Rznomics

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Rznomics to Lead Next-Generation Gene Therapy with Trans-splicing Ribozyme Platform

Rznomics Inc. (CEO Seong-Wook Lee) held an Investor Relations (IR) conference on the 3rd December, attended by the CEO and key executives, to share its strategic roadmap and vision following its upcoming listing on the KOSDAQ market.Founded in 2017, Rznomics specializes in developing innovative therapies for cancer and rare intractable diseases using its proprietary RNA replacement enzyme-based RNA editing and correction platform. This platform is uniquely capable of performing three functions—targeting RNA cleavage, splicing, and replacement—within a single therapeutic agent. The technology is garnering significant attention from global pharmaceutical companies and academia due to its scalability across various mutations, its safety, and its optimized delivery efficiency tailored to specific indications.The company’s lead pipeline asset, RZ-001, targets hepatocellular carcinoma (HCC) and glioblastoma (GBM). Both indications have received Orphan Drug Designation (ODD) and Fast Track Designation from the U.S. FDA, raising expectations for early commercialization. For glioblastoma, the therapy has been approved for an Expanded Access Program (EAP) and is currently being administered to patients. For hepatocellular carcinoma, Rznomics is conducting clinical trials in combination with immune-checkpoint inhibitors through clinical collaborations with Roche and Celltrion, which provide the necessary therapeutic agents for the trials free of charge.Beyond RZ-001, the company maintains a diverse pipeline addressing high unmet medical needs, including RZ-003 for Alzheimer’s disease and RZ-004 for retinitis pigmentosa (RP), aiming to provide fundamental solutions for these conditions.Rznomics’ technological benefit has been recognized both domestically and globally. Last year, the company was designated as the first "National Strategic Technology Company" and a "National Strategic Technology Possessing and Managing Company" by the Ministry of Science and ICT (MSIT) and the Korea Institute of Science & Technology Evaluation and Planning (KISTEP). Based on these credentials, Rznomics is pursuing the newly established "Deep Tech Special Listing". Upon listing, it is poised to become the first company among those managed by the MSIT to list under this specialized track.In May, Rznomics validated its technological competitiveness and commercial potential by signing a strategic global licensing agreement with Eli Lilly and Company, valued at approximately KRW 1.9 trillion (USD 1.3 billion), to co-develop RNA editing therapies. This partnership is significant as it secures a first-mover advantage in the next-generation gene therapy market with proprietary RNA editing technology.Rznomics plans to utilize the funds raised through its KOSDAQ listing to: advancing its RNA editing platform & accelerating global clinical trials and commercialization of core pipeline assets. Given the high scalability of its technology platform across various diseases, the company intends to expand collaborations with global pharmaceutical leaders through licensing and joint development based on its original patents and core technologies."Rznomics is presenting a new paradigm in the market for intractable cancer and rare disease treatments, where unmet medical needs are high, based on the world's first Trans-splicing Ribozyme platform," said Dr. Seong-Wook Lee, CEO of Rznomics. "We are committed to establishing our technology as the next-generation standard of care and evolving into a global leader in gene therapy, offering hope to patients suffering from intractable diseases."Rznomics is offering 2,060,000 shares for public subscription. The indicative price range is set between KRW 17,000 and KRW 22,500, with a target fundraising amount of KRW 35.0 billion to KRW 46.4 billion. Demand forecasting for institutional investors will take place from November 27 to December 3, followed by public subscription on December 9 and 10. Samsung Securities and NH Investment & Securities are serving as the joint underwriters.

