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[Press released] Cha Vaccine Institute and Rznomics jointly develop next-generation RNA vaccines
Rznomics, Non-linear RNA Structure
Platform company, apply the Lipo-pam™ delivery from Cha Vaccine Institute Rznomics announced on the 9th that
it has signed an "agreement for joint development of next-generation
vaccines" based on the next-generation RNA platform with Cha Vaccine
Institute, an affiliate of Cha Biotech. Based on this agreement, Rznomics and
Cha Vaccine Institute will jointly research and develop next-generation RNA
vaccines, and will also cooperate for the exchange of physical and human
resources necessary for the development process.. The RNA vaccine developed by the two
companies this time is the next-generation technology of the mRNA vaccine
currently used in the COVID-19 vaccine. The company explained that the
next-generation RNA vaccine is based on a non-linear RNA structure and has the
advantage of long-lasting efficacy due to its high stability compared to the
linear mRNA vaccine. The Cha Vaccine Institute first
selects a target disease to be applied to the vaccine and designs an antigen
accordingly. Rznomics is in charge of producing, optimizing, manufacturing, and
processing RNA vaccines that express antigens designed by Cha Vaccine Institute
based on its own nonlinear RNA structure platform. The Cha Vaccine Institute
uses "Lipo-pam™" as a delivery material for RNA vaccines to
non-clinically study vaccines and derive vaccine candidate substances. The Cha Vaccine Institute has
developed its own immune enhancers L-pampo™ and Lipo-pam™, which are necessary
for the manufacture of recombinant protein vaccines. Based on this, clinical
development of preventive and therapeutic vaccines for infectious diseases such
as hepatitis B and shingles is underway, and pipelines are being expanded to
anti-cancer vaccines and immuno-cancer drugs. Rznomics is an RNA-based new drug
developer. The core technology is developing therapies for anticancer drugs,
degenerative diseases, and genetic diseases based on the mechanism of editing
target RNA that causes diseases with trans-Ribozyme and replacing the cut part
with other treatment RNA at the same time. It also has its own nonlinear RNA
structure platform technology. Jung-sun, Yeom, CEO of Cha Vaccine
Institute, said, "As the two companies with their own unique technologies
cooperate, the development of next-generation vaccine platforms and new
pipelines using them will be smoother. Through this MOU, Cha Vaccine Institute
will also expand its business area to new areas such as RNA vaccines. "We expect this collaboration
to accelerate the development of next-generation vaccines based on our new RNA
structure technology," said Seong-wook, Lee, CEO of Rznomics. "We
will prove the versatility, usability, and excellence of Rznomics’ unique RNA
platform technology."2022-02-09 -
[Press released] K-Bio jumps into the global 'RNA new drug war'
Ribonucleic
acid (RNA) treatment, which was an unexplored field, is receiving a lot of
attention from the global pharmaceutical and bio industry. RNA therapy is a
method that blocks the expression of a disease-causing gene. Until recently,
the task of developing an RNA treatment was how to deliver it into cells
because of its large size. However, with the commercialization of the messenger
ribonucleic acid (mRNA) COVID-19 vaccine as an opportunity, the problem has
been resolved and development is accelerating.Rznomics
is developing a treatment for liver cancer. It is a treatment that cuts off
target RNA that appears in cancer cells and implants RNA that induces cancer
cell death in its place. At end of last year, the company applied for approval
of phase 1 and 2a clinical trials with the Ministry of Food and Drug Safety.Rznomics
is jointly developing a treatment for intractable diseases using a
next-generation RNA platform with GC Biopharma. Celltrion is also developing an
mRNA-based anticancer drug with TriLink, an American bio company.The
movement to develop RNA therapeutics is more active overseas than in Korea.
