AI technology-based system applied in rare
disease treatment leads to the development of new treatments
Under a ‘pilot project to build bio-big
data for all ministries,’ promoted by three ministries including the Ministry
of Science and ICT, big data on genomic information, such as rare diseases, of
20,000 people is being established. With this, there is a growing interest in
developing medicines for rare diseases. Dr. Noa Biotech, a drug development
company that uses AI technology, continues to develop new drugs with focus on
rare disease treatment through its own platform.
◆ Development of Rare Disease Treatment Using
an Independent Platform
DR stands for doctor, as well as "drug
repositioning," which means creating new drugs from existing drugs. Noah
is a biblical figure who made an ark based on his faith in the Bible to help
save the world. As such, the mission of Dr. Noah Biotech is conveyed in its
name. Lee Ji-hyun, the CEO of Dr. Noah Biotech, explains that the company is
determined to develop treatments for rare diseases more quickly and efficiently
by exploring the hidden effects of existing drugs that are confirmed to be safe
and using them to compose new compounds.
"People laughed at Noah for making his
own ark in the Bible. But during the great flood, all life was destroyed, but
the life on Noah's ark. Dr. Noah Biotech strives for the same. It may seem
reckless to use AI to develop treatments for rare diseases, but we are
confident that our technology and strategy are right. We hope that many
patients can benefit from our technology."
Dr. Noah Biotech recently signed an
agreement with Kainos Medicine to develop new drugs using the ARK platform. CEO
Lee said, "From predicting and validating the effects to optimizing them to
make final products for clinical tests, each company will lead and take on
responsibilities for the parts it is best at. I am looking forward to the
synergy effect during this joint effort."
The ARK platform was developed by Dr. Noah Biotech
to predict new drugs and optimal combinations. The platform is composed of
three databases, including NOTE-R, which contains research information on drugs
(literature, clinical, and side effects), and NOTE-P, a drug genomics DB.
In addition, artificial intelligence prediction
systems that utilize this data include CombiNet, which predicts compounds using
all data; AI Target Finder, which seeks new disease target genes; AI Virtual
Compound Screening System, which seeks new drug structures; and AI Virtual
Target Screening system, which can find off-targets for drugs. CEO Lee
explained that they are using various DBs and systems because each rare disease
requires different strategies for developing new drugs. In addition, various
methods of analysis are introduced to overcome the limitations of relatively
limited data available for rare diseases, compared to cancer or metabolic diseases
that have been studied a lot.
“The ARK platform can be used to develop
treatments for a variety of diseases as well as rare diseases. Internally, we
are focusing on developing treatments for rare diseases, and in joint
development with external researchers, we are studying a variety of diseases,
◆ Accelerating the development of new drugs
Companies working on developing new drug
are very interested in predicting the structures of new compounds. Dr. Noah
Biotech presents Hit Compound using AI Virtual Compound Screening system for
disease target proteins. This is a mandatory process in drug development because
we need to find out what side effects can appear. AI Virtual Target Screening
System explores the possibility of unexpected side effects.
While numerous drugs entering the clinical
trial stage are suffering from insufficient data on medicinal effects, Dr. Noah
Biotech presents alternatives with its original technology. CEO Lee remarked, "Even though sufficient
research has been conducted during R&D stage, clinical trials on actual
patients often do not show enough effectiveness to prove statistical
significance.” "From the perspective of pharmaceutical companies, they are
forced to abandon their research results." On that note, Dr. Noah Biotech
offers two options to overcome this crisis. First is to develop a new drug with
higher efficacy, to the extent it could mark a new turning point. Second is to
re-develop a drug to target the same disease, but recommend a compound partner
drug to increase the effectiveness. Lee added, "If we can find a new drug
that has enhanced effectiveness of existing drugs, we can proceed with the
development more easily."
Based on these technologies, Dr. Noah Biotech
is currently discussing and proceeding on various projects with Korean new drug
developers to predict the structure of new compounds, predict side effects, and
develop treatments for rare diseases. Lee emphasized the significance of these
joint development projects, explaining that the collaboration with experts in
various fields is essential in the long and complex new drug development
◆ Targeting 5 or more clinical entries within
The key component in the AI system is data.
Fortunately, due to the nature of the bio industry, a lot of research data has
been already gathered as it is recommended practice to disclose data used in a research
when publishing papers. In an attempt to secure meaningful data, Dr. Noah Biotech
created Data Management Team to perform manual curation of data and exclude unsuitable
data. CEO Lee Ji-hyun said, "It may seem like nothing from the outside,
but when we develop an AI system based on carefully selected data, the results
will show a big difference."