2025-12-03
Rznomics to Participate in ‘BIO-Europe 2025’ to Expand Global Collaborations

Rznomics Inc. announced on the 3rd November that it will officially participate in BIO-Europe 2025, a premier global biopharma conference held in Vienna, Austria, from November 3 to 5. BIO-Europe is the largest partnering event in Europe, bringing together over 5,000 industry professionals from more than 60 countries.During the event, Rznomics plans to showcase its proprietary RNA editing platform and gene therapy development strategies. The company will actively seek opportunities for licensing agreements and co-development collaborations through strategic partnering meetings with global pharmaceutical companies, biotech firms, and investors.Rznomics has garnered significant attention within international academic and industrial circles for its differentiated technological platform: "Trans-splicing Ribozyme-based RNA Editing Therapeutics." In May, the company proved its technological innovation and global competitiveness by signing a research collaboration and licensing agreement with the U.S. pharmaceutical giant Eli Lilly and Company, with a total deal value reaching approximately KRW 1.9 trillion (USD 1.3 billion).As global interest in RNA-based therapeutic technologies continues to rise, Rznomics intends to discuss various collaboration models, including platform-based out-licensing and joint R&D, while further refining its plans for global market expansion through these high-level meetings."Since our major licensing deal with Eli Lilly, interest from leading global companies has surged significantly," said Sungwoo Hong, Vice President of Rznomics, who is attending the event. "Our participation in BIO-Europe 2025 will serve as a catalyst to accelerate additional licensing deals and expand our collaborative network with global industry leaders."

2025-11-04
Rznomics Receives Preliminary KOSDAQ Listing Approval

Rznomics Inc., a biotechnology company specializing in RNA-based gene therapeutics, officially announced on the 22nd Sep that it has received approval for its preliminary listing review from the Korea Exchange (KRX). With NH Investment & Securities and Samsung Securities serving as joint underwriters, the company is now initiating the full-scale listing process with the goal of completing its Initial Public Offering (IPO) by the end of the year.Founded in 2017, Rznomics develops innovative treatments for cancer and intractable rare diseases using its proprietary RNA replacement enzyme-based RNA editing and correction platform.Prior to this approval, Rznomics received "A" grades from two evaluation institutions designated by the KRX. Furthermore, the company was recognized as the first "National Strategic Technology Company" by the Ministry of Science and ICT (MSIT) and the Korea Institute of Science & Technology Evaluation and Planning (KISTEP) in May last year. In September, it was officially designated as a company possessing and managing National Strategic Technology.Leveraging these credentials, Rznomics is set to list via the "Deep Tech Special Listing". It is poised to become the first company among those managed by the MSIT to complete an IPO under this specialized track. Notably, the approval process was expedited, taking only 40 business days—five days shorter than the standard 45-day review period.Rznomics currently maintains a robust pipeline of core assets targeting intractable cancers, including hepatocellular carcinoma (HCC) and glioblastoma (GBM), as well as rare diseases such as Alzheimer’s, hereditary retinitis pigmentosa (RP), and rare neurological disorders. Its lead candidate, RZ-001, an oncology gene therapy, has significantly enhanced its commercialization potential by receiving both Fast Track Designation and Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA).In a major strategic milestone last May, Rznomics signed a global platform-based licensing agreement with Eli Lilly and Company to develop RNA editing therapies, validating the global competitiveness and commercial viability of its platform technology.As domestic and international pharmaceutical companies increasingly seek new modalities—such as ADCs, Targeted Protein Degraders (TPD), and Cell and Gene Therapies (CGT)—Rznomics is expected to achieve rapid growth through further partnerships with global biopharma leaders."Rznomics is developing innovative therapeutics that overcome existing limitations based on the world’s first Trans-splicing Ribozyme platform," said Dr. Seong-Wook Lee, CEO of Rznomics. "Through this KOSDAQ listing, we will open a new paradigm for next-generation RNA editing therapies on the global stage."