News of cooperation, such as mergers and acquisitions (M&A) and technology
transfer for RNA therapeutics market, is following one after another. The
announcement of the acquisition of Dicerna Pharmaceuticals by Danish
pharmaceutical company Novo Nordisk is an example.Novo
Nordisk, the world's 16th largest pharmaceutical company, announced in November
last year that it would acquire Dicerna, a leading RNA company, for $3.3 billion
USD. There are also constant observations that Novartis will acquire Alnylam,
which was the first to receive FDA approval for an RNA treatment.At the
recently held JP Morgan Conference, the world's largest bio-investment event,
the "RNA trend" was clearly visible. Eli Lilly announced its roadmap
to fill 20% of the preclinical pipeline with nucleic acid therapy.Pfizer
and BioNtech, which made a fortune with the COVID-19 mRNA vaccine, started
developing a shingles virus vaccine. Moderna has started administering to
patients in a phase 1 clinical trial of the Epstein-Barr Virus (EBV) vaccine,
which is known to cause cancer. EBV is known to cause lymphoma and gastric
cancer by B cells.2022-01-21 -
[CEO Interview] Attracted billions of investment by developing a gene-based target treatment system for incurable diseases
Meet
Lee Seong-wook, a professor of Department of Biotechnology and Bioengineering
and CEO of Rznomics, who stands out as the CEO of a promising biopharmaceutical
company from an aspiring researcher
“When
I went to study in the United States, my advisor had already finished research
on the viral replication process and started researching gene therapy using viruses
as vector. Participating in these studies, he decided to pursue research in the
field of intractable treatment. In addition, my advisor taught me to always
think about how to develop value in the research in the laboratory, and he
showed the realization of that value through starting a business.
By
maximizing my research in incurable diseases such as cancer can be treated with
minimal damage to normal tissues, and I believed it would be of sufficient
value. And in order to realize the value, from the beginning of R&D I had
the mindset that 'start-up is the way to go'. Maybe it was because I got
insight from the professors who started a business, and also I already have a
student who started a business. After he finished master’s degree in molecular
biology, he went to medical school to become a research doctor, but after
becoming a medical professor, one day, he came to me with a business card stating
CEO.”Professor
Seong-wook Lee (Department of Biotechnology and Bioengineering, Graduate
School) from Dankook University obtained a bachelor’s degree in microbiology
from Seoul National University and a doctorate in molecular biology from
Cornell University, USA which he also worked as a postdoctoral researcher. He who
is also the former president of the Korean Society for Gene and Cell Therapy,
compiled the results of his 20 years of research and founded Rznomics.Rznomics
is developing an RNA-based gene therapy using the trans-splicing ribozyme
platform technology. This technology is a platform technology that recognizes
the target site and removes the problematic RNA sequence from this site, and at
the same time connects the RNA that delivers or injects a specific substance to
the target site for the purpose of improving the function of the target. As the
excellence of the technology is spreading widely, Rznomics is attracting large
amounts of investment every year, and plans to list it on the stock market in
the future.
On January 4th, I met Professor
Seong-wook Lee and was able to hear detailed story about the prospects and
goals of Rznomics and the technology.
Q. Congratulations on
becoming a CEO who is attracting attention not only in academia but also in the
biopharmaceutical industry.
All I've done is try
to make sure that valuable technology doe not go dead. What kind of technology
is that? It is a technology that was developed through sweat of researchers
with the support of tax money collected by the government to help cure someone.
Although it has received attention within Korea, Rznomics' goal is to provide
licenses to global biopharmaceutical companies. If you have commercialized the
technology that you have researched for decades, I think you should aim for it
to growth on a global scale. There is still much work to be done to achieve
that goal.
Q. Could you please
explain the trans-splicing ribozyme platform technology that you have been
researching for many years?The key is to inject
a ribozyme into a patient in the form of a DNA vector that removes bad RNA that
causes incurable diseases and substitute it with a good RNA. Ribonucleic acids
that function as enzymes are called ribozymes. Although there are various types
of ribozymes, the ribozyme we have focused on researching and developing
conceptually has the ability to selectively induce the activity of therapeutic
genes in cells expressing therapeutic RNA by editing the target RNA to be
treated with therapeutic RNA. The technology we've been researching is to take
advantage of these functions of ribozymes, and our lab has for 20 years been
focused on optimizing these functions beyond concept to actually have
therapeutic effects. Previous studies have had marginal efficacy or safety
concerns.
I have achieved the
result of developing a ribozyme with therapeutic function in vivo for the first
time by making the therapeutic gene RNA to be expressed only in target diseased
cells (specificity), increasing therapeutic efficacy, and at the same time
improving the safety of the DNA vector.
