RIBOZYME AND OMICS
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Rznomics announced on the 14th that they signed an MOU for joint research with the University of Missouri in the United States.Under the agreement, two sides will cooperate in all areas of research for the development of gene therapy products, including ribonucleic acid (RNA) editing, establishment of human gene transduction animal models, preclinical trials and clinical research.The MOU is expected to promote mutual exchange of human and material resources and technological development.The University of Missouri, established in 1839, is a state university that is prominent in the fields of agriculture, veterinary medicine, medicine, and pharmacy. Which recently, they shared their interest in gene therapy research.Seongwook Lee, CEO of Rznomics, stated, "By combining the RNA editing platform technology possessed by Rznomics and the research infrastructure of the University of Missouri, we will be able to strengthen our competitiveness in the field of RNA-based gene therapy research. We will leap forward as a global gene therapy company.”
Rznomics Inc. shared its platform technology and program development progressRznomics revealed its platform technology and program development progressSeongwoo Hong, vice president & head of development department made a presentation on '2022 LOG IN IR EXPO 100' held at the FKI Hall in Yeouido.Rznomics develops RNA-targeting drugs using an RNA replacement enzyme platform.Rznomics' platform uses a ribozyme that causes trans-splicing and a therapeutic transgene that encodes a therapeutic effect inducer.It is a method in which DNA linked to a ribozyme and a therapeutic gene is delivered into the cell using a vector such as adenovirus, and drug RNA transcribed from the cell targets the target RNA.A ribozyme specifically binds to a target RNA and then replaces a portion of the target RNA with a therapeutic gene RNA by trans-splicing method. The function of the target disease RNA is lost and the protein encoded by the therapeutic gene is expressed.Global drug development based on trans-splicing is still in its infancy, and Ascidian Therapeutics, which develops RNA-targeted therapeutics using trans-splicing, announced its $50 million Series A funding last month.
'Pros and Cons of In Vitro Methods for Circular RNA Preparation' by Ph.D. Kyung Hyun Lee, Researcher Seongcheol Kim, Ph.D. Seong-wook Lee, of Rznomicshas been published in the 'International Journal of Molecular Sciences.'
[CEO Interview] Rznomics aims to serve two ends via RNA replacementOriginal article: hankyung BIO Insight October 2022
SEONGNAM, South Korea, Oct. 25, 2022 /PRNewswire/ -- Rznomics Inc., a South Korea based biopharmaceutical company specialized in the development of RNA-based gene therapeutics, recently received Phase 1/2a IND approval from the U.S FDA in October 10th for its hepatocellular carcinoma (HCC) treatment called RZ-001 and thus has achieved an important milestone for the company and the RNA editing field. Being the first U.S. FDA-approved ribozyme-based RNA reprogramming approach to be evaluated in patients, RZ-001, a gene therapy approach utilizing the company's proprietary trans-splicing ribozyme-based RNA reprogramming and editing technology, is a replication-incompetent adenoviral vector that expresses an hTERT targeting ribozyme with multiple additional MoA to treat HCC patients. Rznomics also received IND approval of RZ-001 from the South Korean Ministry of Food and Drug Safety this June and already initiated a phase 1/2a clinical trial in Korea. Therefore, U.S. FDA approval allows Rznomics to start an international clinical study in HCC patients treating them with RZ-001 and therapeutic RNA editing. The trans-splicing ribozyme is derived from the self-splicing Tetrahymena group I intron, which both recognizes and reprograms the target RNA into the therapeutic transcript of interest. Ribozyme-based RNA editing technology developed by Rznomics has unique features, differentiating it from other nucleic acid-based editing approaches, as follows: (1) A single RNA molecule is capable of both suppressing target RNA expression and simultaneously expressing a therapeutic RNA. Thus, no potentially antigenic proteins or cofactors are required. (2) Safety can be improved by selectively inducing therapeutic RNA expression only in cells/tissues where the target gene is expressed. (3) Therapeutic gene expression can be regulated proportionally to endogenous cellular target RNA levels. (4) Editing