News
TOTAL89 (1/1 page)
-
Rznomics "Hepatocellular Carcinoma Drug ‘RZ-001’ Designated as an Orphan Drug by FDA"
Phase 1/2a
clinical trial in patients with liver cancer that does not respond to transarterial
chemoembolization
Rznomics (CEO Seong-wook Lee) announced on the
22nd that its developmental cancer treatment, “RZ-001(developmental code name),
targeting hepatocellular carcinoma, has received Orphan Drug Designation from
the U.S. Food and Drug Administration (FDA).
With Orphan Drug Designation, the company
becomes eligible for benefits such as tax credits for research and development
expenses, exemption from approval application fees, and a seven-year market
exclusivity period starting from the approval date. The designation is expected
to provide favorable conditions during the priority and accelerated review
decisions.
RZ-001 is currently undergoing Phase 1/2a
clinical trials, having received approval for its clinical trial plan from the Ministry
of Food and Drug Safety in Korea and the FDA. The trial focuses on patients
with hepatocellular carcinoma who do not respond to transarterial
chemoembolization(TACE) and aims to develop a treatment option that can be
prioritized before using systemic therapies.
Seong-wook Lee, CEO of Rznomics, said, "Effective
treatments for hepatocellular carcinoma are urgently needed at various stages,
and we will do our best to make RZ-001 a new therapeutic option."
In addition to the ongoing clinical
development, Rznomics has expanded the application of RZ-001 to include glioblastoma,
receiving Fast Track designation from the FDA in November last year. The company is actively
engaged in global clinical development through collaborations with
pharmaceutical companies, including a clinical partnership with Roche.2024-01-23 -
Rznomics Launches Clinical Trials in Australia for Genetic Retinal Disease Treatment
'RZ-004' Approved by OGTR in
Australia... Plans to expand clinical development countries in the future
Rznomics (CEO Seong-wook Lee) has announced on
January 16th that it has received approval for the review of the phase 1
clinical trial for ‘RZ-004’ in Australia, a therapeutic candidate for genetic
retinal diseases, including Retinitis Pigmentosa. The review is being conducted
by the Office of the Gene Technology Regulator (OGTR) in Australia.
Retinitis Pigmentosa is a rare and incurable
genetic retinal disorder where visual cells progressively deteriorate, leading
to a narrowing field of vision and eventual vision loss. It occurs globally at
a rate of approximately 1 out of every 3,500 to 4000 people.
‘RZ-004’
aims to eliminate mutated Rhodopsin RNA by using ‘Trans-splcing ribozyme’, a
RNA correction platform technology owned by Rznomics. Notably, Rznomics
explains that its technology can correct over 150 different known mutations
with a single therapeutic agent.
Sung-woo Hong, the Head of
Development at Rznomics, stated, “Australia possesses elements conducive to the
early clinical development of genetic ocular diseases, such as an excellent
healthcare system, medical expertise, patient numbers, patient compliance and
regulatory support.” He further mentioned plans to expand clinical development
to the United States and Europe, with Australia as the starting point.
Seong-wook Lee, the CEO of
Rznomics, emphasized the significance of ‘RZ-004’ by highlighting the lack of
approved treatments and the scarcity of ongoing therapeutic developments for
the targeted condition. He expressed the company’s commitment to providing
treatment opportunities for patients suffering from diseases and enhancing
their quality of life through swift development efforts.2024-01-17 -
Rznomics’s Liver Cancer Treatment, Clinical trials in combination with immuno-oncology drugs
Rznomics, a gene therapy development company, announced that its Phase 1b/2a clinical trial plan (IND) for the administration of liver cancer drug candidate “RZ-001” along with immune-oncology drugs has been recently approved by the Ministry of Food and Drug Safety.Rznomics plans to evaluate the efficacy and safety of “RZ-001” as a first-line treatment in about 50 patients diagnosed with hepatocellular carcinoma along with the immune-oncology drug ‘Ticentric’ and the targeted therapy ‘Avastin’. ‘Ticentric’ and ‘Avastin’ are Roche’s most widely used anti-cancer drugs for the first-line treatment of hepatocellular carcinoma. Roche will provide Rznomics for the ‘Ticentric’ to be used in this clinical trial.RZ-001 received MFDS and FDA clinical trial approval for HCC and GBM as a single administration therapy.Rznomics is conducting an independent clinical trial of RZ-001 in patients with hepatocellular carcinoma.