Q. Why did you choose liver cancer as a target disease for treatment development?Cancer is still one of the most incurable diseases. In the early 2000s, when there was very little public interest in gene therapy, hepato-cancer specialists and researchers were the people who proposed research after hearing about my research field ‘trans-splicing enzyme platform’. As a result of working together with these people and immersing ourselves in the research, the liver cancer-based treatment material optimization was completed. Since it is a target molecular substance for treatment, not a drug composed of chemicals, which it is a platform technology, it has sufficient potential to be applied to all cancers.Currently, research is being conducted to expand indications to glioblastoma of malignant brain tumors and hereditary retinal pigment degeneration. We are also investigating the possibility of using it as a treatment for neurological/cognitive diseases such as Alzheimer's and Rett's syndrome. In particular, the innovative new drug lead material for Rett Syndrome, a hereditary neurodevelopmental disorder that has no underlying treatment, was developed by Rznomics and was recently selected as a governmental new drug development project.
Q. Based on this
technology, you founded Rznomics, as a subsidiary of Dankook University
Industry-University Cooperation Foundation.
Rznomics was founded
in August 2017 as a research company that develops gene therapy products using
RNA substitution enzymes. We are researching with the goal of developing
bio-drugs for cancer/incurable diseases through gene therapy development. In
particular, we are researching a treatment for primary liver cancer, and the
results of the research that specifically kills cancer cells based on the
'trans-conjugated ribozyme' platform for RNA replacement enzymes were published
in the official journal (Molecualr Therapy Nucleic Acids) of the American
Society of Gene and Cell Therapy (ASGCT). We have applied for clinical approval
to the Korean Ministry of Food and Drug Safety at the end of November last year
for pipeline 'RZ-001' to which this technology has been applied targeting liver
cancer treatment. As it is a field that I have researched for 20 years and
achieved a high level of optimization, I decided that there was a certain
possibility of commercialization, and I was able to start a business without hesitation
because gene therapy is a field where investment is made actively.
Q. It is a field
that is meaningful enough for pure research, but I am curious as to why you
decided to start a business. When I was doing my PhD in Molecular
Biology at Cornell University, my advisor was passionate about starting a
business. 'Developing skills and writing a thesis is important, but you
shouldn't be content with just leaving your name on a few pages of paper. “It
would be even better if we could study the application method that would
actually affect society to the end and start a company based on it.” It was a
time when the biopharmaceutical industry was revitalized in the United States,
and I was aware from an early age that my research could be commercialized
based on the latest research results such as the concept of ribozyme. Eventually,
I started a company when the business plan was materialized for my research on
gene therapy to be injected in the form of a vector based on RNA.
Gene therapy was introduced as a
concept in the 1990s, but it was said that it was impossible to commercialize
it. In the 2010s, developed countries’ authorities began to approve gene
therapy. Since I started my business in 2017, so I guess I tend to adapt well
to the global trend.
Q. Did you have any
difficulties in the early days of going out of the lab and running the company
yourself?
Since it was a company that started
with two students and a school laboratory, of course, in the beginning, we did
not have a lot of money. But we didn't worry too much because we had certainty
in commercializing our research. In 2017, we was selected for a private-led start-up
support project (TIPS) to raise funds for establishment, and with some help from
venture capitals, we were able to receive funds as well as conducting research
projects from government agencies. The research results have been attracting
attention every year, and so far we have been able to attract a total of 23.7
billion KRW of investment. We had difficulties in management, administration,
and legal affairs, but thanks to Dankook University Industry-University
Cooperation Foundation we were provided a lot of help from early stage of the
company in various areas. I would like to extend my gratitude for the support.
Q. What does it
mean for a company started with technology developed in the laboratory to
receive tens of billions of investment?
When a science and engineering
researcher actually start a business, establishes a company, and receives
investment, researchers often joke with each other, saying, “I have crossed a
river of no return.” It is because there is no guarantee that you will be
successful just because you receive an investment, and you cannot be pushed
back just because there is no guarantee of success. However, I have had
experience of starting a small business before, and from this experience, I
personally decided that in order to succeed as a biopharmaceutical company, I
had to build a unique platform technology and a pipeline that can prove the
value of these technologies. Rznomics was founded by trusting our technology as
the preparation process has matured enough. A researcher starts a business
because he thinks his research is great, but an investor makes an objective
judgment and evaluation, so receiving an investment is meaningful. Investors
invest in us because they believe in our company, and the amount of investment
reflects our evaluation.
