RIBOZYME AND OMICS
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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.
announced on the 21st that it has received approval from the Ministry of Food
and Drug Safety for phase 1 and 2a clinical trials of "RZ-001", which
is being developed as a primary liver cancer treatment.
1 for safety verification and phase 2a for effectiveness search were designed
to be implemented in one clinical trial.
has a platform technology that can remove targeted RNA using RNA trans-splicing
ribozyme while expressing the desired gene.
platform was also applied to RZ-001. It is explained that it targets telomerase
(hTER) RNA, which is specifically expressed in cancer cells, to secure safety
and at the same time to express genes that induce anticancer action. In this
process, it is said to induce immune cells in the body, causing triple
plans to perform multinational clinical trials after obtaining approval from
the U.S. Food and Drug Administration (FDA) for the same clinical design in the
second half of this year. In Korea, Samsung Medical Center, Seoul National
University Hospital, Severance Hospital, Seoul St. Mary's Hospital and
Kyungpook National University Hospital will participate as clinical trial
Lee, CEO of Rznomics, said, "It is a monumental task to administer a treatment
substance using RNA trans-splicing ribozyme technology to primary liver cancer
patients for the first time," adding, "We will do our best to provide
new treatment opportunities to patients in need of anti-cancer treatment."
is also promoting the expansion of indications to various cancer types. In the
case of malignant glioblastoma, it is planned to enter the clinical stage
within this year.
Rznomics Inc. (https://www.cortellis.com/intelligence/qsearch/rznomics) recently secured ₩37.2 billion
(US$30 million) from private investors and the Korean government in a series C financing round. “The
$30 million series C proceeds will be used for the phase I/IIa clinical trial of RZ-001 (https://www.cortellis.com/intelligence/qsearch/RZ-001), and the additional R&D costs for candidates RZ-004
(https://www.cortellis.com/intelligence/qsearch/RZ-004) and RZ-003 (https://www.cortellis.com/intelligence/qsearch/RZ-003),” Rznomics CEO Seong-wook Lee told BioWorld.
RZ-001, Rznomics’ lead candidate, is an investigational RNA replacement enzyme vector-based cancer
therapy to treat hepatocellular carcinoma (HCC). A phase I/IIa trial of the candidate is planned for initiation in the second half of 2022. “It will be a dose escalation/expansion study, to be held at major study
sites in South Korea including the Samsung Medical Center. The clinical trial’s expected milestone dates
are to be determined,” said Lee. He hailed the financing as one of great value in “the depressed bio-market atmosphere.”
RZ-004 is a preclinical gene therapy candidate targeting retinitis pigmentosa. The IND for that indication
is to be submitted to FDA in the first half of 2023. RZ-003 is the company’s adeno-associated virus (AAV)
delivered candidate, intended for the potential treatment of Alzheimer’s disease.
Also in Rznomics’ pipeline is in RZ-002, another adenovirus candidate for glioblastoma. An IND submission for the telomerase reverse transcriptase targeting candidate planned for this year. “Rznomics is planning to submit the IND to the FDA for RZ-004 in 2023 and for RZ-001 with the indication
of glioblastoma in 2023,” said Lee.
The most recent financing brings Rznomics’ cumulative investment total to date to ₩60.9 billion. Investors in the RNA-focused biopharma include the Korea Development Bank, AON investment, Partners
Investment, IBK Capital Corp, Quad Ventures, SBI Investment Co. Ltd., Shinhan Venture Investment Co.
Ltd., and UTC Investment Co. Ltd. “The investment was made by the consideration of specialty and capabilities of trans-splicing ribozyme
in RNA based treatments, competitive pipelines, and the high level of R&D data,” said Byung-kyu Ahn,
the director of AON Investment, a new participant that joined in the series C. “After the COVID-19 pandemic, the needs and expectations of gene therapy have increased. I believed
Rznomics showed capabilities of satisfying the global needs. In contrast to the CRISPR-Cas9 system that
causes permanent gene editing, trans-splicing ribozyme technology induces gene editing at the RNA
level,” said Junsoo Kim, a managing director in Partners Investment.
Partners investment has been onboard with Rznomics from its series A to C financing rounds. Government funding, IPO In addition, the company's work has since been recognized by the South Korean government as several
government funding projects, including the development of RZ-004 as a 'national new drug development project'. The recognitions won it a separate cumulative ₩8 billion in research funding. “Overall, the environment for the development of gene therapy in South Korea is pretty active and
bright. We have received proper support and reviews from the Ministry of Food and Drug Safety and
with that, also have plans to develop our pipelines globally,” he added. That’s not all the financing news that Rznomics has for now. The company is currently busy preparing the
evaluation of its technology to be included as a technology specially listed company. It is also exploring
the potential for an IPO as a technology specially listed company, possibly for the second half of 2023.
