RIBOZYME AND OMICS
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The National New Drug Development Project Group (Director Muk Hyun-sang) held the 2023 National New Drug Development Project R&D Workshop at the Yeoju Sun Valley Hotel on February 2 and 3.About 160 researchers who participated in the newly selected R&D institution under the National New Drug Development Project in 2022 identified the latest industries and research trends and discussed ways to increase the global competitiveness of new drug development tasks.On the first day, Muk Hyun-sang, head of the business group, announced the "Transformation of Global Big Pharma," conveying the recent industrial trends and suggesting the direction of developing new drugs in Korea in a changed environment.
It was followed by the introduction of the national new drug development project R&D project operation plan and commercialization support project. Major research projects that received positive responses at last year's workshop were also carried out.
A total of seven new drug development companies (▲Rznomics Co., Ltd., QRIGIN Co., Ltd., IM Biologics Co., Ltd., Uptera Co., Ltd., Wellmarker Bio Co., Ltd., Genome & Company Co., and GI Innovation Co., Ltd.) announced the research status.In particular, participants exchanged opinions and personal exchanges in various ways at this workshop.Participants discussed the process of developing new drugs and key issues and solutions under the theme of different substances in the group discussion, and they derived development strategies tailored to global trends and freely talked about the status of individual task research in poster sessions.The announcement of "National New Drug Development Project Q&A" and "2022 Global Pharmaceutical Bio-Technology Transactions and M&A Trends" was also made, which receive and respond to business-related questions on the spot.Company researchers who participated in the workshop who performed the project
"It was a valuable opportunity to look around various research projects, share opinions with new drug development researchers in the same field, and be stimulated," he said. "This exchange will be of great help to the ongoing research on new drug development."
Charles River Laboratories and Rznomics, a South Korea-based biopharmaceutical company specializing in the development of RNA-based gene therapeutics, entered into a viral vector contract development and manufacturing organization (CDMO) partnership. Rznomics will leverage Charles River’s viral vector CDMO experience to begin clinical development of its RNA-based anticancer gene therapy in liver cancer patients.RZ-001 reportedly is the first ribozyme-based RNA reprogramming approach approved by the FDA for evaluation in patients. The treatment was developed utilizing Rznomics’ RNA reprogramming and editing technology and takes the form of an adenoviral vector that expresses an hTERT targeting ribozyme to treat hepatocellular carcinoma (HCC) patients. HCC is the most common type of primary liver cancer, accounting for 80% of cases worldwide.Rznomics received Phase I/IIa IND approval for RZ-001 from the FDA in October, allowing the start of an international clinical study in HCC patients. Early-phase trials have also begun 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.“This collaboration with Rznomics will tap into our 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 COO, Charles River.“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,” added Seong-Wook Lee, PhD, president and CEO, Rznomics.
Rznomics will attract equity investment (pre-IPO) before
listing this year.
It plans to apply
for a technical evaluation for listing on the KOSDAQ in the second half of this
year and enter the stock market in 2024.
Rznomics is a
biotech that develops RNA-based bio-new drugs.Seong-wook Lee, CEO of Rznomics, recently met E-Daily
and said, "We will push for an initial public offering (IPO) with the goal
of 2024."IPO lead manager is Samsung
Securities(016360).Currently, the leading pipeline liver cancer treatment
"RZ-001" is undergoing clinical trials.
Clinical trials for
malignant glioblastoma are also scheduled to begin within this year.
In the case of
"RZ-001", if excellent data such as safety are confirmed in phase 1
CEO Lee explains
that it is worth looking forward to license out (technology transfer) in the
second half of this year.Other pipelines are also receiving a lot of attention
from global pharmaceutical companies.
CEO Lee said,
"We can't disclose the details, but for verification purposes on the
premise of licensing at the top 10 global pharmaceutical companies, requested
multiple pipeline substances. We are in the final stages of the contract" The core technology of Rznomics is "RNA
editing."Investors are interested in the core technology of
Rznomics as it is about to enter the stock market.
gene therapy based on the RNA substitution enzyme platform.