Q. Established as a research institute on campus, the
company has grown so rapidly that it has received tens of billions of
investment over the past four years. You said that you express your special
thanks to the Dankook Industry-University Cooperation Foundation, and I am
curious as to why.
In the early days of the founding, all members of the company were
researchers. In that situation, you need to distinguish between what you can do
on your own and what you must get help from. One of them is public relations
and external cooperation such as PR, IR, and website production. The
Industry-University Cooperation Foundation, together with the school
headquarters, provided support and provided PR expenses. In addition to the
BRIDGE+ commercialization development cost, Dankook University's technology
holding company also made a direct equity investment in Rznomics. I express my
gratitude once again for sharing our achievements with professors and other
school members at important events.
Q. From the perspective of managing a promising
biopharmaceutical company, would you give advice to researchers who are
preparing for technology commercialization based on university research?I too am still in a starting position, so providing 'advice' would not
be the correct wording. If I dare say, I would like to point out that it is
necessary to diagnose the technology soberly and accurately, and to respect the
area of expertise as much as possible. I recommend that you first get an
objective evaluation of your skills. All the professors are the highest
authority in their field, so I think that the technology developed by them is
also the best technology. However, unlike academic research, commercialization
must directly generate profits. To do this, you need to take a good look at the
industry, see who your competitors are in Korea and in the world, and think
about where your company will stand. If I'm the first, I need to review why
others haven't jumped into it, make sure it's not in an area that's unrelated
to monetization, and there's no catastrophic risk. If I am the best, I need to
diagnose where the technology can be applied and what the revenue will be if it
is applied.
In addition, although a professor may have authority in research or
technology development, but he at the same time can be unfamiliar with
technology research, development for commercialization of actual technology,
and other fields. As the pace of technological development is fast, the first
or best technology is good, but it must be made into a company that has built
an efficient system that can successfully commercialize it with the agility to
present the optimal technology for the purpose at the right time. To do this,
you need to open your mind to recruiting experts in each field or to
collaborate with them. I want you to keep in mind that investment includes not
only R&D expenses, but also personnel expenses and external activity
expenses.2022-01-21 -
[Press released] Rznomics, CJ and Batavia signed agreement for the production and development of gene therapy products
Rznomics,
Inc. announced on January 19th that it had signed a three-party business agreement
(MOU) with CJ and Batavia Bioscience for cooperation in the production and
development of gene therapy products based on virus delivery systems.Three
companies plan to cooperate in the exchange of material and human resources to
conduct research and development on the production process of gene therapy
products. The companies decided to explore collaboration for mass production in
the future commercialization stage, Batavia
is a contract development and manufacturing (CDMO) company based in the
Netherlands. CJ acquired Batavia last year to speed up its business in 'health
(wellness)' as one of the four future growth engines. They possess process
development and manufacturing technology for vaccines and gene therapy
products.Rznomics
is a gene therapy development company established in 2017. Based on ribonucleic
acid (RNA) substitution enzyme (Trans-Ribozyme), the company is developing gene
therapy for anticancer and intractable diseases. The company also plans to
transfer its technology to multinational pharmaceutical companies in the
future.“Through this
collaboration, we will be able to accelerate the development of next-generation
gene therapy products based on our unique platform technology,” said Seong-wook
Lee, CEO of Rznomics.A CJ
official said, "Starting with the agreement with Rznomics, we will help
domestic bio companies with capabilities to advance into international markets.Rznomics
is pursuing the IPO process with the goal of second half of next year. In
Series A and B, it maintained investments of KRW 12 billion and KRW 10.5
billion, respectively.The
government project support totaled KRW 3.3 billion. The series C investment is
expected to be completed in the first quarter of this year.2022-01-19 -
[Medigate] K-Bio in the process of developing global new drug, and noteworthy companies in 2021
According
to a fact-finding survey conducted by the Korea Biotechnology Association, the
domestic bio-industry sales have grown at an average annual rate of 8.8% for
the past 10 years, which is higher than other industries such as the
manufacturing industry at 1.7% and pharmaceuticals at 5.6%. In particular,
despite the overall industrial activity stagnant due to the COVID-19 pandemic
that has swept the world since 2020, the bio industry has achieved high exports
thanks to increased biopharmaceutical consignment production, increased
biosimilar market share in the U.S. and Europe, and increased exports of
COVID-19 diagnostic kits. At the
same time, many domestic bio companies are focusing on R&D to create global
new drugs. As of 2019, domestic bio industry R&D expenditure was about 1.8