2025-09-22
Rznomics’ Anti-cancer Gene Therapy, RZ-001, Selected for National New Drug Development Project Supporting Clinical Development

Rznomics, a biopharmaceutical company specializing in RNA-based gene therapies (CEO Seong-wook Lee), announced today that its lead anti-cancer candidate, RZ-001, has been selected as a clinical-stage project for the “2025 1st National New Drug Development Project” by the Korea Drug Development Fund (KDDF).The National New Drug Development Project is a pan-governmental R&D initiative aimed at strengthening the global competitiveness of the Korean pharmaceutical and biotech industries. Following this selection, Rznomics will receive R&D funding for the next two years to support the clinical development of RZ-001 for the treatment of Glioblastoma (GBM).Glioblastoma is the most aggressive and fatal form of glioma, an intractable brain tumor with a five-year survival rate of less than 10%. As the most common primary brain tumor in adults, it affects approximately 3 to 4 per 100,000 people. Despite standard-of-care treatments—including surgery, chemotherapy, and radiation therapy—the recurrence rate remains high, representing a significant unmet medical need. Notably, the field has seen a lack of effective new therapies for over a decade, creating an urgent demand for treatments with innovative mechanisms of action.RZ-001 is a next-generation gene therapy that specifically targets and cleaves human telomerase reverse transcriptase (hTERT) mRNA, which is overexpressed in cancer cells. It then replaces the targeted sequence with a therapeutic RNA to induce selective apoptosis (cell death) of the cancer cells. This process is powered by Rznomics’ proprietary RNA trans-splicing ribozyme technology.Currently, RZ-001 is undergoing Phase 1 clinical trials for GBM in South Korea. With the recent Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA), Rznomics expects to accelerate its global clinical development.The innovation and safety profile of RZ-001 have been recognized by the U.S. FDA through both Orphan Drug Designation (ODD) and Fast Track status. Furthermore, RZ-001 is being administered under an Expanded Access Program (EAP), which allows the use of investigational drugs for patients with serious conditions who have no comparable alternative therapies. This ongoing clinical experience is anticipated to have a positive impact on patient recruitment for upcoming clinical trials in the United States.“The selection for this national project will serve as a powerful catalyst for the global clinical advancement of RZ-001,” said Seong-wook Lee, CEO of Rznomics. “Leveraging our long-standing research expertise in trans-splicing ribozymes, we are dedicated to proving the potential of RZ-001 as a First-in-Class anti-cancer gene therapy.”

2025-06-20
Rznomics Passed Technology Evaluation, Advancing Toward KOSDAQ Listing

