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Rznomics gets approves U.S. FDA For anticancer Candidate Based On Gene Therapy
Rznomics gets approves U.S. FDA For anticancer Candidate Based On Gene Therapy
RZ001+T-Sentric + Avastin
Approved Phase 1b/2a clinical trial for hepatocellular carcinoma patients
Rznomics, a gene therapy drug-based anticancer drug developer, will start
clinical trials in the United States that use its candidate and immune
anticancer drugs in combination.
Rznomics announced on the 19th that it has received approval from the U.S. Food
and Drug Administration (FDA) for its phase 1b/2a clinical trial in which the
anticancer drug candidate "RZ-001" and immuno-cancer drugs are
combined.
The plan is to evaluate both effectiveness and safety by co-administering
RZ-001 together with the first standard treatment (Tscentrick + Avastin) to
about 50 patients diagnosed with hepatocellular carcinoma.
RZ-001 is an anticancer drug candidate that is being developed by applying the
RNA editing platform technologies owned by Rznomics. It targets telomerase
(hTERT) RNA, which is specifically expressed in cancer cells, by delivering RNA
enzymes with adenovirus as a vector.
Normal cells become shorter in telomeres, and when there is not much time left,
they perceive themselves to be 'old' and die without further dividing. However,
in cancer cells, telomerase, an enzyme that increases the length of telomeres
that is decreasing again, is overactive, and telomeres may continue to
lengthen. This is the cause of cancer cells continuing to divide without dying.
RZ-001 removes hTERT RNA from hepatocytes that have become cancerous cells so
that telomerase is not expressed. It induces apoptosis by making telomeres
shorten normally. HSV-TK is also expressed at the cut hTERT site. Antiviral
drugs (balgancyclover) administered with RZ-001 selectively attack only
cancerous cells in response to this gene.
It is a dual mechanism that removes the telomerase gene that causes infinite
proliferation of cancer cells and inserts virus-derived genes to cause antiviral
drugs to attack cancer cells.
The FDA-approved clinical trial was combined with ticentric + avastin, which is
the most widely used first-line treatment for hepatocellular carcinoma. If
differentiated safety and efficacy are demonstrated compared to existing
treatments, it could open the way for them to enter the largest market of
first-line treatments.
Rznomics plans to collaborate with large domestic and international
pharmaceutical companies for this clinical trial. Among the clinical drugs,
atetzolizumab has been contracted to receive supply from Roche and bevacizumab
from Celltrion.
Seong-wook Lee, CEO of Rznomics, said, "As many researchers and
institutions cooperate in RZ-001 clinical development, we will do our best to
succeed as an innovative anticancer drug."2024-08-28 -
SK pharmteco and Rznomics Explore Collaboration for RNA-based Gene Therapy Development and Manufacturing
SEONGNAM, South Korea, Aug. 5, 2024 /PRNewswire/ -- SK pharmteco, a global contract development, manufacturing, and analytical testing organization serving both the small molecule and cell & gene therapy industry, and Rznomics Inc., a South Korea-based biopharmaceutical company specializing in the development of RNA-based gene therapeutics, have signed a memorandum of understanding (MOU) for a contract development and manufacturing partnership.When finalized, the multi-year partnership will establish a strategic collaboration between SK pharmteco and Rznomics to develop and commercialize multiple gene therapy products. SK pharmteco will provide technical expertise and resources, including the facilities and personnel, to enable a smooth transition from clinical to full-scale commercial manufacturing of Rznomics' innovative ribonucleic acid (RNA)-based biopharmaceuticals."We are thrilled about the opportunity to partner with Rznomics on their groundbreaking initiatives," said Andy Fenny, Chief Commercial Officer of SK pharmteco. "By combining our significant expertise in gene therapy manufacturing with Rznomics' innovative RNA-based therapeutic approach, we believe this collaboration has the potential to deliver life-changing treatments to patients in need quickly."Seong-Wook Lee, President and Chief Executive Officer of Rznomics, commented: "This partnership will mark late-stage development and manufacturing of our gene therapy pipeline to prepare the path towards Phase II and later stage clinical trials and potential commercialization. We look forward to working closely with SK pharmteco."About SK pharmtecoSK pharmteco is a global contract development and manufacturing organization (CDMO) with 13 offices and manufacturing facilities across the U.S., Europe, and Korea. The company partners with biopharmaceutical companies of all sizes to manufacture Active Pharmaceutical Ingredients (API) and intermediates, cell and gene therapy technologies, registered starting materials, and analytical services for the biopharmaceutical industry worldwide. SK pharmteco is a subsidiary of SK Inc. (KRX: 034730) (SK), the strategic investment company for SK Group, South Korea's second-largest conglomerate.About RznomicsRznomics is a South Korea-based innovative RNA biopharmaceutical company, with vision to treat/conquer various human intractable diseases.Rznomics has developed and focused on several pipelines with RNA editing platform technologies of RZ-001 (Hepatocellular Carcinoma, Glioblastoma), RZ-003 (Alzheimer), RZ-004 (Retinitis Pigmentosa), RZ-005 (neurodegenerative disorder) and Circular RNA platform.For RZ-001, the lead pipeline, Rznomics has received Phase I/IIa IND approvals from the FDA for Hepatocellular Carcinoma and Glioblastoma in 2022 and 2023 each. In addition, FDA has granted Fast Track Designation for GBM in November 2023 and Orphan Drug Designation for HCC in January 2024.Early-phase trials have also commenced in Korea, upon IND approval for RZ-001 from the Ministry of Food and Drug Safety (MFDS). Rznomics has received IND approval for combination of RZ-001 and Immunotherapy from the MFDS as well, in December 2023.
