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Rznomics Says Genetic Disease 'RNA Replacement Enzyme' 'Mutational Effect'
Rznomics Says
Genetic Disease 'RNA Replacement Enzyme' 'Mutational Effect'
[ASGCT 2023] What are the main preclinical results of
"RZ-004," a candidate for autosomal dominant retinal pigment
degeneration.."It will be submitted to FDA IND at the end of this year."
Rznomics has unveiled a follow-up program following a
trans-splicing ribozyme-based anticancer drug in the initial clinical
development stage. Rznomics announced on the 23rd that it had confirmed the
effect of editing mutation-independent RNA based "RZ-004" developed
as a treatment for retinitis pigmentationosa (RP) at the American Society for
Gene Cell Therapy (ASGCT 2023) in Los Angeles.Rznomics announced the
optimization of candidate materials for RZ-004 and non-clinical results at the
ASGCT, and the presentation was conducted by Dr. Ji-hyun Kim, head of the
genetic disease task at Rznomics. Rznomics will submit a clinical trial plan
(IND) to the U.S. Food and Drug Administration (FDA) later this year to enter
clinical trials. According to Rznomics abstract information, group I
introns-based trans-splicing ribozyme, an RNA replacement enzyme, is developing
treatments using an RNA replacement mechanism. RZ-004 removes the mutated
rhodopsin RNA and operates as a mechanism to replace it with normal rhodopsin
RNA and edit it.RZ-004 was transferred to the eye via the AAV
(adeno-associated virus) vector. Rznomics has discovered the most accessible
rhodopsin RNA target site through RNA mapping in the ribozyme library. It has
been confirmed that the most efficient RNA editing occurs in the uracil (U)
base of RHO RNA 5' UTR. Rznomics also optimized ribozyme structures and
sequences to increase the trans-splying specificity and efficacy of RNA
replacement enzymes. Rznomics has identified candidates with the highest
trans-splicing editing efficiency in the in vitro cell line, and with notable
data, it has confirmed that various RHO RNA mutations are replaced by normal
(WT) RHO RNA.Rznomics delivered RZ-004 via the AAV vector to evaluate whether
it actually affects the invivo function. Rznomics delivered RZ-004 to both eyes
as a subretinal injection to the retinal pigment epithelial rat model
(P23HRHOKI). As a result, it was confirmed that the dose-dependent ERG b-wave
amplitude (amplitude) increased when RZ-004 was administered, unlike the
control group. In addition, in molecular and cell-level analysis, P23H RNA in
retinal tissue was efficiently changed to WTRHO, and the outer nuclear layer
composed of photoreceptor cells mainly targeted by ribozyme was thickened.Rznomics
also showed an electric retinal potential (ffERG) reaction when the AAV-based
RHO target ribozyme was injected under the retina in the pig model (TgP23HRHO
pig), and confirmed that the outer nuclear layer was thickened. In other words,
RZ-004 ribozyme inhibits photoreceptor degradation and maintains function in
both mouse and pig disease models, confirming the possibility of development as
a therapeutic agent. Rznomics added that it confirmed safety in primates.
Rhodopsin mutant dominant retinal pigment degeneration targeted by RZ-004 is
very demanding because there is no existing treatment, said Seong-wook Lee, CEO
of Rznomics. "We will do our best to provide innovative treatment opportunities
as soon as possible to patients suffering from vision loss."2023-05-25 -
Rznomics announces result of New Drug Pre-clinical Treatment for Retinal Dysplasia' at American Society of Gene & Cell Therapy (ASGCT)
Rznomics announces result of New Drug Pre-clinical Treatment
for Retinal Dysplasia' at American Society of Gene & Cell Therapy (ASGCT)
RNA Editing 'RZ-004' pipeline plans
To apply for FDA Clinical Trial at year-end of 2023.
Rznomics, announced on the 22nd that
it has announced the results of a pre-clinical study of its pipeline
"RZ-004," a candidate for treatment for retinal pigment degeneration,
at the American Society of Gene & Cell Therapy (ASGCT) held in Los Angeles.
RZ-004 received a lot of attention
on its potential as a new treatment through the announcement of the Association
for Research in Vision and Ophthalmology (ARVO) last month. About a month
later, the American Society of Gene & Cell Therapy selected it as oral
presentation topic. Research results such as material optimization and
pre-clinical test results have been announced.
The presentation was made by Dr.
Ji-hyun Kim, in-charge of genetic diseases at Rznomics. RZ-004 has an RNA
editing mechanism that removes mutated rhodopsin ribonucleic acid (RNA) and
replaces it with a normal rhodopsin gene be in the pre-clinical stage of completion.
Pre-clinical results that can enter clinical trials, such as efficacy, safety,
and toxicity test results confirmed by animal models with various diseases,
have been accumulated by the company.
The effectiveness was demonstrated
through electroretinogram (ERG) and optical coherence tomography (OCT). It also
confirmed safety by using rodent, pig models and non-human primates. It is
planning to apply for clinical trials with the U.S. Food and Drug
Administration (FDA) at the end of the year.
Dr. Ji-hyun Kim said, "The
results of RZ-004 were selected as the topic of oral presentation by ASGCT, the
most prestigious academic society in the field of gene and cell therapy,"
adding, "I am happy for this opportunity to announce RZ-004 to the
world."
Seong-wook Lee, CEO of Rznomics,
said, "RZ-004's target rhodopsin mutation-mediated autosomal dominant
Retinitis Pigmentosa is very demanding because currently there is no
treatment," adding, "we will do our best to provide patients with
innovative treatment opportunities as soon as possible."2023-05-22 -
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
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