- Received “A, A” ratings from two technology evaluation institutions designated by the Korea Exchange- Plans to submit preliminary review application for KOSDAQ listing in the second half of the yearRznomics, a developer of RNA-based gene therapies, announced on June 11 that it has passed the technology evaluation required for a technology-special listing on the KOSDAQ market.The company received “A” grades from two technology evaluation institutions designated by the Korea Exchange (KRX), recognizing both its technological excellence and business potential. Rznomics plans to leverage the Deep Tech Track of the technology-special listing system starting in the second half of the year, with the goal of completing its KOSDAQ listing in the first half of next year.The technology evaluation is a mandatory step for the technology-special listing and assesses technological maturity, competitiveness, commercialization potential, and marketability. Under the Deep Tech Track—a newly introduced fast-track system—companies based on advanced science and national strategic technologies can apply for preliminary listing review with a single technology evaluation.In May 2024, Rznomics was recognized as the "National Strategic Technology Company No. 1" by the Ministry of Science and ICT (MSIT) and the Korea Institute of Science and Technology Planning and Evaluation (KISTEP). Later in September, the company was designated as a "National Strategic Technology Possession and Management Company," thereby qualifying for the Deep Tech Track. If successfully listed through this route, Rznomics would become the first company designated under MSIT’s national strategic technology program to achieve listing via the deep tech special track.Founded in 2017, Rznomics is developing anti-cancer and rare disease therapies using its proprietary RNA editing and correction platform based on RNA replacement enzymes. Founder and CEO Dr. Seong-Wook Lee began his RNA replacement enzyme research at Duke University Medical Center in the U.S., and has continued this work for over 20 years as a professor at Dankook University since 1997.Currently, the company is conducting Phase 1b/2 and Phase 1/2a clinical trials in South Korea and the United States for “RZ-001,” a gene therapy targeting hepatocellular carcinoma (HCC) and glioblastoma. Both indications have received Orphan Drug Designation (ODD) and Fast Track designation from the U.S. Food and Drug Administration (FDA). For glioblastoma, the therapy is also being provided to patients under an Expanded Access Program (EAP) due to the lack of alternative treatments. In addition, “RZ-004,” a therapy for hereditary retinitis pigmentosa, has received approval for a Phase 1 trial in Australia, with patient enrollment expected to begin soon.In May, Rznomics signed a strategic global licensing agreement with U.S. pharmaceutical company Eli Lilly and Company to co-develop RNA editing therapies, further validating the global competitiveness and commercial potential of its platform technology. The company is also expanding its scope beyond RNA replacement enzymes to include circular RNA platforms and is collaborating with various companies on vaccine and therapeutic development and validation.Despite a challenging biotech investment environment in the second half of last year, Rznomics successfully raised KRW 20.3 billion (around USD 15M) in a Pre-IPO Series round from investors including KB Investment, Aon Investment, Yuanta Investment, Samsung Venture Investment, and Quad Ventures, demonstrating its strong market potential.CEO Dr. Seong-Wook Lee commented, “Passing the technology evaluation marks official recognition of Rznomics’ proprietary RNA-based platform and its growth potential,” adding, “Through our KOSDAQ listing, we aim to establish a solid foundation for business expansion and become a global leader in RNA editing.”

2025-06-11
Rznomics Enters Global Licensing Agreement with Eli Lilly for RNA-editing Therapeutics

Co-development of precision RNA therapeutics for inherited hearing loss treatmentRznomics Inc., a South Korea-based biopharmaceutical company specializing in RNA-based therapeutics, announced today that it has entered into a strategic global research collaboration and licensing agreement with Eli Lilly and Company (NYSE: LLY) to develop and commercialize novel RNA-editing therapies using Rznomics' proprietary trans-splicing ribozyme platform.The collaboration focuses on the discovery and development of RNA-editing therapeutics for sensorineural hearing loss. Rznomics will conduct early-stage research according to the jointly approved research plans, while Lilly will assume responsibility for further development and commercialization.If Lilly exercises all available options under the agreement, the total deal value could reach more than $1.3 billion, as well as separate royalties on product sales. In line with the mutual understanding between the two companies, the upfront payment was not disclosed. “We are thrilled to collaborate with Lilly, a global leader in pharmaceutical innovation,” said Dr. Seong-Wook Lee, CEO of Rznomics. “This partnership validates our trans-splicing ribozyme platform and opens the door to treating previously intractable diseases with precision RNA therapeutics. Together with Lilly, we aim to accelerate the development of transformative therapies for patients around the world.”This partnership marks a significant milestone for Rznomics as it seeks to expand its presence in the global biotech arena. For Lilly, the deal aligns with its broader strategy to build a leading RNA therapeutics pipeline and address high unmet needs in hearing loss and other therapeutic areas.About Rznomics Rznomics is a clinical-stage biopharmaceutical company based in South Korea focused on developing RNA-based gene therapies. The company’s proprietary trans-splicing ribozyme platform enables precise RNA editing and has broad applicability across multiple indications.