2024-08-06 -
Rznomics gets approval from Clinical Trial Plan for Genetic Retinopathy
Rznomics gets approval from Clinical Trial Plan for Genetic Retinopathy
Rznomics announced on the 8th that it has received a
notification from the Australian Federal Drug Administration (TGA) on the
completion of the approval process for its phase 1 clinical trial plan for
RZ-004, which is being developed as a treatment for hereditary retinal pigment
degeneration.
In order to conduct clinical trials of gene therapy drugs in
Australia, it is necessary to undergo preliminary deliberation from the Office
of the Gene Technology Regulator (OGTR) as a preliminary stage for approval of
clinical plans.
Rznomics completed the procedure in January of this year.
This means that all deliberation and administrative procedures for clinical
initiation in Australia have been completed.
Retinitis Pigmentosa is a disease in which visual cells are damaged and the visual field
becomes narrower, resulting in loss of vision. As pigment builds up in the
retina, retinal function disappears, which is well known as a rare and
incurable disease. It occurs in one out of every 3,500 to 4,000 people
worldwide.
RZ-004 removes the mutated Rhodopsin ribonucleic acid (RNA)
and replaces it with a normal rhodopsin gene using the RNA trans-splicing
ribozyme Editing Technology owned by RZ-004 to induce vision damage suppression and
vision recovery. In particular, RZ-004 has a groundbreaking feature that can
correct more than 150 different rhodopsin mutations known so far with a single
treatment.
Australia has excellent medical system and medical staff
capabilities, the number of target patients, patient compliance, and support
from regulatory agencies, said Sung-woo Hong, head of the development division
of Rznomics. "We will expand our clinical development countries to the
United States and Europe, starting with Australia."
The approval of the clinical plan means that it has entered
the clinical stage for genetic diseases that can make good use of the
characteristics of the company's platform technology following the clinical
entry of several anticancer drug pipelines, said Seong-wook Lee, CEO of Rznomics.
"Since the target disease of RZ-004 is a disease with very high unmet
medical demand without any approved treatment, we will do our best to provide
treatment opportunities to suffering patients and improve their quality of
life."2024-07-09 -
First Case in National Strategic Technology Verification System : Rznomics’ ‘RNA Gene Therapy’
The RNA-based new drug development company Rznomics has
been selected as the first case in the national strategic technology
verification system, with their technology ' RNA
Trans-splicing Ribozyme-based gene therapy and Circular RNA Platform.'
The Ministry of Science and ICT and the Korea Institute
of S&T Evaluation and Planning (KISTEP) announced on the 28th that they
have notified the results of the "1st National Strategic Technology
Verification Application."
This system, introduced on March 15th, verifies whether
technologies held or under R&D by research institutions, universities, or
companies fall under national strategic technologies.
A total of 130 applications were received this time, and
the review results notified that one was eligible and five were deferred.
Rznomics' technology was confirmed to fall under the
category of gene delivery technology R&D among national strategic
technologies. This technology can remove target RNA that causes intractable
diseases and express therapeutic RNA, making it a gene therapy technology.
The five technologies deferred for further review were
notified as such due to insufficient evidence and will be re-evaluated upon
resubmission of supplementary data during the next announcement of the
verification system.
Kwon Seok-min, Director General of the Science and
Technology Policy Bureau at the Ministry of Science and ICT, said, "As the
national strategic technology nurturing and support system is still being
established, with public-private efforts to secure national strategic
technologies ongoing, we expect that the number of national strategic
technology verifications will increase in the future."
Applications for national strategic technology
verification are possible quarterly, with the second round of applications to
be conducted in July.
If a company confirmed to possess national strategic
technology has a market capitalization of at least 100 billion KRW and has
raised over 10 billion KRW in venture financing in the last five years, they
can apply for a technology exception listing with just one technology
evaluation rated A or higher.