The last time (https://www.bioworld.com/articles/499139-rznomics-on-track-for-adenoviruses-development-plan) BioWorld spoke to the company in 2020, it was using Cologne, Germany based-Cevec Pharmaceuticals GmbH’s technology to manufacture adenoviruses. “The contract has been signed and we
have completed the GMP production for RZ-001,” said Lee. Rznomics is heavily invested in its trans-splicing ribozyme platform, technology designed to remove disease-causing target RNA and trans-ligates, as well as opportunities to out-license the technology to global
Rznomics, raised KRW 37.2 B in series
Rznomics, a new drug developer for RNA-based gene
therapy, announced on the 7th that it has completed attracting KRW 37.2 billion
in Series C round.
In this round, Korea Development Bank, AON Investment,
Partners Investment, IBK Capital, Quad Ventures, SBI Investment, Shinhan
Venture Investment, and UTC Investment participated as investors.
As a result, Rznomics achieved a cumulative total of KRW
60.9 billion, including KRW 23.7 billion in series A and B, and received about KRW
8 billion in government-funded research funds through national new drug
Rznomics has an innovative platform technology so called
Trans-splicing ribozyme, and represents a mechanism to remove disease-causing
target RNA and express therapeutic RNA in its place.
Using this, innovative new drugs are being developed
for liver cancer, glioblastoma, Alzheimer's, and hereditary retinal
Furthermore, it has secured its own circular RNA
structure platform technology that can be applied to various fields such as
treatments and vaccines.
In the case of liver cancer treatments, which are the
most advanced in the new drug pipeline, clinical trials (phase 1/2) are
scheduled in Korea and the United States from the second half of this year, and
a clinical trial plan for glioblastoma is also planned within this year.
In the first half of next year, a clinical trial plan
for Retinitis pigmentationosa, a rare eye disease, will also be submitted to
the U.S. FDA.
In addition, it is planning to take a technology
evaluation for Special Listing of Technology Growth in the first half of next
year and is pushing for an IPO with the goal of the second half of next year.
Seong-wook, Lee, CEO of Rznomics, said, "It is a
great help because it seems to have been recognized for its potential and value
despite the stagnant bio-market atmosphere, and we will accelerate research and
development even more." "We are actively discussing with leading
multinational pharmaceutical companies for license-out, so we expect to deliver
good news soon."
Regarding the reason for the decision to participate
in this investment since Series A, Kim Joon-soo, senior team leader of Partners
Investment, said, "The need and expectations for gene therapy are rising
after COVID19 pandemic, and in particular, Rznomics has unrivaled global
RNA trans-splicing technology implements RNA-level
correction unlike gene scissors that cause permanent genetic modification, and
operates as an enzyme's own mechanism without needing help from external
proteins for correction function, allowing for permanent development and
commercialization of new drugs.
In other words, we decided to invest not only because
we could fundamentally solve the medical unmet demand of genetic and
intractable diseases, but also because we could expect the scalability and
profitability of the business," he said.
Byung-kyu, Ahn, director of AON Investment, who
participated in this investment with a large scale as a new Venture Capital,
also said, "We decided to invest this time by comprehensively considering
the mechanical differentiation of R&D technology owned by Rznomics in the
RNA therapy area, pipelines that can maximize it, and R&D data at the
trans-splicing ribozyme based gene therapy technology platform company, announced
on the 12th that their innovative new drug treatment, which is being developed
for hereditary retinal pigmentation, was selected as a support project for the
National New Drug Development Project's first "New Drug R&D Ecosystem
Construction Research" in 2022.The
national new drug development project is a pan-ministerial national R&D
project, which is promoted by the Ministry of Science and ICT, the Ministry of
Trade, Industry and Welfare, and has been supporting the whole cycle of new
drug development since 2021 with the goal of strengthening global
competitiveness of the national pharmaceutical and bio industries.Rznomics'
selected research on building a national new R&D ecosystem is a task to
support candidate materials and non-clinical stages, and this selection will
receive a total of KRW 2 billion in national research funds for 22 months with
the goal of non-clinical research and IND approval.Retinal
pigmentation is a blindness disease that occurs in one out of every 4,000 to
5,000 people worldwide and loses central vision due to abnormal photoreceptor
cells distributed in the retina.Up
to now, there is no fundamental treatment method, and it has relied on some
adjuvant treatments that prevent vision from being damaged by ultraviolet rays
or slow vision loss by taking nutritional supplements.The
cause of retinal pigmentation is a defect in the genes involved in the
mechanism of converting light into electrical signals in visual cells.Among
several gene mutations, mutations in the RHO gene are known to be the most
common form of mutations in the disease, accounting for 20 to 30% of autosomal
dominant retinal pigmentation.The
progress rapidity of vision damage depends on individual, but it usually starts
with night blindness, gradually narrowing the field of vision, and losing the
central field of vision, leading to complete blindness.The
treatment under development by Rznomics is an RNA-based gene therapy that is
being developed for a single administration, and it is expected that the
disease can be fundamentally treated by replacing the mutant RHO gene with the
normal RHO gene using "Trans-splicing ribozyme" technology, which
removes and corrects the mutant gene.Dr.