2025-05-15
Rznomics Develops the World’s First RNA Editing Therapy: “TSR will be the Standard Treatment”

Rznomics develops
anticancer drug and rare disease therapies using the world’s first RNA editing
platform, Trans-Splicing Ribozyme(TSR). Through a special track, the company aims
to be listed on KOSDAQ market as early as this year, or next year at the latest.
Seong-Wook Lee, CEO of Rznomics said, “TSR has a potential for expanding to a package-deal
with asset and platform technology.” He added, “We will sign a licensing-out contract
soon, and apply for a technology evaluation.”A Pioneer of
RNA Editing Technology

In 2017, Dr. Lee
established Rznomics Inc., after 20 years of TSR research. Since 1994, he began
researching TSR during his postdoctoral fellowship at Duke University Medical
Center, one of the institutions initially developed and published TSR
technology. At that time, he focused on RNA-based autoimmune disease therapeutics
and gene therapy, which was a novel approach. The work was reported on the cover
of Nature Biotechnology, January 1997. After then, he returned to Korea
and joined Dankook University as a professor.

Dr. Lee continued
developing TSR in Korea but it was a challenge, because many scientists shifted
their path to RNA interference (RNAi) research, which was newly discovered in
the early 2000s.

“When I
developed TSR at the first time, it was not enough to verify its specificity
and efficacy. Moreover, in vivo delivery systems required more study. In the
late 2000s, I was finally able to achieve tumor-specific treatment data with
animal models. Gene therapy using virus vector as delivery system was tested in
the market, so it accelerated TSR development.”

The R&D
project was on track as Rznomics was established in 2017. Dr. Lee emphasized, “Not
only the platform, but we will make a meaningful deal with our own asset based
TSR technology. We’ve already organized the development system and clinical/pre-clinical
Pipelines.”

Safer than
any other Gene Editing

TSR technology
is specifically designed to avoid direct DNA editing. It removes targeted RNA
and then replaces it with therapeutic RNA; it demonstrates the possibility of dual
function with a single substance. It also can be programmed to deliver customized
drugs to the target gene.

Depending on the
indication, it utilizes proper viral vectors. Adenovirus is used for temporary
treatment including cancer therapy, and adeno-associated virus (AAV) is used
for one-shot genetic cure.

TSR technology
is frequently compared with CRISPR gene editing, one of the promising platforms
in the gene therapy field. Although CRISPR therapy provides the correction of
abnormal genes, it causes concerns that might be functioned at off-target permanently.
At present, there is no approved in vivo gene therapy yet.

Dr. Lee
explained that TSR shows off-target effects rarely. “If we try to edit a specific
part of RNA A, but RNA B works normally and will be produced in the cell. Even
if TSR inadvertently targets RNA B, but any off-target effects are transient
and limited.”

RNAi
therapeutics inhibit target RNA for treatment, but there is a limited option
and the current delivery system can reach liver cells only. For that reason,
most global RNAi pipelines target liver-related diseases and they have limited
potential to expand the target indication to other oncology.

Rznomic’s lead pipeline
RZ-001 is in Phase 1b/2a trials for hepatocellular carcinoma (HCC) in the U.S.
and Korea, and in Phase 1/2a for glioblastoma multiforme (GBM). The U.S. FDA
has granted RZ-001 Orphan Drug Designation (ODD) and Fast Track program for
both indications. Moreover, RZ-001 received approval for an Expanded Access
Program (EAP) in GBM, allowing medication prior to official approval to the patients
who have no approved treatment.

Rznomics plans
to enter a combination clinical trial with immunotherapy and already contracted
to Roche and Celltrion for receiving Tecentriq and Vegzelma (Avastin
biosimilar) for free of charge. Tecentriq and Avastin are the FDA-approved first-line
therapy for HCC.

Dr. Lee noted,
“In preclinical studies, RZ-001 improved biomarkers associated with
immunotherapy. The result encouraged our partners to pay attention to the potential
of RZ-001.”

The company is
accelerating the development of rare disease therapeutics with TSR. Dr. Lee
said, “Not only the cancer therapy, but TSR is also competitive for rare and
incurable diseases treatment. It comes from the unique property of TSR, which
appears one-source multi-effect. TSR regulates gene expression at the RNA level,
exhibiting high specificity and efficacy without exogenous protein or intracellular
mechanisms. It also has the potential to correct multiple mutations with a
single enzyme, meeting medical unmet needs related to rare and incurable
diseases.”