The key is to remove
disease-related targeted RNA and replace it with a therapeutic RNA at the same
Although it was an
old concept, it did not meet the safety, efficiency, and specificity aspects,
so it was not developed into actual technology.
However, Rznomics is
emerging as a rising star as it catches all those three factors.The specification of is Rznomics’ core technology is that
it targets RNA, not DNA, unlike Christopher-Cas9, a genetic scissor well-known for
targeting DNA. CEO Lee said, "Our technology does not modify DNA, and has
a temporary effect, therefore it has advantage in treating disease such as
cancer. It is also safe since it is not
necessary to put external proteins and not necessary to utilize internal essential
elements inside "
He added, "On
the other hand, in the case of indications targeting non-dividing cells such as
muscle, nerves, and retinal cells, using DNA virus vectors, such as
adeno-assisted viruses(AAV) make it possible to express RNA substitution
enzymes permanently. It allows gene editing without DNA modification.” CEO Lee also stated, "We are finding and developing
indications that our unique technology can be applied well. We will develop a
'niche blockbuster' by promising rare diseases and inaccessible diseases." Overseas competitor deals with Big Pharma with several
billion dollarsTo see how valuable RNA editing technology is in the
market, it is worth referring to deals from overseas competitors.
Theraputics (PRQR) signed a license agreement with Eli Lilly (LLY) in 2021.
Stock prices have
rebounded sharply recently as they decided to expand. At that time, the deal
between ProQR and Lily was $1.25 billion (about 1.6 trillion KRW).Roche made a bigger bet. Roche implemented RNA target
platform of Biotech Shape Theraputics, an American RNA editing technology.
A total of $3
billion (about 3.8 trillion KRW) agreements was signed. Rznomics' RNA editing technology has a distinct characteristic
from these companies."Rznomics RNA editing technology is a mechanism that
replaces and corrects the whole wrong RNA part after the target site with
normal RNA," It's different from competitor's technology in that
point. Even if each patient has a myriad of different mutations, the strength
is that it can be treated by a single treatment." Leading Pipeline Clinical Momentum Line UpThe main pipeline is 'RZ-001', which is being developed
as a liver cancer treatment.
Clinical trial plan
(IND) , was approved for phase 1/2a clinical trial in Korea in June last year,
and in October of the same year, it was approved by the U.S. Food and Drug
Administration (FDA), it will be performed as multinational clinical trial.
RZ-001 is targeting
the telomerase RNA which is expressed specifically in cancer cell with an
adenovirus vector that delivers ribonucleic acid substitution enzymes.
At the same time, it
is a mechanism that expresses genes that induce anticancer action.
CEO Lee said,
"Recently, phase 1 administration has begun," adding, "At the
end of last year, the company applied for a clinical IND for malignant
glioblastoma in Korea. In the first half of this year, we will apply for IND to
the U.S. FDA,"
Rznomics is developing
not only a pipeline of anticancer drugs, but also Alzheimer's drugs, hereditary
retinal pigmentation, and rett syndrome therapeutics etc.
It is developing various
therapeutics in various fields using platform technology. Especially they are
focusing on “RZ-004”, a treatment for retinal pigmentation.
of this year, we will apply for IND for clinical trials in the United States, we
will eliminate harmful genes, considering the modality of expressing good
genes, I think it's a disease that can be implemented well with Rznomics’s
technology," he said.
He added, "As a
result of my full-time job, I was able to receive encouraging results, and
since there are no competitors, innovative treatments are also worth looking
forward to." Development of Circular RNA Structure
Platform…"Possibility of replacing mRNA"
developed a new structural platform that can create circular RNA.
CEO Lee said, "Domestic patents have been
registered, and we are waiting for the registration result with patents application
to the United States and major countries."