trillion won, and it is expected to exceed the 3 trillion won level by 2024.Medigate News is
conducting an interview with the CEO of a promising domestic bio company in the
early stage to highlight the growing domestic bio industry and help researchers
with outstanding technology to revitalize their startups. As we wrap up the
year of 2021, 20 companies we met this year summarized what kind of technology
and competitiveness they are moving toward the global market.Rznomics:
Development of RNA substitution enzyme platform technology with specificity and
efficacyRNA
replacement enzyme (trans-splicing ribozyme) has the function of artificially
combining separate RNAs by cutting itself off and linking it with the RNA next
to it. It can remove bad RNA and replace it with good RNA. However, RNA itself,
in nature, has low specificity and efficacy, so there is a limit to using this
function as a therapeutic agent.After
conducting an optimization study, Professor Seong-wook Lee of Dankook
University's Department of Biotechnology and Bioengineering developed a
technology that captures both specificity and efficacy by allowing the gene RNA
with therapeutic effect to be expressed only in diseased cells, and started Rznomics
in 2017. There are several groups that research RNA replacement enzymes, but Rznomics
is the first company to research and develop therapeutics in the form of a
company.This
technology is unique in that there is no risk of normal cells by acting only
where bad RNA is expressed. First, the company focuses on autosomal dominant
genetic diseases in which the wrong gene is dominant. Currently, the company
have pipelines that focuses on disease such as liver cancer, glioblastoma,
Alzheimer's, and retinitis pigmentosa. In addition, the company plans to focus
on finding indications that can prove the value of the technology. Following
the 12 billion KRW Series A investment in 2019, it also followed with a bridge
investment worth 10.5 billion KRW this year. In the first half of 2023, when
two clinical trials are expected to be in progress, the company is expected to
request technology evaluation and IPO will follow.2021-12-30 -
[Press released] Rznomics selected for ‘National new drug development project for rare genetic diseases’
Rznomics announced on the 16th
that a candidate substance as an innovative new drug for Rett Syndrome under
development was selected for national drug development project by the National
New Drug Development Foundation.With this selection, Rznomics will
receive a national research grant for 24 months for research on lead material
derivation and verification.Rett syndrome is a hereditary
neurodevelopmental disorder with an incidence of 1 in 10,000 to 15,000. To
date, there is no approved treatment.For this reason, patients have
been treated with physical therapy or nutritional support.More than 90% of Rett syndrome
is known to be caused by mutations in the 'MeCP2' gene.Disease occurs when MeCP2 does
not function normally. It develops relatively normally from 6 to 18 months of
age, and then shows a decrease in brain development, loss of acquired cognitive
and motor skills, and loss of language function.The treatment under development
by Rznomics is the world's first gene treatment with single dose. The 'RNA
replacement enzyme', a platform technology of Rznomics that removes mutated
genes and replaces them with normal genes at the same time, is applied. By
replacing the mutant MeCP2 gene with a normal gene, it is expected that a
fundamental treatment of the disease will be possible.Seong-wook Lee, CEO of Rznomics,
said, "It is the result of a positive evaluation of the potential of
Rznomics' gene therapy by the National New Drug Development Foundation”,.
Director Han Seung-ryeol, who was selected as the research director, said, "since
Si-yoon Kim, a professor of veterinary medicine at Konkuk University
participate as a co-researcher, synergy is expected through industry-university
collaboration."2021-12-16 -
[thebell] Rznomics Inc. prepares for IPO... Samsung Securities as lead underwriter
Rznomics Inc., a new drug development company, will select Samsung
Securities as the leading underwriter for listing for an IPO. The company plans to list on the KOSDAQ after major pipelines
enter clinical trials in Korea and the US.Rznomics is an RNA-based gene therapy company established in August 2017
by Professor Lee Seong-wook of Dankook University. Their main platform is a
therapeutic technology with a mechanism of editing a disease-causing target RNA
with an RNA replacement enzyme (trans-splicing ribozyme) and generating a
therapeutic RNA in its place. It is characterized by being able to access
incurable diseases that were previously inaccessible by differentiating its mechanism
of action from existing treatments.The company’s RNA-substituting enzyme-based pipeline is a treatment for
liver cancer (RZ-001), glioblastoma (RZ-002), Alzheimer's disease (RZ-003), and
hereditary retinitis pigmentosa (RZ-004). RZ-001 is currently in the final
stages of GMP production and GLP toxicity studies, and plans to apply for IND
in Korea and the United States in the fourth quarter of 2021 to enter clinical
trials from next year.The company said, "We held a pre-IND meeting with the US Food and
Drug Administration (FDA) and received very positive responses about the
R&D results and future plans."RZ-004 has recently selected a candidate material and is expected to start
production soon. The goal is to apply for an IND to the US FDA and enter
clinical trials in the first quarter of 2023.Rznomics is trying to pioneer new RNA technology, including expanded
indications. To this end, a new platform team was recently established in the
company-affiliated research institute, and an external RNA expert was recruited
and a new RNA platform development based on RNA substitution technology began.