Rznomics is
building a pipeline RZ-004 for inherited retinitis pigmentosa (RP) treatment
which is one of rare genetic disorders. RP is a progressive inherited degenerative
disease leading to vision loss due to photoreceptor damage and it occurs in 1
in 3,500~4,000 people globally.

30% of autosomal
dominant RP cases are caused by mutations in rhodopsin gene. Rhodopsin,
pigment-containing sensory protein converts light into an electrical signal.
More than 150 rhodopsin mutations have been identified so far, and each mutation
can trigger serious eye disease including retinitis pigmentosa. Current
therapeutic pipelines typically target individual rhodopsin mutations, limiting
their applicability to less than 10% of Western patients; the clinical trial is
suspended at the moment.

It is notable
that RZ-004 can target all rhodopsin mutations by replacing mutant RNA with
normal RNA. Its proprietary engineering skills control the gene expression
level according to the mutation level. There are no competitors in the market,
so once the company passed Phase 2 clinical trial, then it can apply to Accelerated
Approval Program. Rznomics has already received IND approval for RZ-004 in
Australia and patient recruitment will begin within 2025.

A New
Circular RNA Platform

To overcome
limitations of current RNA platforms, Rznomics developed a novel circular RNA
platform. Most RNA-based vaccines and therapeutics are based on linear RNA,
which is vulnerable to Ribonuclease due to its open structure.

Circular RNA, single-stranded
RNA that forms a covalently closed loop, expresses high stability and resistance
against Ribonuclease. Though it is still in early stages globally, this
technology is spotlighted as a new platform to develop vaccines and
therapeutics.

Ribozyme-based
circular RNA synthesis is more prevalent than purely chemical or enzymatic
methods due to its efficiency and scalability. Ribozyme-based technologies rely
on the Permuted Intron-Exon (PIE) approach, which has a limitation that PIE leaves
unintended sequences in the final output. On the other hand, Rznomic’s circular
RNA, based on the Tetrahymena group I ribozyme shows higher efficiency compared
to existing methods.

“Our circular
RNA platform overcame limitations of the previous technologies. What is more
important, it produces the equivalent or higher gene expression compared to PIE,”
Dr. Lee mentioned.

Top Priority:
Recruiting Industry Experts

When Dr. Lee
started the business, his top priority was recruiting experts. “Fortunately, I’m
working with top experts in each field. Thanks to them, I could handle pre-clinical
and clinical development, financial operation which I’m not good at.”

Seongwoo Hong, vice
president of Rznomics, has over 20 years of business development experience in domestic
and global pharmaceutical companies, successfully leading multiple IND filings
and approvals. He is managing the entire development process, including RA, CMC.
Seung-Ryul Han, Head of R&D Center, has researched TSR for a long time
through his BSc, MSc, and PhD at Dankook University. As leading numerous
R&D projects, he acquired a reputation as a specialist in TSR field.

CFO Jong-sun Rim
has over 13 years of experience at PwC Korea, supporting IPO preparations,
internal control advisory, and financial consulting for both public and private
companies.

Making TSR
technology a global standard,
that is Dr. Lee’s vision. He emphasized, “TSR will become an essential tool for
DNA and RNA editing industries. In special indications, it will be the standard
treatment in the near future.”

He is looking
forward to commercializing therapeutics Rznomics researched and developed. “Within
10 years, we will build an in-house manufacturing system from initial research
to GMP production. It will be the stepping stone that Rznomics grow as a global
biopharmaceutical company.”

