Rznomics

RIBOZYME AND OMICS

고객지원

홍보/투자정보

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[보도자료]알지노믹스, 美미주리대와 유전자치료제 공동연구 협약 체결

알지노믹스는 미국 미주리대와 공동연구를 위한 업무협약(MOU)을 체결했다고 14일 밝혔다.협약에 따라 양측은 리보핵산(RNA) 편집, 인간 유전자 도입 동물모델 확립, 전임상 시험 및 임상 연구 등 유전자치료제 개발을 위한 연구 분야 전반에 대해 협력한다. 인적 및 물적 자원의 상호 교류, 기술 개발 등의 추진을 예상하고 있다.미주리대는 1839년에 설립돼 농학 수의학 의학 약학 등 생명과학 분야에서 두각을 나타내고 있는 주립대란 설명이다. 최근 유전자 치료제 연구에 많은 관심을 가지고 있다고 했다.이성욱 알지노믹스 대표는 "알지노믹스가 보유한 RNA 편집 플랫폼 기술과 미주리대의 연구 인프라를 접목해,RNA 기반 유전자 치료제 연구 분야에서 경쟁력을 강화할 수 있을 것"이라며"앞으로도 국내외 연구기관들과의 파트너십을 확대해 글로벌 유전자 치료제 기업으로 도약할 것"이라고 말했다.

2022-11-14
알지노믹스(Rznomics) 자사 플랫폼 기술과 프로그램 개발 진행현황 공개

알지노믹스가 자사 플랫폼 기술과 프로그램 개발 진행현황을 공개했다 홍성우 알지노믹스개발본부 부사장은 지난 1일 여의도 전경련회관에서 열린 '2022 LOG IN IR EXPO 100'에서발표를 진행했다알지노믹스는 RNA 치환효소(RNA replacement enzyme)플랫폼으로 RNA 타깃 약물을 개발하고 있다 알지노믹스의 플랫폼은 트랜스 스플라이싱(trans-splicing)을 일으키는리보자임(ribozyme)과 치료효능 유도물질을 암호화한 치료유전자(therapeutic transgene)를이용한다. 리보자임과 치료유전자가 연결된 DNA를 아데노바이러스(adenovirus)등의 벡터를 이용해세포내로 전달하며 세포에서 전사된 약물 RNA가 표적 RNA를 타깃하는 방식이다.리보자임으로 표적 RNA에 특이적으로 결합한 뒤 트랜스 스플라이싱으로 표적 RNA의 일부분을치료자유전자 RNA로 대체시키는 방식이다. 이를 통해 질병을 일으키는 표적 RNA의 기능은상실되며 치료유전자가 암호화한 단백질이 발현된다.트랜스 스플라이싱에 기반한 글로벌 약물개발 상황은 아직 초기단계이며 지난달 트랜스 스플라이싱을이용해 RNA타깃 치료제를 개발하는 아시디안 테라퓨틱스(Ascidian Therapeutics)가5000만달러의 시리즈A 유치와함께 출범소식을 알렸다.

2022-11-04
[논문게시]Pros and Cons of In Vitro Methods for Circular RNA Preparation

알지노믹스의 이경현박사, 김성철연구원, 이성욱박사의 'Pros and Cons of In Vitro Methods for Circular RNA Preparation' 논문이International Journal of Molecular Sciences 에 게시되었습니다.

2022-10-31
<대표이사 인터뷰> RNA치환으로 1석2조 효과 노린다, 알지노믹스

대표이사 인터뷰가 <한경바이오인사이트> 매거진 2022년10월호에 게시되었습니다.상세 내용은 상단의 기사 원문을 참고 부탁 드립니다.

2022-10-26
[보도자료]RZNOMICS Inc. received FDA approval to initiate clinical development of trans-splicing ribozyme-based RNA editing technology in liver cancer patients