It is a field that can be applied to various indications, such as RNA vaccines
and expression constructs for next-generation gene therapy products.It is also continuing its business partnerships with domestic and foreign
institutions. Last year, a license agreement was signed with german company CEVEC,
for the use of cell line technology, and a CDMO contract for the production of
RZ-001 was also signed with US based company, Vigene. They have signed a
license for hereditary retinitis pigmentosa disease animal model with Baylor
University in the United States, and signed a contract with the University of
Louisville to collaborate on RZ-004's efficacy experiments in large animals.
Recently, it joined hands with GC
Biopharma and MOGAM Institute for Biomedical Research
to jointly develop a treatment for intractable diseases based on a
next-generation RNA platform.Major financial investors include Synergy IB Investment, Quad Asset
Management, Korea Development Bank, SBI Investment, LSK Investment, Partners
Investment, and eBest Investment & Securities.
In October 2019, the company received investment of 12 billion KRW in the
Series A round, and in March, it attracted a Series B investment of 10.5
billion KRW. About 2.5 billion KRW was received through the selection of government
support projects.2021-07-27 -
GCBiopharma·Mogam Bio Research, Rznomics cooperate to develop new RNA drugs
GC Biopharma
(CEO Eun-Chul Huh) and MOGAM Institute for Biomedical Research (CEO Jeong
Jae-wook, hereafter referred to as MOGAM) announced on the 11th of
June that they had signed an agreement with Rznomics Inc. (CEO Lee Seong-wook)
to jointly develop a treatment for intractable diseases based on a
next-generation ribonucleic acid (RNA) platform.This
agreement is about cooperation not only for joint research for the development
of therapeutic agents, but also for the exchange of material and human resources
necessary for the development process.In this
process, Rznomics will be in-charge of developing next-generation technology
that can be applied to rare intractable diseases based on its own RNA platform.
Rznomics is a new drug development company based on ‘trans-ribozyme’.
Which its main
business is the development of gene therapy for cancer and intractable
diseases.MOGAM has
the research base capability on disease mechanisms and models (in vitro/in
vivo), and will cooperate organically with Rznomics to derive drug candidates
based on the technology and research capabilities acquired through mRNA and drug
delivery research. In addition, GC Biopharma will provide resources and