Ji-hyun, Kim who was selected as the research director of the project, said,
"Rznomics' platform technology, which has accumulated technology for a long
period of time, has been recognized for its excellence, and we will do our best
to approve IND with the goal of clinical progress."Seong-wook,
Lee, CEO of Rznomics, said, "This is the second national new drug project
since the development of Rett’s syndrome treatment was selected as a national
new drug project at the end of last year, and it is believed to be a good
example of new drug development as a bio-venture in Korea."
Rznomics Inc. has been listed in the '51 Top BioPharma Startups & Companies (South Korea)' by Biopharma.Please refer to the link for the full list.
[K Startup Industry Map] bio-healthcare IndustryJust
a few years ago, the bio-healthcare industry was considered a new growth engine
to feed Korea.
only the modifier "Bio" was added, the corporate value jumped several
times, and investors came with money.
the market froze hard through the COVID-19 pandemic.
for some fields such as diagnostic kits, the "money line" in the bio
industry has dried up.
is because IPO, venture capital (VC) investment, and CB issuance, which are key
funding channels, have been delayed or canceled one after another.Boronoi,
which was expected to be the "Unicorn Special List No. 1" in March,
withdrew its listing due to low demand forecasts, and Ari-bio, a new dementia
drug R&D company, failed in the technology evaluation for Special Listing
of Technology Growth. Mezzion's stock price fell down sharply due to not
approving of Fontan surgery (heart deformity surgery) treatment by the U.S.
Food and Drug Administration (FDA).This
phenomenon doesn’t happen only in Korea.Since
the pandemic, volatility in the global bio-healthcare sector has continued to
expand. However, it is analyzed that this increase in volatility does not stem
from the damage to fundamentals and growth potential of the bio and healthcare
say that investment sentiment has temporarily worsened due to delays in
clinical trials due to COVID-19, interest rate hikes, and falling pipeline
values due to geopolitical risks due to bio companies, whose future value
accounts for most of their corporate value.Actually,
the global new drug development market is expected to grow by 7.5% annually by
2026 and grow to USD 304.3 billion (about KRW 386 trillion).According
to Mirae Asset Securities, the number of clinical trials, a barometer of the
new drug development market, recorded 24,914 cases (excluding COVID-19 clinical
trials) as of 2021.The
digital healthcare market, which has emerged as a future medical system, is
also rapidly expanding.According
to the Korea International Trade Association, the global digital healthcare
market is expected to grow by nearly 30% annually from $106.3 billion (about
135 trillion won) in 2019 to $639.4 billion (about 810 trillion won) in 2026.Fortunately,
as the Yoon Seok-yeol government announced plans to actively foster the bio and
healthcare industries and build infrastructure to support biotechnology fields
such as new drug development, the bio industry is gaining momentum.Bio-healthcare
startups that stand out as they seek to enter overseas markets such as the U.S.
and Europe are also emerging one after another. It introduces bio and healthcare
startups that should be noted.▶ New drug development▷ Rznomics' performance is becoming visibleNew drug development is
the 'flower of the bio industry'. It's not easy, but it's because once you
succeed, you get a huge profit. In particular, as global big pharmaceutical
companies are actively engaged in open innovation such as JV(Joint Venture),
M&A (Merger and Acquisition), licensing, and joint development for new
pipeline transfusions, more opportunities are opening up for new drug
development start-ups.Choong-hyun, Kim, an
analyst at Mirae Asset Securities, said, "Among the top 30 drugs in the
world, 22 have currently expired patents or expired patents. Investment in new
drug development is inevitable for new pipeline transfusions, he said. "It
is an opportunity for domestic bio companies with competitiveness in new drug
development to quantum jump."Cell and gene
therapy is the most notable field in the recent development of new drugs.Rznomics is a
new drug development company based on RNA (ribonucleic acid) established by
Seong-wook, Lee, a professor of bio-convergence at Dankook University in 2017.
It is a mechanism by which RNA replacememt enzymes are used to remove target
RNA that causes disease and generate therapeutic RNA in its place. Therapies
are being developed for liver cancer, glioblastoma, Alzheimer's, and hereditary
retinal pigmentation. In the fourth quarter of 2021, it plans to apply for
domestic and U.S. IND (clinical test plan) and enter clinical trials from 2023.(Extract from the original article)
announced on the 28th that it has won consecutive orders from the Korea Patent
and Strategy Development Institute for IP-R&D Strategy Support Project in
response to infectious diseases and the Korea Research Foundation's "2022
Basic Technology Development Project for next-generation vaccines.Rznomics
said it has been recognized for its technology for the company's new circular
ribonucleic acid (RNA) structure platform.According
to the company, the circular RNA structure was developed to allow self-circularization
to occur during transcriptional reactions in vitro. In addition, it is more
stable than messenger ribonucleic acid (mRNA), which is a linear structure, so
its efficacy lasts for a long time. It is expected to be applied to various
fields including vaccines and therapies."It
is an independent platform technology that can overcome patent issues of mRNA therapy
and vaccine technologies," said Seong-wook, Lee, CEO of Rznomics.
"Through cooperative research with groups which has delivery technology,
we will optimize it to be applied to various fields such as RNA vaccines and therapy."