SEONGNAM, South Korea, Oct. 25, 2022 /PRNewswire/ -- Rznomics Inc., a South Korea based biopharmaceutical company specialized in the development of RNA-based gene therapeutics, recently received Phase 1/2a IND approval from the U.S FDA in October 10th for its hepatocellular carcinoma (HCC) treatment called RZ-001 and thus has achieved an important milestone for the company and the RNA editing field. Being the first U.S. FDA-approved ribozyme-based RNA reprogramming approach to be evaluated in patients, RZ-001, a gene therapy approach utilizing the company's proprietary trans-splicing ribozyme-based RNA reprogramming and editing technology, is a replication-incompetent adenoviral vector that expresses an hTERT targeting ribozyme with multiple additional MoA to treat HCC patients. Rznomics also received IND approval of RZ-001 from the South Korean Ministry of Food and Drug Safety this June and already initiated a phase 1/2a clinical trial in Korea. Therefore, U.S. FDA approval allows Rznomics to start an international clinical study in HCC patients treating them with RZ-001 and therapeutic RNA editing. The trans-splicing ribozyme is derived from the self-splicing Tetrahymena group I intron, which both recognizes and reprograms the target RNA into the therapeutic transcript of interest. Ribozyme-based RNA editing technology developed by Rznomics has unique features, differentiating it from other nucleic acid-based editing approaches, as follows: (1) A single RNA molecule is capable of both suppressing target RNA expression and simultaneously expressing a therapeutic RNA. Thus, no potentially antigenic proteins or cofactors are required. (2) Safety can be improved by selectively inducing therapeutic RNA expression only in cells/tissues where the target gene is expressed. (3) Therapeutic gene expression can be regulated proportionally to endogenous cellular target RNA levels. (4) Editing occurs at the RNA level, not the genomic level, thus eliminating concerns about genomic toxicity and eternal genome changes. (5) Indications with multiple mutation sites scattered throughout a target RNA can be edited with a single RNA designed to react upstream of all mutations and by replacing and editing large stretches of RNA. (6) Additional safety can be conferred by building control mechanisms into the ribozyme itself, without the need to modulate intrinsic cellular mechanisms or external proteins. More specifically, RZ-001 engenders effective anti-HCC activity by suppressing hTERT expression selectively in cancer cells, which over-express hTERT, and simultaneously inducing a cytotoxic effect by trans ligating an HSVtk-encoding sequence into the reprogrammed hTERT mRNA. Moreover, the result of such editing efficiently induces immune cell infiltrations into HCC tumors in preclinical animal models. (http://www.rznomics.com/pipeline/RZ-001.php). The Phase 1/2a clinical trial will be a dose escalation/expansion study to assess the safety and tolerability of RZ-001 and to determine the most effective dose with the least toxicities of RZ-001 in HCC patients with no extrahepatic metastasis. "The translation of the first trans-splicing-based RNA editing approach into an FDA-approved phase 1/2a clinical trial is an exciting achievement and a critical milestone for the RNA editing field. I am very excited about Rznomics' preclinical progress on ribozyme design and gene delivery optimization. The advances have allowed them to create and now translate a promising therapy, RZ-001, into the clinic. I and the entire editing field are eager to learn if RZ-001 and mRNA reprogramming is safe and able to combat hepatocellular carcinoma in patients. Rznomics is clearly a leader at bringing novel editing strategies to cancer patients that desperately need innovative, breakthrough therapies," said Dr. Bruce Sullenger, Joseph, and Dorothy Beard Professor of Surgery at Duke University. Dr. Sullenger is a scientific advisory board member of Rznomics and the initial pioneer developing approaches to therapeutically edit RNA and DNA using RNA guided endonucleases (RGENs) such as the group trans-splicing I ribozyme.. "It's a monumental achievement of Rznomics that RZ-001, the first trans-splicing ribozyme therapy at the front of our therapeutic pipeline, has successfully received the IND approval in both Korea and the United States. I am really grateful that RZ-001 earned the opportunity to potentially fulfill the unmet needs of HCC patients. Through the advanced development phase, I hope Rznomics can provide more new therapeutic options to patients suffering from intractable diseases. Rznomics will further expand our pipeline by targeting indications with highly unmet medical needs for which the unique characteristics of our platform technology may be the most competitively applied." said Dr. Seong-Wook Lee, CEO and founder of Rznomics.In addition to the HCC, Rznomics is expanding the indication of RZ-001 to glioblastoma multiforme and planning to submit the IND this year. Also under development are ribozyme-based RNA editing treatments for Alzheimer's disease (RZ-003) and inherited retinal dystrophies, called Retinitis pigmentosa (RZ-004).