occurs at the RNA level, not the genomic level, thus eliminating concerns about genomic toxicity and eternal genome changes. (5) Indications with multiple mutation sites scattered throughout a target RNA can be edited with a single RNA designed to react upstream of all mutations and by replacing and editing large stretches of RNA. (6) Additional safety can be conferred by building control mechanisms into the ribozyme itself, without the need to modulate intrinsic cellular mechanisms or external proteins. More specifically, RZ-001 engenders effective anti-HCC activity by suppressing hTERT expression selectively in cancer cells, which over-express hTERT, and simultaneously inducing a cytotoxic effect by trans ligating an HSVtk-encoding sequence into the reprogrammed hTERT mRNA. Moreover, the result of such editing efficiently induces immune cell infiltrations into HCC tumors in preclinical animal models. (http://www.rznomics.com/pipeline/RZ-001.php). The Phase 1/2a clinical trial will be a dose escalation/expansion study to assess the safety and tolerability of RZ-001 and to determine the most effective dose with the least toxicities of RZ-001 in HCC patients with no extrahepatic metastasis. "The translation of the first trans-splicing-based RNA editing approach into an FDA-approved phase 1/2a clinical trial is an exciting achievement and a critical milestone for the RNA editing field. I am very excited about Rznomics' preclinical progress on ribozyme design and gene delivery optimization. The advances have allowed them to create and now translate a promising therapy, RZ-001, into the clinic. I and the entire editing field are eager to learn if RZ-001 and mRNA reprogramming is safe and able to combat hepatocellular carcinoma in patients. Rznomics is clearly a leader at bringing novel editing strategies to cancer patients that desperately need innovative, breakthrough therapies," said Dr. Bruce Sullenger, Joseph, and Dorothy Beard Professor of Surgery at Duke University. Dr. Sullenger is a scientific advisory board member of Rznomics and the initial pioneer developing approaches to therapeutically edit RNA and DNA using RNA guided endonucleases (RGENs) such as the group trans-splicing I ribozyme.. "It's a monumental achievement of Rznomics that RZ-001, the first trans-splicing ribozyme therapy at the front of our therapeutic pipeline, has successfully received the IND approval in both Korea and the United States. I am really grateful that RZ-001 earned the opportunity to potentially fulfill the unmet needs of HCC patients. Through the advanced development phase, I hope Rznomics can provide more new therapeutic options to patients suffering from intractable diseases. Rznomics will further expand our pipeline by targeting indications with highly unmet medical needs for which the unique characteristics of our platform technology may be the most competitively applied." said Dr. Seong-Wook Lee, CEO and founder of Rznomics.In addition to the HCC, Rznomics is expanding the indication of RZ-001 to glioblastoma multiforme and planning to submit the IND this year. Also under development are ribozyme-based RNA editing treatments for Alzheimer's disease (RZ-003) and inherited retinal dystrophies, called Retinitis pigmentosa (RZ-004).
Rznomics announced on october 11th,2022 that it had received approval for phase 1 and 2 clinical trial (IND) of 'RZ-001' from the US Food and Drug Administration (FDA).The clinical trial plan was approved by the Ministry of Food and Drug Safety in June,2022 and is an extension of the ongoing domestic clinical trial.Rznomics has the technology to express the desired gene while removing the target ribonucleic acid (RNA) based on RNA trans-splicing ribozyme.RZ-001 targets telomerase (hTERT) RNA specifically expressed in cancer cells by delivering a ribonucleic acid substitution enzyme to an adenovirus vector.At the same time, it is a mechanism for expressing genes that induce anticancer activity.Rznomics is pushing to expand indications to other carcinomas, and is planning a clinical trial for malignant glioblastoma within this year.In addition to the anticancer drug pipeline, Rznomics is developing therapeutics in various fields using platform technology, such as Alzheimer's treatment, hereditary retinopathy, and Rett's syndrome.Seongwook Lee, CEO of Rznomics, said, "The pipelines under development are scheduled to begin clinical trials in korea and abroad sequentially, and we will do our best to provide new treatment opportunities to patients."
