Ticentric’ to be used in this clinical trial. RZ-001 received MFDS and FDA clinical trial approval for HCC and GBM as a single administration therapy. Rznomics is conducting an independent clinical trial of RZ-001 in patients with hepatocellular carcinoma. https://lnkd.in/gFpM2D9U