2024-01-04 -
Ministry of SMEs and Startups(MSS), Open Innovation Performance Sharing Meeting
The Ministry of
SMEs and Startups held the 2023 Public-Private Cooperation Open Innovation
Performance Sharing Meeting at JW Marriott Dongdaemun Square in Jung-gu, Seoul
on the 7th to spread the results of open innovation (open innovation)
performance.
The public-private partnership open innovation
project is a project that discovers and links the demand for collaboration
between large companies and startups, and to activate open innovation with
follow-up support from the government. This year, it supported the
collaboration of 87 startups. Selected startups received up to 100 million won
in funds for commercialization of collaboration, mentoring, and R&D
support.
At the event, 37 companies, including
outstanding collaborative start-ups and large companies, were awarded. Eight
people, including Jae-hwan Park, manager of SK Telecom, Lee Jung-joong, head of
Lotte World at Hotel Lotte, and Kim Sung-sung, manager of the Seoul Creative
Economy Innovation Center, received the MSME Minister’s Commendation. Lotte
World, May Eye, Moorim P&P, and YooSeung In Nature Nature were introduced
as outstanding examples of collaboration
.
Five companies, including Thoth, CA Lab,
Rznomics, The Wave Talk, and MANGOSLAB, were selected as support companies for
the Super Gap Startup Open Innovation Challenge. The Ministry of SMEs and
Startups selected 5 start-ups with high potential through cooperation with
large companies in the 10 Super-gap fields and provide them up to 50 million
won in collaboration funds and preferential benefits when applying for next
year's Super-gap project.
Lim Jeong-Wook, head of the Startup and
Venture Innovation Office at MSME, said, "Open innovation has now
established itself as one of the pillars of mutual growth between large
companies and startups," and added, "We will continue to create a
win-win collaboration ecosystem where large companies and startups can grow
together as a team."2023-12-11 -
Rznomics Inc. Secures Fast Track Designation from the U.S. FDA for RZ-001
SEONGNAM-SI, South Korea , Nov. 10, 2023 /PRNewswire/ -- Rznomics Inc., a South Korea based biopharmaceutical company specialized in the development of RNA-based gene therapeutics, announced today that it has received Fast Track Designation from the U.S. Food and Drug Administration (FDA) for RZ-001, for the treatment of patients with Glioblastoma (GBM).Fast Track Designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and address unmet medical needs, thus enabling drugs to reach patients sooner. If relevant criteria are met, the drug with Fast Track Designation may also be eligible for Accelerated Approval and Priority Review.GBM is known as the most malignant tumor in Central Nervous system with high mortality rate but lacks effective therapies. RZ-001, the RNA replacement enzyme-based cancer gene therapy for the treatment of GBM, targets and cleaves hTERT mRNA and replaces the mRNA with the therapeutic gene RNA. This induces anti-cancer activity and cytotoxic effect by trans-ligating an HSVtk-encoding sequence into the reprogrammed hTERT mRNA. RZ-001 has demonstrated very promising responses in preclinical studies in which an enhanced anti-cancer efficacy and a higher survival rate was observed."We are proud to receive Fast Track Designation from the FDA," said Dr. Seong-Wook Lee, Chief Executive Officer of Rznomics. "This designation signifies an important milestone in developing a safe and effective treatment for patients with GBM who are in need of new therapeutic options."Rznomics received Phase I/IIa IND approval for RZ-001 from the FDA and the South Korean Ministry of Food and Drug Safety (MFDS) in Glioblastoma and the clinical trial will investigate the safety, tolerability, and efficacy of RZ-001 in patients with GBM. Subjects will be treated with RZ-001 according to the planned dose escalation design.About RZNOMICSAs a biopharmaceutical company founded in the laboratory of Professor Seong-Wook Lee, Dankook University Department of Bioconvergence Engineering, Rznomics is researching with the goal of developing new RNA-based gene therapeutic bio-drugs for cancer and incurable diseases. Rznomics' core platform technology is based on an RNA replacement enzyme, known as trans-splicing ribozyme, which can edit target RNA through simultaneous destruction and repair (and/or reprogramming) to yield the desired therapeutic RNA, thus, selectively inducing therapeutic gene activity in cells expressing the target RNA.Rznomics' lead candidate (RZ-001) is a treatment for Hepatocellular Carcinoma & Glioblastoma (Phase 1/2a IND approval from both in South Korea and in the U.S.), with other treatments for Alzheimer's disease and hereditary retinal dystrophy (RD)For more information, please visit https://www.rznomics.com/SOURCE Rznomics Inc.