announced on the 16th that it won the most promising pipeline award in the gene
therapy category at the "Asia-Pacific Cell & Gene Therapy Excellence
Awards" (ACGTEA) of the "Cell & Gene Therapy World Asia
2022" (CGTWA 2022) held in Singapore.
is an event to share representative technologies of cell and gene therapy.
the conference which is followed after the award, the "next-generation
cell gene therapy" session was announced.
the presentation, Rznomics explained the current status of candidate substances
(pipelines) including trans-splicing ribozyme based gene therapy platform
technology and liver cancer therapy currently undergoing clinical trials. As
a company with first-in-class platform technology, it presented technological
innovation and vision.
plans to apply for clinical trials for glioblastoma patients in the United
States and Korea in the first quarter of next year. In the second half of next
year, the U.S. is also planning to perform clinical trials on hereditary
Lee, CEO of Rznomics, said, "This award will be a great help to executives
and employees who have a desire to develop new drugs and those who supported
Rznomics," adding, "We will do our best to achieve better
Rznomics announced on the 9th that it has applied to
the U.S. Food and Drug Administration (FDA) for a phase 1/2a clinical trial of
the hepatocellular cancer therapy "RZ-001".
is an extension of the domestic clinical trial that was approved by the
Ministry of Food and Drug Safety in June and is being submitted for the
originally planned multinational clinical trial.
on RNA trans-splicing ribozyme, Rznomics is developing a technology that
removes target RNA and simultaneously expresses the desired gene.
is a mechanism that targets the telomerase (hTER) RNA that is specifically
expressed by cancer cells by transferring ribonucleic acid substitution enzymes
through the adenovirus vector and expresses genes that induce anticancer
is also pushing to expand indications to other cancer species, and especially plans
to perform clinical trials on malignant glioblastoma within this year.
addition to the anticancer drug pipeline, it is developing therapy in
various fields using platform technologies such as Alzheimer's therapies,
hereditary retinopathy, and Rett’s syndrome.
retinal pigmentosa was registered with the Korean Intellectual Property Office
in May, followed by a notification of the registration decision from the U.S. Patent
Office in August to prove its technological progress, the company added.
Lee, CEO of Rznomics, said, "The pipelines under development will be
launched sequentially at home and abroad, and we will do our best to provide
patients with new therapy opportunities."
CEVEC’s license partner RZNOMICS Inc. receives approval to initiate clinical development of CAP® Ad Technology-produced gene therapy vector in liver cancer patientsRZNOMICS uses CEVEC’s CAP® Ad viral vector manufacturing technology for manufacturing gene therapy vectors targeting various cancer indicationsSouth Korean Ministry of Food and Drug Safety granted IND approval for a clinical phase 1/2a trial to evaluate safety and efficacy of RZNOMICS lead gene therapy RZ001 for the treatment of primary liver cancerAnother important milestone has been reached for CEVEC’s unique CAP® cell line which enables by design the efficient production of high-quality, RCA-free adenoviral vectorsCologne, Germany, July 13, 2022CEVEC Pharmaceuticals GmbH (CEVEC) today announced that an important milestone in the partnership with RZNOMICS, Inc. (RZNOMICS) was achieved when the South Korean Ministry of Food and Drug Safety granted IND (Investigational New Drug) approval to initiate a phase 1/2a clinical trial with RZNOMICS’ lead candidate RZ001. RZ001, a gene therapy based on the company’s proprietary trans-splicing ribozyme technology and being developed for the treatment of primary liver cancer, uses CEVEC’s CAP® Ad Technology to manufacture RCA-free adenoviral vectors for the efficient and safe drug delivery to the target cells. With this regulatory clearance, RZNOMICS plans to submit an IND to the U.S. Food and Drug Administration (FDA) in the second half of this year.