RNA Editing
Technology Emerges as a Novel Tool for Cancer Gene Therapy

Published in Molecular Therapy – Nucleic Acids (Journal
of the American Society of Gene and Cell Therapy)

Title of the
Study: Targeted Suicide
Gene Therapy for Liver Cancer Based on Ribozyme-Mediated RNA Replacement
Through Post-Transcriptional Regulation

This study aimed
to develop a novel gene therapy for hepatocellular carcinoma (HCC) using a
ribozyme-mediated RNA replacement strategy, leveraging adenovirus-delivered
Tetrahymena group I trans-splicing ribozymes. The therapeutic approach induces
selective apoptosis in cancer cells by targeting human telomerase reverse
transcriptase (hTERT) RNA, which is abundantly expressed in tumor cells, and
replacing it with a suicide gene transcript. Post-transcriptional regulatory
elements were introduced to enhance therapeutic specificity and minimize
toxicity to normal hepatocytes.

To confer
tumor-specific cytotoxicity, the authors engineered the ribozyme to catalyze
trans-splicing of hTERT RNA, replacing it with a transcript encoding Herpes
Simplex Virus thymidine kinase (HSV-tk). Expression and stability of the
therapeutic RNA were improved by incorporating splicing donor and acceptor
(SD/SA) sequences and the Woodchuck Hepatitis Virus Post-transcriptional
Regulatory Element (WPRE). Additionally, to restrict ribozyme activity to
malignant hepatocytes, a target site complementary to miR-122a—a liver-specific
microRNA—was added (miR-122aT), thereby suppressing expression in normal liver
cells.

The optimized
ribozyme showed potent and selective cytotoxicity toward HCC cells in vivo.
In intrahepatic multifocal HCC mouse xenograft
models, the therapy significantly inhibited tumor growth. Preclinical
toxicology and biodistribution studies demonstrated minimal hepatotoxicity, underscoring
the safety and translational potential of this platform.

These findings
highlight ribozyme-mediated trans-splicing RNA replacement as a promising
next-generation strategy for gene therapy in HCC, offering selective tumor
targeting while sparing healthy liver tissue—an essential advancement over
conventional approaches.

This study was
featured as a highlighted article in Molecular Therapy – Nucleic Acids,
a leading journal published by the American Society of Gene and Cell Therapy
(ASGCT).

Rznomics’ TSR
(Trans-Splicing Ribozyme) platform has garnered international recognition. It
was identified as the first RNA editing technology to enter clinical
development in the 2023 Nature Biotechnology RNA editing spotlight
issue, and was cited by Nature in 2024 as one of only three RNA editing
platforms to have progressed to clinical trials.

2025-04-11
Rznomics: "The World's First RNA Editing Technology for Developing Treatments for Incurable Cancers"

Safer Than Direct Gene Therapy,
Enhanced Specificity for Anticancer Drug Development

Aiming for IPO Through Licensing-Out
Within the Year, "Platform Deals Also Possible"

"We aim to develop the world's first treatment
for incurable cancers using RNA editing technology."

In an interview on March 7, Seongwook Lee,
CEO of Rznomics, stated, "Our proprietary RNA editing platform, Trans-Splicing
Ribozyme (TSR), simultaneously expresses therapeutic RNA while
suppressing unintended RNA expression."

Rznomics was founded in 2017 after CEO Lee
dedicated over 20 years to TSR research. He began studying TSR in the 1990s as
a postdoctoral researcher at Duke Medical Center in the U.S., where the first
research papers on TSR were published.

However, the emergence of RNA interference
(RNAi) in the 2000s led to a decline in TSR development momentum. As global
interest in RNAi-based therapeutics surged, many scientists who had been
working on TSR shifted their focus to RNAi research. Lee returned to Korea and
continued his TSR research as a professor at Dankook University.

"When TSR was developed at first, it
lacked specificity and efficacy, and there was little research on delivery
vectors for in vivo applications," he explained. "However, in the
mid-2000s, through advancements in bioengineering, we achieved the ability to
target specific genes for cancer treatment. The emergence of gene therapies
utilizing viral vectors as drug delivery systems further accelerated TSR
development."