Circular RNA is a new type of RNA that has been
shown to have greater stability in vivo than mRNA, and is evaluated to have the
potential to produce a larger amount of therapeutic protein in the body.
In August last year, Biotech ORNA, which can be
seen as a competitor, signed a deal worth $3.65 billion (about 4.7 trillion KRW)
with global Big Pharma Merck (MRK). It was a large contract worth 150 million
dollars (about 200 billion KRW) in down payment alone.
"Circular RNA has the potential to replace
mRNA," mRNA has several patent issues and the process is complicated, but circular
RNA of Rznomics can be done in a single process." "Rznomics has its
own circular RNA technology and has different structural and component
characteristics from existing technologies, so it will be competitive,"
Attempt to extend RZ-001 indication Rznomics announced on January 4 that it applied to the Ministry of Food and Drug Safety for phase 1 and 2 clinical trials for malignant glioblastoma (brain cancer) of "RZ-001", a new anticancer drug candidate.RZ-001 was approved by the Ministry of Food and Drug Safety and the U.S. Food and Drug Administration (FDA) last year and is undergoing clinical trials. Malignant glioblastoma is an incurable disease with a 5-year survival rate of less than 3 percent and no clear treatment yet. Immuno-cancer drugs also showed no therapeutic effect, so a new mechanism of treatment is urgently needed.Rznomics has a technology that removes target RNA based on RNA trans-splicing ribozyme and expresses the desired gene. RZ-001 is an adenovirus that carries ribonucleic acid substituent. It targets telomerase (hTERT) RNA that is specific to cancer cells and expresses genes that induce anticancer action. It is said that telomerase targeted by RZ-001 can be applied to various carcinomas because it is expressed in most cancer cells.Rznomics is pushing to expand its indications to other intractable cancers, starting with hepatocellular carcinoma undergoing clinical trials and this malignant glioblastoma. It is also developing treatments in various fields using platform technologies such as Alzheimer's, hereditary retinal pigmentation, and retinal syndrome as well as anticancer drugs.Seong-wook Lee, CEO of Rznomics, said, "The candidate materials under development are entering clinical trials as planned. We will do our best to speed up the progress of clinical trials and quickly provide new treatment opportunities to patients suffering from incurable diseases."
The company's cumulative investment attraction, which aims to develop innovative bio-new drugs for cancer and incurable diseases, amounts to 60.9 billion. The reason why investors value Rznomics highly is the "Trans-Splicing Ribozyme" platform technology. This technology removes the bad-tempered RNA sequence that causes incurable diseases in the body and injects enzymes (ribozyme) into the area so that good-tempered RNA can be produced. Professor Lee has been studying a technology that induces the activity of therapeutic genes in cells by editing targeted RNA, which is a treatment target, into therapeutic RNA for 20 years. In particular, through research to increase the safety of enzymes, enzymes with therapeutic functions were developed for the first time in vivo.Rznomics is challenging the development of liver cancer treatments using this technology. Following the announcement of the results of research on liver cancer cell death using the RNA substitution enzyme platform in the official journal of the U.S. Genetic Cell Therapy Association, the domestic Ministry of Food and Drug Safety and the U.S. Food and Drug Administration approved phase 1 and 2a clinical trials for "RZ-001", a candidate for liver cancer treatment.It is also studying glioblastoma of malignant brain tumors and a treatment for hereditary retinal pigmentation. And It is also considering the possibility of developing treatments for neurological and cognitive diseases such as Alzheimer's and Ret syndrome.Professor Lee had been considering starting a business early on thanks to his advisor's teaching at the time of his Ph.D. ‘s saying that "he should not be satisfied with leaving research results and names on a few pages of paper.""I believe that the technology developed through sweat with researchers with the support of taxpayers' money should not be replaced," he said. "Our goal is to grow into a global company based on unique platform technology and pipelines that prove the value of this technology."
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