Previously, it required two technology evaluations, each
rated at least A and BBB, respectively.2024-05-30 -
Rznomics selected for the ‘Deep Tech Incubator Project for Startup’
Development of RNA
trans-splicing ribozyme Editing Technology
"Will grow into an Innovative Therapeutics
Development Company"
Rznomics, a new drug development company based
on RNA trans-splicing ribozyme editing
technology, announced on the 24th that it has been selected as a new company
for the 'Deep tech Incubator Project for Startup (DIPS) 1000+'.
The 'Deep tech Incubator Project for Startup
1000+' project is led by the Korea Institute of Startup & Entrepreneurship
Development under the Ministry of SMEs and Startups. From last year until 2027,
the project aims to select and support startups with unique technological
superiority in ten key future-leading fields such a bio, healthcare, system
semiconductors, and next-generation nuclear power, to foster them into global
unicorns.
Through this
project selection, Rznomics will receive direct business funding of up to 600
million KRW over the next three years, along with linked support for policy
funds, guarantees, exports, and specialized support tailored to industry
characteristics.
Seong-wook Lee,
CEO of Rznomics, said, "We are delighted that the selection for the DIPS
1000+ project has once again recognized the innovation and growth potential of
Rznomics’ technology. With the support from this selection, we will focus on
ongoing clinical trials and technology commercialization to grow into a leading
company in developing innovative therapeutics for various rate and intractable
diseases.2024-05-29 -
Rznomics Signs Clinical Trial Collaboration Agreement With Celltrion for Hepatocellular Carcinoma
Rznomics Signs Clinical Trial Collaboration Agreement With Celltrion for Hepatocellular Carcinoma Combination of RZ-001, Atezlizumab, and Bevaciuzumab to be evaluated for safety and efficacy in Hepatocelluar Carcinoma clinical trial.Rznomics and Celltrion announced on the 14th that they have signed a clinical collaboration agreement to evaluate the combination of RZ-001 and Bevacizumab for hepatocellular carcinoma patients.Through this partnership, Celltrion will provide Rznomics with Bevacizumab (product name Begzelma) and, in return, will receive the right of first negotiation for the Korean regional license after Rznomics’ RZ-001 Phase 1b/2a clinical trial.Rznomics plans to explore the efficacy and safety of adding its RZ-001 to the combination therapy of Atezolizumab and Bevacizumab, the first-time standard treatment for hepatocellular carcinoma, in a Phase 1b/2a clinical trial. Last November, Rznomics entered into a clinical collaboration agreement with Roche, including the free supply of Atezolizumab (product name: Ticentric).By incorporating RZ-001, which employs its proprietary trans-splicing ribozyme-based RNA reprogramming and editing technology, into the combination therapy of atezolizumab and bevacizumab, Rznomics expects to achieve stronger effects and higher response rates.RZ-001, an RNA trans-splicing enzyme-based cancer gene therapy, targets and cleaves telomerase messenger RNA (hTERT mRNA, replacing it with therapeutic gene RNA. It selectively suppresses hTERT expression in cancer cells expressing hTERT and reprograms the hTERT mRNA to encode HSVtk, inducing cytotoxic effects and anticancer activity. Preclinical studies have shown that when used in combination with atezolizumab and bevacizumab, RZ-001 significantly enhances anticancer efficacy in a dose-dependent manner. Sung-woo Hong, vice president and head of development at Rznomics' expressed confidence that RZ-001 could overcome the limitations of the atezolizumab and bevacizumab combination therapy.Seong-wook Lee, CEO of Rznomics, stated, “We are delighted to collaborate with a global biotechnology company like Celltrion,” and added, “We hope this collaboration will help provide new therapeutic options for patients in need.”Sung-hyun Kim, head of medical affairs at Celltion, also emphasized their commitment to continuous efforts in new drug development through partnerships with biotech companies possessing promising technologies and platforms like Rznomics.
2024-05-17 -
Rznomics registered a U.S. patent for its original ‘circular RNA' source technology
Rznomics announced
on the 26th that it has registered a U.S. patent for its foundational
technology platform, the ‘self-circularization RNA structure’ which enables efficient
and simple production of circular RNA, overcoming the limitations of the
existing technologies.
Rznomics has also completed applications in
Europe, Japan, China, Australia, Canada, Singapore, Israel and Brazil.
Circular RNA (circular RNA or circRNA), unlike
linear RNA used in mRNA vaccines, has a closed structure, providing superior
stability against nucleases.
Rznomics has developed a new technology fundamentally
different from the existing ‘PIE(Permuted Intron-Exon) method’ based on the
conventional ‘Group I Intron ribozyme’ for RNA circularization. While the PIE
method leaves unwanted nucleotide sequences in the final product, Rznomics’ RNA
circularization technology ensures that specific nucleotide sequences are
completely absent from the circular RNA after the self-circularization reaction.