A Safer Alternative to Gene Editing

Gene-editing therapies such as CRISPR gene
scissors pose concerns about irreversible genetic modifications if unintended
targets are altered. Furthermore, there are currently no treatments that enable
gene editing inside the human body post-administration. RNAi therapies also
face limitations, as existing drug delivery systems are primarily designed for
targeting liver cells, requiring further research for development as anticancer
drugs.

On the other hand, TSR does not directly
modify DNA. Instead, it removes targeted RNA and replaces it with therapeutic
RNA. Rznomics designs optimized drug delivery systems for each disease,
allowing precise targeting of the intended site. Depending on the indication, adenoviral
vectors are used for temporary effects, while adeno-associated virus (AAV)
vectors are used for long-lasting, one-time treatments.

Lead Pipeline: RZ-001

Rznomics’ lead pipeline candidate is
RZ-001, an anticancer drug currently in clinical trials. In both the U.S. and
Korea, RZ-001 is undergoing Phase 1b/2a trials for hepatocellular carcinoma
(HCC) and Phase 1/2a trials for glioblastoma (GBM). The U.S. Food and Drug
Administration (FDA) has granted Fast Track designation for both indications.
Additionally, the glioblastoma program has been designated under the Expanded
Access Program (EAP), which allows patients with no available treatment options
to receive the investigational drug.

RZ-001 is also set to enter combination
trials with immune checkpoint inhibitors. Rznomics has secured free-of-charge
supply agreements for Roche’s Tecentriq and Celltrion’s Vegzelma (Avastin
biosimilar) for domestic combination studies with RZ-001. Tecentriq and Avastin
have already been approved by the FDA as a first-line treatment for
hepatocellular carcinoma.

"In preclinical studies, RZ-001 showed
improvements in biomarkers associated with immune checkpoint inhibitor
responses," Lee said. "Due to the anticipated synergy with Tecentriq
and Avastin, we were able to secure these drugs free of charge for our
trials."

IPO Target and Global Licensing Plans

Rznomics aims to list on the KOSDAQ stock
market through a special listing track as early as this year or by next year at
the latest. "Our TSR technology offers not only pipeline assets but also platform
licensing opportunities," Lee emphasized. "We plan to achieve licensing-out
deals within the year and apply for a technology evaluation to proceed with our
IPO."

2025-03-12
Rznomics’ Liver Cancer Treatment Receives FDA Fast Track Designation

Gene Therapy-Based Cancer Drug Candidate RZ-001 Gains Fast Track Status for Hepatocellular Carcinoma, Following GlioblastomaRznomics announced on the 17th that its gene therapy-based cancer drug candidate, RZ-001, has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of hepatocellular carcinoma.This is the second Fast Track designation for RZ-001, following its 2023 designation for glioblastoma, an aggressive and hard-to-treat brain cancer.Fast Track is an FDA program designed to expedite the development and review of drugs intended for serious or life-threatening conditions. It allows companies to have more frequent communication with the FDA, benefit from priority review, and submit their application for rolling review—where different sections of the application are reviewed as they become available, rather than waiting for the full submission—potentially speeding up the approval process.RZ-001 is being developed using Rznomics' proprietary RNA editing and correction platform, which is based on ribonucleic acid substitution enzymes. The company is currently conducting Phase 1b/2a clinical trials for hepatocellular carcinoma and Phase 1/2a trials for glioblastoma in South Korea, with FDA approval to conduct the same trials in the United States.For glioblastoma, Rznomics has also received FDA approval for an Expanded Access Program (EAP), allowing compassionate use of RZ-001. This program is currently being conducted at Harvard University Hospital.Seong-wook Lee, CEO of Rznomics, stated, "This Fast Track designation reaffirms the potential of RZ-001 as an innovative cancer treatment. We are committed to accelerating clinical development to provide effective treatment options for patients suffering from difficult-to-treat cancers."

2025-02-17

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