2022-10-25
[보도자료]알지노믹스, 간암 치료제 美FDA 임상시험계획 승인

알지노믹스가 미국 식품의약국(FDA)으로부터 ‘RZ-001’의 1·2상 임상시험계획(IND) 승인을 받았다고 11일 밝혔다.지난 6월에 식품의약품안전처로부터 임상시험계획 승인을 받아 진행중인 국내 임상시험의 연장선상으로, 원래 계획했던 다국가 임상시험 목적의 승인이다.알지노믹스는 리보핵산 치환효소(RNA trans-splicing ribozyme)를 기반으로 표적 리보핵산(RNA)을 제거하면서 동시에 원하는 유전자를 발현시키는 기술을 보유하고 있다.RZ-001은 아데노바이러스(adenovirus) 벡터로 리보핵산 치환효소를 전달해 암세포 특이적으로 발현하는 텔로머라아제(hTERT) RNA를 표적한다.동시에 항암작용을 유도하는 유전자를 발현시키는 기전이다.알지노믹스는 다른 암종으로도 적응증 확대를 추진하고 있으며, 특히 연내 악성교모세포종에 대한 임상시험을 계획하고 있다. 또 항암제 파이프라인 뿐만 아니라 알츠하이머 치료제, 유전성 망막생소변성증, 레트증후군 등 플랫폼기술을 활용한 다양한 분야의 치료제를 개발하고 있다.이성욱 알지노믹스 대표는 “개발중인 파이프라인들이 순차적으로 국내외 임상에 착수될 예정이며 환자들에게 새로운 치료기회를 제공할 수 있도록 최선을 다하겠다”고 말했다.

2022-10-11
[보도자료] 알지노믹스, 국제 행사서 유전자치료제 유망 후보물질상 수상

알지노믹스는 싱가포르에서 개최된 'Cell & Gene Therapy World Asia 2022'(CGTWA 2022)의 'Asia-Pacific Cell & Gene Therapy Excellence Awards'(ACGTEA) 시상식에서 유전자 치료제 부문 가장 유망한 파이프라인상을 수상했다고 16일 밝혔다.CGTWA는 세포·유전자 치료제 대표 기술들을 공유하는 행사다. 수상 이후 이어진 콘퍼런스에서는 '차세대 세포 유전자치료제' 세션 발표를 진행했다.발표에서 알지노믹스는 치환효소(Trans-splicing ribozyme) 기반의 유전자 치료제 플랫폼 기술 및 최근 임상시험을 진행 중인 간암 치료제를 포함한 후보물질(파이프라인)들의 현황을 설명했다. 혁신신약(First-in-class) 플랫폼 기술 보유 기업으로서 기술 혁신성과 비전을 제시했다고 전했다.알지노믹스는 내년 1분기 교모세포종 환자를 대상으로 하는 임상을 미국과 한국에 신청할 예정이다. 내년 하반기에는 유전성 망막색소변성증(Retinitis pigmentosa)에 대한 미국 임상도 계획하고 있다.이성욱 알지노믹스 대표는 "이번 수상이 신약개발 열망을 가진 임직원들과 알지노믹스를 성원해 주신 분들께 많은 힘이 될 것"이라며 "더욱 좋은 성과를 달성하기 위해 최선을 다하겠다"고 말했다.

2022-09-16
[보도자료]알지노믹스, FDA에 임상시험 IND 신청

알지노믹스(Rznomics)는 지난 9일 미국 식품의약국(FDA)에 간세포암치료제 “RZ-001”의 1/2a상 임상시험 IND(임상시험계획)를 신청했다고 밝혔다. 지난 6월에 식품의약품안전처로부터 임상시험계획 승인을 받아 진행중인 국내 임상시험연장선상이며, 원래 계획했던 다국가 임상시험을 위한 제출이라는 설명이다.알지노믹스는 리보핵산 치환효소(RNA trans-splicing ribozyme)를 기반으로 표적 RNA를 제거하면서 동시에 원하는 유전자를 발현시키는 기술을 개발하고 있다. RZ-001은 아데노바이러스(adenovirus) 벡터로 리보핵산 치환효소를 전달함으로써 암세포 특이적으로 발현하는 텔로머라아제(hTERT) RNA를 표적, 항암작용을 유도하는 유전자를 발현시키는 기전이다.알지노믹스는 다른 암종으로도 적응증 확대를 추진하고 있으며, 특히 연내 악성교모세포종에 대한 임상시험을 계획하고 있다. 또한 항암제 파이프라인 뿐만 아니라 알츠하이머 치료제, 유전성 망막생소변성증, 레트증후군 등 플랫폼기술을 활용한 다양한 분야의 치료제를 개발하고 있다. 유전성 망막색소변성증은 지난 5월 국내 특허청 등록이 완료 되었으며, 이어서 8월에는 미국 특허청으로부터 등록결정 통지를 받아 기술의 진보성을 입증 받았다고 회사측은 부연했다.이성욱 알지노믹스 대표는 “개발중인 파이프라인들이 순차적으로 국내외 임상에 착수될 예정이며 환자들에게 새로운 치료기회를 제공할 수 있도록 최선을 다하겠다”고 말했다.

2022-09-13
[보도자료]CEVEC’s license partner RZNOMICS Inc. receives approval to initiate clinical development of CAP® Ad Technology-produced gene therapy vector in liver cancer patients

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.

2022-07-14

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