News
TOTAL91 (1/1 page)
-
RZNOMICS Inc. received FDA approval to initiate clinical development of trans-splicing ribozyme-based RNA editing technology in Glioblastoma Multiforme Patients
SEONGNAM, South Korea, May 19, 2023 /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 on May 6th for its Glioblastoma Multiforme (GBM) treatment called RZ-001 and thus has achieved an important milestone for the company and the RNA editing field. RZ-001 initially obtained the IND approval with the indication for HCC, but Rznomics also found the great pre-clinical efficacy in GBM models and submitted the IND for the GBM. 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 GBM patients.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 catalytically 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-GBM 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 GBM tumors and hampers angiogenesis in the tumor tissues 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 recurrent GBM patients without extracranial metastases. "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 another IND approval in the United States with the indication for the GBM. I am really grateful that RZ-001 earned the opportunity to potentially fulfill the unmet needs of GBM 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, Ph.D., CEO, and founder of Rznomics.
2023-05-20 -
Rznomics announces results of preliminary retinal pigmentation in international society
Proof of efficacy and safety in large animals
Rznomics announced on the 3rd that it has announced the results of a preclinical study of "RZ-004," a candidate for the treatment of Retinitis pigmentationosa, at the American Academy of Ophthalmology (ARVO).
ARVO is the world's largest ophthalmic society founded in 1928. It has more than 10,000 members in more than 75 countries. This year's conference was held in New Orleans, the United States.
The presentation was given by Dr. Archana Jalligampala of the University of Louisville in the United States. The content was the evaluation of the animal model of rhodopsin mutant dominant retinal pigment degeneration targeted by RZ-004. RZ-004 has an RNA editing mechanism that removes mutated rhodopsin ribonucleic acid (RNA) and replaces it with a normal rhodopsin gene. It is in the preclinical completion stage after deriving the final candidate material.
Rznomics said it demonstrated the efficacy of RZ-004 including retinal potentiogram (ERG) through preclinical trials. Toxicity and safety tests are also in the final stages. It is planning to apply for clinical trials with the U.S. Food and Drug Administration (FDA) at the end of the year.
Ji-hyun Kim, a senior researcher at Rznomics, said, "The results of this study are meaningful in that safety and effectiveness have been confirmed in the animal disease model."
Seong-wook Lee, CEO of Rznomics, said, "Tenocular pigment degeneration is in great demand because there are few existing treatments," adding, "Global pharmaceutical companies are also very interested in RZ-004, So I feel a sense of responsibility" He added, "We will do our best to provide innovative treatment opportunities to patients through rapid clinical verification."2023-05-09 -
Rznomics Applies to U.S. FDA for Phase 1 and 2a of Glioblastoma Clinical Trials
Hepatocellular carcinoma followed by expanded indications
Rznomics announced on the 12th that it has applied to the U.S. Food and Drug Administration (FDA) for phase 1 and 2a clinical trials for glioblastoma of "RZ-001. This is to expand the indication following the ongoing clinical trial for hepatocellular carcinoma.Glioblastoma is a malignant tumor that occurs in the brain. The average survival period is less than 15 months and the five-year survival rate is less than 3%. There is no clear cure yet.Based on RNA trans-splicing ribozyme, Rznomics is developing a technology that removes target RNA and expresses the desired gene at the same time. RZ-001 delivers ribonucleic acid substituent to adenovirus. It targets telomerase (hTERT) RNA, which is specifically expressed in cancer cells, and expresses a gene that induces anticancer action.Therefore, it is universally applicable to all carcinomas in which target substances are expressed. Non-clinical results through glioblastoma animal models showed excellent anticancer ability. Rznomics is developing treatments such as Alzheimer's, hereditary retinal biological degeneration, and retinal syndrome using platform technology as well as anticancer drugs.Seong-wook Lee, CEO of Rznomics, said, "Even in a difficult bio-industry environment, candidate substances under development are entering the clinical stage smoothly," adding, "We will do our best to provide new treatment opportunities to patients as soon as possible."2023-04-14 -
Nature Biotechnology news article
Rznomics was introduced in the Nature
Biotechnology Journal. We are proud to be introduced as one of the leading RNA
editing companies in the Nature Biotechnology Journal. The article mentioned
one of the advantages of the trans-splicing ribozyme, such as the capability of
large-scale changes that can be introduced in a mature mRNA transcript. The
article also mentioned our RZ-001 (HCC) IND approval from both MFDS & FDA.
Rznomics is currently running the P1/2a trial in South Korea.