“We are delighted to see RZNOMICS advance its lead compound, a gene therapy produced using our CAP® Ad Technology, into clinical development,” said Dr. Nicole Faust, CEO of CEVEC. “By providing a unique technology that enables the manufacturing of safe adenoviral vectors by eliminating the generation of replication competent adenovirus (RCA), we help our partners to address one of the major challenges in adenovirus-based gene therapy and vaccine production. We look forward to continuing the work with RZNOMICS and supporting them through clinical development and further onto the market.”“This is a monumental achievement of administering a therapeutic substance using ribonucleic acid splicing enzyme technology to patients with primary liver cancer for the first time in the world”, said Seong-Wook Lee, Ph.D., CEO of RZNOMICS. “The advantages of the CAP® Ad Technology in terms of safety and scale up convinced us to select it as the technology to produce our gene therapy candidate based on adenoviral vector for the clinical purporse”, he added.In 2020, CEVEC and RZNOMICS signed a license agreement for the use of CEVEC’s CAP® Ad Technology for manufacturing of adenoviral vectors in combination with RZNOMICS’ proprietary development portfolio of gene therapies targeting various cancer indications.About CAP® Ad TechnologyThe CAP® Ad Technology is an innovative technology platform developed by CEVEC for the scalable production of RCA-free adenoviral vectors for gene therapy applications and vaccines.Recombinant adenoviral vectors are among the most efficient vectors for gene therapy purposes and have become the vehicle of choice in many human gene therapies. Today, many cell lines used for production of adenoviral vectors generate certain levels of replication-competent adenovirus (RCA). The presence of RCA in adenoviral vector preparations which are intended for use in humans is increasingly considered to be a potential risk, especially for immuno-compromised patients. The CAP® cell line is specifically designed to avoid the production of RCA.CEVEC’s CAP® cell line is based on an engineered human suspension cell line of non-tumor origin, derived from human amniocytes. CAP® cells can be grown in all formats and all sizes of bioreactors, providing a robust, fully scalable production platform for the manufacturing of adenoviral vectors from research grade and smaller amounts up to industrial volumes. The CAP® cell line has been fully documented and reviewed by regulatory authorities. Since 2016, a Biologics Master File has been available for reference with the U.S. FDA. GMP Master Cell Banks are available and ready for licensing.About CEVECCEVEC is a leading provider of high-performance cell technology for the manufacturing of advanced biotherapeutics.With the ELEVECTA® Technology, CEVEC offers a unique solution for large-scale production of AAV vectors using helper virus-free inducible producer cell lines with all necessary components stably integrated into the cell. The technology is based on suspension cells and does not require any expensive transfection reagents nor cGMP plasmids. CEVEC’s CAP® Ad Technology is the ideal production platform for RCA-free adenoviral vectors. Based on human suspension cells, it allows for a robust manufacturing process, easy scale-up from research grade to industrial volumes and thus opens the way for various applications, from gene therapy to vaccine production.About Rznomics:Rznomics is a biopharmaceutical company founded in 2017 dedicated to the development of gene therapies for cancers, degenerative diseases, and genetic diseases based on cutting-edge RNA technology. Core platform technology of Rznomics is based on RNA replacement enzyme called ‘trans–splicing ribozyme’, which can edit target RNAs through simultaneous destruction and repair (and/or reprograming) to yield the desired therapeutic RNAs, thus selectively inducing therapeutic gene activity in cells expressing the target RNAs. Rznomics has developed and optimized the ribozymes to be applied as therapeutics for intractable human disease by developing them to have high target specificity and efficacy, target accuracy, and minimal off-target ability. Rznomics has established pipelines targeting indications with high unmet medical demand for which the unique properties of the ribozymes can be the most competitive. The leading candidate is treatment for hepatocellular carcinoma, and treatments for glioblastoma, Alzheimer’s disease and hereditary retinal dystrophy are also under development. For more information, please visit www.rznomics.com.