know-how necessary for clinical research and commercialization.“Through this
collaboration, we will develop next-generation RNA technology based on our
unique platform technology and spur the development of innovative
therapeutics,” said Seong-wook Lee, CEO of Rznomics.Jung
Jae-wook, head of the MOGAM institute, said, “We will work closely with collaborators
to develop a treatment based on the excellent basic research capabilities of
the MOGAM Institute.”Heo
Eun-cheol, CEO of GC biopharma, said, “We will continue to seek various
opportunities for cooperation to meet unmet needs in the treatment of
intractable diseases.”2021-06-11 -
[thebell] 알지노믹스, 'RNA치환효소'로 희귀질환 치료 도전
임정요 기자, 공개 2021-05-24 08:19:48 이성욱 대표, 20년 R&D 매진 "내년 초 시리즈 C 준비" 신약개발 업체 알지노믹스를 규정하기는 쉽지 않다. 아직까지 생소한 'RNA치환효소(trans-splicing riboz yme)'라는 기술 때문이다. 질병을 일이키는 RNA를 잘라내 없애고 그 자리에 치료제 RNA를 대신 연결시키는 메커니즘이다. 이를 통해 간암, 알츠하이머, 망막색소변성증 등의 치료제를 연구하고 있다. 알지노믹스 사명은 언뜻 유전자치료제 회사들이 사용하는 '지노믹스(Genomics)'로 착각할 수 있다. 회사 영문명은 사실 "Rznomics"로, 리보자임학이라는 의미의 'Ribozyme and omics'에서 착안했다. 리보자임 효소역할을 하는 RNA인데 특히 치환 기능을 하도록 디자인됐다. 알지노믹스 대표는 20년 리보자임 연구를 통해 한국에 특허 12건, 미국 3건, 일본 3건, 유럽과 중국에 각각 1건씩의 특허를 등록한 상태다. 이 대표는 "효소란 주로 단백질로 이뤄져 있다. 단백질을 인체에 투약하면 체내의 외부물질이 유입되는 것이라 면역반응이 일어난다. 과다한 면역반응은 염증을 일으키는 부작용이 있는데, RNA로 구성된 효소는 면역원성이 없기 때문에 안전성이 두드러진다"고 설명했다. 그는 "유전자편집가위(CRISPR)는 인간유전체(지놈/Genome)를 영구적으로 변형시켜버리지만 RNA 치환은 DNA를 건드리지 않는 장점이 있다"고 말했다. 알지노믹스의 RNA치환 방식에선 체내에 유입된 RNA가 계속해서 치료물질을 생성하며 병환을 개선시킨다. 이 대표는 "대신 약물이 주입된 세포가 계속 분열하는 경우엔 약효가 희석(Dilution)되기 때문에 약을 꾸준히 주입해야하는 단점이 있을 수 있다"며 "이 때문에 세포분열이 활발하지 않은 근육세포, 신경세포, 망막세포 치료제에 집중하고 있다"고 덧붙였다. 이 대표는 1995년 미국 코넬대학교에서 분자유전학 박사과정을 밟고 듀크대학교에서 RNA 연구에 매진했다. 1997년 단국대 교수로 부임하며 한국에 돌아와 리보자임 기술을 계속 다듬었고 연구 성과가 가시화 되자 2017년 8월 알지노믹스를 설립했다. 알지노믹스는 2019년 9월 시리즈 A로 120억원을 유치하고 2021년 4월 시리즈 B에서 105억원을 조달했다. 주요 재무적 투자자(FI)로는 시너지IB투자, LSK인베스트, 파트너스인베스트, 쿼드자산운용, 산업은행, SBI인베스트 등이 있따. 현재 임직원은 25명 정도다. 핵심인력은 설립 때부터 연구개발이사 역할을 수행 중인 한승렬 박사와 작년 1월 합류한 홍성우 부사장(CDO)이다. 한 박사는 단국대에서부터 이 대표와 RNA 연구를 해온 사제 관계다. 홍 부사장은 CJ, 오스카, 보령제약 등 제약회사와 바이오벤처에서 20년 경력을 쌓았다. 전임상, CMC, 임상 등 개발을 총괄하기 위해 2020년 1월 영입됐다. 올해 말 또는 내년 초 단행할 250억~300억원 규모의 시리즈 C에 앞서 재무총괄(CFO), 사업 운영총괄(COO) 등의 인사도 확충할 계획이다. 이 대표는 "전임상 단계에서 기술수출(L/O)을 준비하고 있다"며 "2023년 기술성특례상장을 위해 7월 초까진 주관사 선정을 마무리 할 계획"이라고 말했다. 알지노믹스 파이프라인은 RNA치환효소 다발이 커서 체내에 전달할 때 DNA 벡터를 이용한다. 항암용 DNA 벡터엔 아데노바이러스를 쓰기 때문에 적절한 생산세포주가 필요하다. 작년 독일 세벡(CEVEC)에서 생산세포주를 도입하고 미국 바이진 바이오사이언스와 위탁개발생산(CDMO) 계약을 체결했고 현재 GMP 생산 중이다. 간암치료제 핵심파이프라인 RZ-001의 미국 및 국내 임상 추진을 위해서다. 이 외 유전성망막질환 치료 파이프라인 RZ-004와 알츠하이머 치료 파이프라인 RZ-003은 각각 2023년과 2024년 임상을 계획하고 있으며 AAV벡터를 사용할 예정이다.
2021-05-27
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