2023-11-13 -
RZNOMICS Inc. Announces Clinical Trial Collaboration and Supply Agreement to Evaluate RZ-001 in Combination with anti-PDL1 in liver cancer patients
SEONGNAM, South Korea, Nov. 10, 2023 /PRNewswire/ -- Rznomics Inc., a South Korea based biopharmaceutical company specialized in the development of RNA-based gene therapeutics, announced today that it has entered into a clinical collaboration agreement with F. Hoffmann-La Roche Ltd (Roche) to study RZ-001, a gene therapy approach utilizing the company's proprietary trans-splicing ribozyme-based RNA reprogramming and editing technology, in combination with Roche's atezolizumab, in patients with hepatocellular carcinoma (HCC). Under the terms of the agreement, Roche will provide atezolizumab to be used in combination with RZ-001 for the planned Phase 1b/2a study.RZ-001, the RNA replacement enzyme based cancer gene therapy, targets and cleaves hTERT mRNA and replaces the mRNA with the therapeutic gene RNA. Specifically, RZ-001 induces anti-HCC activity by suppressing hTERT expression selectively in cancer cells, which express hTERT, and simultaneously inducing a cytotoxic effect by trans-ligating an HSVtk-encoding sequence into the reprogrammed hTERT mRNA. It also induces effective immune cell infiltration into treated tumors and reduces VEGF expression in the tumors, which leads to the anti-cancer efficacy and transformation of tumor microenvironment to be more responsive to immunotherapy.RZ-001 has demonstrated therapeutic activity in preclinical studies and significant improvement in anti-cancer efficacy in a dose dependent manner in combination with atezolizumab. The clinical study will evaluate the safety and efficacy in liver cancer patients and it is anticipated that the combination of RZ-001 with atezolizumab may enhance response rate and anti-cancer efficacy that could benefit patients with HCC."We are excited to initiate collaboration with longstanding oncology leader, Roche, to further explore the potential benefits of combining checkpoint blockade with RZ-001," said Dr. Seong-Wook Lee, Chief Executive Officer of RZNOMICS. "We look forward to having this collaboration, evaluating combination therapy and continuing our mission to offer innovative therapies for patients in need."About RZ-001RZ-001 is the first Group I intron based trans-splicing ribozyme-based RNA reprogramming approach approved by the FDA for evaluation in patients. The treatment was developed utilizing Rznomics' proprietary RNA reprogramming and editing technology and takes the form of an adenoviral vector that expresses an hTERT targeting trans-splicing ribozyme to treat hepatocellular carcinoma (HCC) patients. HCC is the most common type of primary liver cancer, accounting for 80 percent of cases worldwide.Rznomics received Phase I/IIa IND approval for RZ-001 from the FDA and the South Korean Ministry of Food and Drug Safety (MFDS) in HCC and Glioblastoma as well.About Rznomics Inc.Rznomics Inc., is a biotech company specialized in the development of RNA-based gene therapies for cancers and intractable diseases. Founded in 2017, its RNA replacement enzyme platform is being developed for numerous indications such as hepatocellular carcinoma, glioblastoma, retinitis pigmentosa, rett syndrome and alzheimer's disease.For more information, please visit https://www.rznomics.com/ Media Contact:rznomics_bd@rznomics.com SOURCE Rznomics Inc.
2023-11-13 -
Rznomics selected as one of the 50 Best Companies in excellent Technology Development for Small and Medium-sized Businesses
Rznomics announced on the 18th that it has been selected as ‘Top 50 Companies in excellent Technology Development for SMEs’ by Korean ‘Ministry of SMEs and Startups.’
The 50 Best Technology Development for SMEs is
in its first year to promote innovation, quality advancement, and
commercialization of excellent technology development for SMEs.