Dr. Kyung-hyun
Lee, a senior researcher at Rznomics, stated, "Due to the unique
characteristics of our self-circularization mechanism, various designs can
enhance self-circularization efficiency compared to the PIE method, resulting
in significantly higher self-circularization and purifications yields."
Seong-wook Lee, CEO of Rznomics, stated,
"In the finally registered patent, we have been granted broad rights by
describing the components of the self-circularization RNA structure based on
their functions, without limiting them to specific sequences in the examples.”
He continued, “With the registration of the U.S. patent, we are actively
engaged in research on various applications and indications using circular RNA,
in addition to establishing mass production processes through advanced self-circularization
and purification efficiency improvements for rapid commercialization."2024-03-27 -
Promising Anticancer Gene Therapy Makes Headway
Promising Anticancer Gene Therapy Makes HeadwayFDA designations underscore the potential clinical impact of a pioneering RNA-based gene therapyRznomics, a South Korea-based biopharma, recently announced two significant advancements on their pathway to develop a novel anticancer therapy. Their lead clinical candidate, RZ-001, a viral vector engineered to deliver a proprietary RNA-based trans-splicing ribozyme, is produced leveraging Charles River’s adenoviral vector contract development and manufacturing (CDMO) experience.RZ-001 has the potential to treat several cancer types due to its multi-faceted mechanism of action. The expressed ribozyme is directly cytotoxic against cancer cells by downregulating expression of hTERT and replacing it with an HSV-TK sequence. Additionally, Rznomics has preclinical data indicating that treatment with RZ-001 can provoke an antitumor immune response in animal models.Both the master virus bank and RZ-001 clinical product have been manufactured at Charles River’s viral vector production site in Rockville, MD. “The needs of the program have changed over time, but we’ve been able to adapt the manufacturing process accordingly,” notes James Cody, PhD, Associate Director, Technical Sales and Evaluations. “We will continue to adapt as needed in order to meet the increased clinical need in the future”.Previously approved for Phase I/IIa clinical trials for hepatocellular carcinoma (HCC) in both the US and South Korea, RZ-001 was granted Orphan Drug designation by the FDA, providing access to regulatory assistance from the Office of Orphan Products Development, and has also shown promise against other cancer types.On the Fast TrackRZ-001 was also recently granted FDA Fast Track designation for the treatment of a type of brain tumor known as glioblastoma multiforme (GBM), the most common malignant brain and central nervous system (CNS) tumors accounting for around half of all cases.The gene therapy has been approved for Phase I/IIa trials for GBM treatment in both the U.S. and South Korea, and this significant designation allows for an expedited review process to improve access to patients in critical need."We are proud to receive Fast Track designation from the FDA. This signifies an important milestone in developing a safe and effective treatment for patients with GBM who require new therapeutic options," commented Dr. Seong-Wook Lee, Chief Executive Officer, Rznomics.This latest announcement highlights the opportunity for RZ-001 to make a clinical impact for patients with HCC, GBM, and potentially other types of cancer in the future. For more information on our advanced therapy development and manufacturing services, visit Cell and Gene Therapy CDMO Solutions.
2024-03-18 -
Rznomics Gene Therapy-Based Anti-Cancer Candidate Approved by FDA for New Manufacturing Process
Rznomics has changed the
manufacturing method of its anti-cancer candidate ‘RZ-001’, which is currently
in clinical trials. It has become possible to produce a sufficient amount of
clinical drug for high-concentration administration cost-effectively.
Rznomics announced on the 6th that it has
received approval from the U.S. Food and Drug Administration (FDA) to use the
newly manufactured RZ-001 in clinical trials.
Changes in the manufacturing method for drugs
that have already entered clinical trials under the approval of the IND require
approval from regulatory authorities. An Rznomics official said, "By implementing
the new manufacturing method, we can obtain a greater quantity of finished pharmaceutical
drugs at the same cost, allowing for more efficient conduct of clinical trials
for safety and efficacy validation." They added, "The development of
the new process was led by Rznomics rather than CDMO."
Rznomics' RZ-001 is currently undergoing
clinical trials for hepatocellular carcinoma (HCC) and glioblastoma (GBM). Last
month, it received Orphan Drug Designation(ODD) from the FDA for the treatment of
hepatocellular carcinoma and was granted Fast Track status for glioblastoma.
Bo-mi Kim, head of Rznomics’ RA team, said,
"From a regulatory perspective, the establishment of the new manufacturing
method’s CMC(Chemistry, Manufacturing and Controls) will likely be
significantly beneficial when considering Biologics License Application(BLA)
submissions."
Seong-wook Lee, CEO of Rznomics, also emphasized,
"The CMC(Chemistry, Manufacturing and Controls) is a crucial aspect to
confirm during discussions with global partners for licensing agreements and it
will serve as a highly positive factor from a business perspective."2024-02-06
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