Lee also said that Dr. Noah Biotech will
produce data on their own in the future, even though it is selecting from
public data right now. This is because limited data is available for use in
developing drugs for rare diseases. On that note, Dr. Noah Biotech is planning to
form partnerships with global companies that specialize in diagnosing rare
diseases. By doing so, it is expected that the stem cells and genetic information
of patients with rare diseases will be obtained, and data and samples of the
same patients will be used in drug prediction and verification. She added,
"There have been regulations in Korea so far on obtaining blood samples and
genetic data, but we are anticipating and preparing for the possible change in the
regulatory conditions in the future."
As a result of its steady efforts, Dr. Noah
Biotech's first pipeline is now cruising. Initially, it was a drug that
targeted rare diseases that had problems with neuronal differentiation.
However, the target disease group has expanded to include post-stroke recovery
and TBI (Traumatic Brain Injury). Lee added, "We have shown good results
in animal testing, so we are working on preclinical development with the aim of
proceeding with clinical trial by next year." Their second pipeline is treatment
for Lou Gehrig's disease. The drug has been tested on the stem cells of Lou
Gehrig patients and now being tested on animals. CEO Lee explained that two
other rare disease pipelines are under development, and that she is hoping to
make five or more pipelines to enter clinical trials within three years.
Furthermore, she hopes to make the company specialized in developing and producing
neurological drugs on its own.
◆ A company that endeavors to make its
employees happy and bring hopes to the patients.
"From the beginning, Dr. Noah Biotech
was founded with the goal of developing new drugs based on data analysis. At
the time of its establishment, there were companies that were interested in
diagnosis and application development, but none of them wanted to develop drugs
directly from system development. I started a business with a single goal of
expanding my research to develop a new medicine."
For CEO Lee Ji-hyun, starting a business
was a challenge to a new world. She was a research professor at Seoul National
University when she started the business. At the time, she did not have any experience
working in a company either. Lee recalled the time, saying that it was
difficult getting investment and she was not familiar with the process. There was
no one willing to invest in a small company that did not have products with verified
medicinal effects. She was looking for a place where drug efficacy can be
verified, and she was able to get the verification through the support task of
the Korea Research Institute of Chemical Technology. Using this as a stepping
stone, she was able to attract investment. Lee said she was able to attract the
first investment with the help of people, and this enabled her to jump into the
business. In its fourth year, Dr. Noah Biotech is still close to a start-up
company, but Lee's eagerness to develop treatments and the technical competence
of the company are giving an assurance to many people. She said that developing
treatment is her calling, and it is not just about making money. Rather, it is
about life and the process of moving toward the final goal. This sense of
calling is also reflected in Dr. Noah Biotech's endeavors, one of which is
organizing a campaign for Rare Disease Day to improve awareness. Last year,
three start-ups gathered to form a "start-up alliance." They agreed
to help patients suffering from rare and incurable diseases by sharing their
strengths in diagnosing those conditions, managing and distributing data, and
developing treatments. Lee said that she wants to open a YouTube channel to
share useful information about rare and incurable diseases with people. Dr. Noah
Biotech will be continuing its campaign.
When it comes to running a company, Lee
places most importance on employee’s satisfaction. She is trying to achieve
work life balance of employees by eliminating overtime work, having the company
get-together during lunch, and discouraging people from communicating through
Kakao Talk. Lee expressed that she would like to see all employees focus on
work during the work time and spend the evening after work with their family
and friends. This is based on her belief that only when the employees get to
refresh outside work that they can concentrate on their job and are able to come
up with more creative ideas.
As Lee runs a venture company, her
philosophy is "to be careful in decision making and be prompt in
execution." She believes that the sense of speed is the biggest strength
of a venture company. Since not all of the company's projects can be
successful, she aims to launch as many projects as possible in a short period
of time and minimize risks by quickly deciding which ones to pursue further,
based on the initial performance and progress. The plan is to quickly determine
a few viable projects for clinical tests.
Finally, she envisions nurturing a great
pharmaceutical company based on technology. She has a dream of developing
treatment to help people, and she believes that having a platform could make
that dream come true in a smart way. As Dr. Noah Biotech Co., Ltd. continues to
develop treatments for the world through its own platform, it is confident that
it will bring a new hope to patients with rare diseases around the world.
People May 2020 p76-79