The 50 Best
Technology Development for SMEs evaluates and selects 30 companies with
excellent commercialization performance, including cumulative sales of
technology development projects, ▲ 6 companies that achieved world-class
technology in the national strategic technology field ▲ 7 companies that
succeeded overcoming technology takeover and management crisis ▲ 7 innovative
product companies that contributed to improving public services such as
industrial safety over the past five years (2018-2023).
For its selected grant projects by the
Ministry of SMEs and Startups, Rznomics has developed RNA replacement enzymes
that can be used as selective and specific treatments as a new way to replace
genes at the mRNA level. Based on the aforementioned development, Rznomics is
selected as one of the final six companies in the national strategic technology
sector in recognition of its excellence and differentiation of technology
development performances, with performances such approval of the clinical trial
plan (IND) of development of liver cancer gene therapy by Ministry of Food and
Drug Safety and U.S. Food and Drug Administration, domestic and international
patent registration, and verification of anticancer efficacy by independent
administration.
Based on the same platform technology recognized, mentioned above, Rznomics has also obtained IND approval from the Ministry of Food and Drug Safety and the FDA for glioblastoma (malignant brain tumor) treatments. And Rznomics is preparing to conduct clinical trials within this year.
In addition,
IND applications for the treatment of autosomal dominant formulation hereditary
retinopathy, which is first pipeline of Rznomics to treat genetic rare disease
currently without treatment, is also being prepared to be submitted in early
2024.
Seong-wook Lee, CEO of Rznomics, said, "I
am pleased to be able to recognize Rznomics' technology for its excellence and
selection as a top 50 company. We will use this opportunity to promote our
technology more widely and do our best to implement Rznomics’ technology as an
innovative treatment that can solve the unmet demand for rare and intractable
diseases.”2023-10-18 -
Rznomics Wins Most Promising Gene Therapy Pipeline Award at International Events for the second consecutive year
Rznomics
Wins Most Promising Gene Therapy Pipeline Award at International Events for the
second consecutive year
ACGTEA
of CGTWA 2023Rznomics
announced on the 18th that it has won the Most Promising Pipeline Award in the
gene therapy category at the ‘Asia Pacific Cell & Gene Therapy Excellence
Awards (ACGTEA)’ at the ‘Cell & Gene Therapy World Asia 2023 (CGTWA 2023)’
held in Singapore. It has been awarded for the second consecutive year since
2022.
CGTWA is an event that shares representative
technologies of cell and gene therapy products of pharmaceutical and biotechnology
companies based in the Asia-Pacific region.
Through this award ceremony, Rznomics introduced
the status of trans-splicing ribozyme-based gene therapy platform technology
and circular RNA technology. The company's circular RNA technology has been
applied for international patents(PCT) and patents in major countries including
the United States, after securing pipelines and domestic original patent
rights.
Last year, Rznomics received clinical approval
for liver cancer treatment from the Korean Ministry of Food and Drug Safety and
the U.S. Food and Drug Administration (FDA). Phase 1 clinical trials of
"RZ-001" are being conducted at five hospitals in Korea. In the
future, phase 2 clinical trials will be conducted simultaneously in Korea and
the United States.
In addition, it received clinical approval
from the Korean Ministry of Food and Drug Safety and FDA in May this year for
glioblastoma treatment. It plans to conduct clinical trials within this year. Rznomics
also plans to apply for clinical trials early next year for autosomal dominant retinitis
pigmentosa, which currently has no treatment at all.