Please find the details about the
attached article and the following link!2023-03-29 -
Development of liver cancer treatment with RNA substitution technology. Interview with CEO Seong-wook Lee of Rznomics.
"Since last year, the liver cancer treatment 'RZ-001' has been in phase 1 clinical trials, and discussions on technology transfer (license out) with two global companies are continuing." Seong-wook Lee, CEO of Rznomics, who recently met with a reporter, explained this. Rznomics was founded in August 2017 by Seong-wook Lee, a professor of bio-convergence engineering at Dankook University, a Ph.D. from Cornell University in the U.S. And Rznomics is a bio company that uses RNA substitution enzyme technology that was studied at Duke University to develop new drugs.
CEO Lee said, "If existing RNA editing technology only cuts targets or corrects specific areas, we cut target RNA and replace the entire cut target area with therapeutic RNA at the same time," adding, "It suppresses harmful gene expression and makes positive effects." For this reason, retinal cells, muscle cells, and nerve cells, which are good to disappear after action, have a strategy to induce gene correction without gene editing by permanently expressing RNA substitution enzymes such as AAV (adenosine virus). CEO Lee explained, "After liver cancer, glioblastoma is being treated, and we expect to apply various intractable and rare cancers, genetic retinal diseases, and Alzheimer's in the future." CEO Seong-wook Lee said, "We are directly discovering pipelines for the development of major treatments, but we are also aiming to find pipelines that can apply our RNA platform technology through active cooperative research with the outside world," adding, "We also have a Circular RNA platform technology that can be used as a vaccine and treatment." Rznomics received approval from the Korean Ministry of Food and Drug Safety and the U.S. FDA last year to conduct phase 1 of RZ-001 clinical trials at five domestic hospitals, and plans to conduct phase 2a clinical trials simultaneously in Korea and the U.S. in the future. Clinical 1/2a for malignant brain tumors has also been applied to the Ministry of Food and Drug Safety in Korea and is being reviewed, and will also apply to the U.S. FDA within the first quarter. CEO Lee said, "The RNA treatment and gene therapy market has been proven to be effective in the U.S. after 2017 with FDA approval," and explained, "Rznomics is one of the world's leading research groups in RNA substitution enzyme technology." Rznomics received a cumulative investment of 60.9 billion won last year, including Series C, and Korea Development Bank, Aeon Investment, Partners Investment, IBK Capital, Quad Ventures, SBI Investment, Shinhan Venture Investment, and UTC Investment participated as investors. It will receive a technical evaluation in the second half of this year and request a preliminary review of KOSDAQ listing in the first half of next year.2023-03-08 -
Rznomics, collaboration with Yonsei Medical Center to develop treatments for incurable diseases.
Rznomics announced on the 6th that it has signed a business agreement with Yonsei University Medical Center to develop treatments for incurable diseases using RNA-based technology.The two institutions decided to cooperate on the overall development of treatments, including preclinical and clinical research. It promotes mutual exchange of academic, human, and material resources and technology development.In particular, it plans to launch a Proof of Concept study to develop a treatment for eye cancer, one of the rare intractable cancers.Jae-young Choi, head of the industry-academic cooperation team at Yonsei Medical Center, said, "We hope to provide treatment opportunities for patients with incurable diseases with high medical unmet demand through joint research and development of RNA-based gene therapy."Seong-wook Lee, CEO of Rznomics, said, "It is expected to accelerate new drug development and create synergy effects by combining Rznomics' next-generation RNA-based platform technology with Yonsei Medical Center's latest medical technology and know-how in clinical research."
2023-02-07 -
KDDF, Global Pharmaceutical and Bio Industry Trends - Share the Status of Major Challenges
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."2023-02-06 -
Charles River and Rznomics Partner on RNA-based Anticancer Gene Therapy Manufacturing
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.
2023-01-25 -
“Aapplication of a technical evaluation for listing in the second half of the year”
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
clinical trials,
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.
Rznomics develops
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
time.
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.
NASDAQ-listed ProQR
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.
"In September
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"
Rznomics recently
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,"2023-01-09
- 본사경기도 용인시 수지구 죽전로 152, 단국대학교 소프트웨어ICT관 327호
- TEL. 031-706-8730
- FAX. 031-8018-8734
- E_mailrznomics@rznomics.com
- 기업부설연구소 및 판교지점경기도 성남시 분당구 판교로 253, 판교이노밸리 C동 801호(판교지점), 803호(기업부설연구소)
- TEL. 031-706-8730
Copyright(C) 2019 Rznomics. All Rights Reserved.