Seong-wook Lee, CEO of Rznomics, said,
"The award for the second consecutive year will be an opportunity to
recognize and promote Rznomics' technology more widely. We will do our best to
implement Rznomics' technology as an innovative treatment that can solve the
unmet demand for rare and incurable diseases."2023-09-18 -
Rznomics Presents Its Own Circular RNA Platform Technology
SEONGNAM, South Korea, Aug. 3, 2023 /PRNewswire/ -- Rznomics developed a novel platform technology termed 'self-circularized RNA structure' that can efficiently and simply generate circular RNA and overcome the limitations of existing technology. Rznomics published an article regarding its development strategy and efficacy in the journal 'Molecular Therapy Nucleic Acids.'Since the success of the COVID-19 vaccine, mRNA technology has been studied with great interest in various fields, such as vaccines and therapeutics. However, conventional linear mRNA is vulnerable to nucleases, and patent-protected modified nucleic acids need to be used for efficient protein expression with the reduction of unwanted immune responses.To overcome the drawbacks of the conventional linear RNAs mentioned above, circular RNA structure technology has recently attracted attention. Circular RNA (circRNA) has a closed structure, so it has great stability against nucleases, and it has been reported that there are no influences on unwanted immune responses and protein expression without using the modified nucleotides. Therefore, if it is commercialized, circRNA vaccines or therapeutics will potentially be more competitive in price and will have the same efficacy equivalent to linear RNA in a smaller amount.Attracting large-scale investments, a couple of major biotech companies in the U.S. lead the circRNA R&D industry and show the expectations from the market. In the case of one of these companies, they use a circRNA manufacturing technology based on group I intron ribozyme, called the Permuted Intron-Exon (PIE) method. The PIE method splits the ribozyme, which was used in the cis-splicing reaction to remove the intron and link the flanking exons, into half and connects each half of ribozyme to both ends of the GOI (Gene of interest) to make the circRNA with specific GOI. Since the immaturity stage of the circRNA field, there are rooms for biotech companies to enter the competition. However, to enter the market with competitiveness, the core & innovative platform technology that overcomes the limitations of existing technologies is essential.Rznomics has developed a new circular RNA manufacturing technology that uses group I intron ribozyme as well. However, the technology is fundamentally different from the PIE method in the circularization mechanism. Rznomics has recently published its own circular RNA technology on line in Molecular Therapy Nucleic Acids (DOI: https://doi.org/10.1016/j.omtn.2023.07.034, Title; Efficient circular RNA engineering by end-to-end self-targeting and splicing reaction using Tetrahymena ribozyme), the official journal of the American Society for Gene and Cell Therapy (ASGCT). Rznomics applied its unique Tetrahymena trans-splicing ribozyme platform technology to effectively convert linear RNA into circular RNA by designing end-to-end self-targeting and splicing (STS) reaction. As shown in the figure, an RNA construct was designed to connect the GOI at the end of the group I intron ribozyme. In this construct, the target site that can be targeted by ribozyme and IGS (Internal Guide Sequence) that guides the targeted trans-ligation by base-pairing with the target site were designed to locate at both ends to enable the circular RNA formation through a self-circularization reaction.The most notable point is that in contrast to the PIE method, Rznomics' circularization technology does not leave traces of non-necessary genetic information in the GOI (Gene of Interest) after the self-circularization reaction. In the case of PIE, the ribozyme needs to connect both ends of the GOI harboring extraneous specific nucleotide sequences to form a circular RNA. Whereas, in the case of STS reaction, the ribozyme leads to self-circularization by designing any target sequence present in GOI to be located at the end of GOI.Also, Rznomics confirmed that circularization efficiency is comparable to the efficiency of the PIE method. In addition, due to the nature of the STS reaction, it is possible to establish various optimization strategies to increase the self-circularization efficiency, unlike the PIE method. It is considered that the self-circularization efficiency decreases as the length of the gene of interest. However, in the case of Rznomics technology, it is possible to screen an optimal target sequence that maximizes the efficacy of self-circularization. Rznomics stated that, through the STS-based reaction, long RNA such as human dystrophin RNA (11,265 nucleotides) was successfully self-circularized.Moreover, a self-circularization reaction occurs simultaneously during in vitro transcription which is the process of synthesizing RNA. Therefore, the purification process can be directly initiated without any additional steps of the process. In the published paper, Rznomics also mentioned a method for purifying the circRNA using the IP-RP HPLC technique. The purified circRNA with unmodified nucleotides enabled more efficient and sustained protein expression without unwanted immune responses, compared with linear RNA with modified nucleotides.This research was conducted with the support of the Ministry of Science & ICT and the National Research Foundation of Korea. The 'Self-circularization RNA structure and Platform technology' is a patent granted in South Korea (10-2442946) and has been applied in the U.S. and major countries.
2023-08-03
- 본사경기도 용인시 수지구 죽전로 152, 단국대학교 소프트웨어ICT관 327호
- TEL. 031-706-8730
- FAX. 031-8018-8734
- E_mailrznomics@rznomics.com
- 기업부설연구소 및 판교지점경기도 성남시 분당구 판교로 253, 판교이노밸리 C동 801호(판교지점), 803호(기업부설연구소)
- TEL. 031-706-8730
Copyright(C) 2019 Rznomics. All Rights Reserved.
