Vision: The Target Factory
An Integrated Research Platform for Novel Target and Lead Discovery
The Medicinal Bioconvergence Research Center of Seoul National University (or Biocon) has set a challenge to establish an “innovative research platform for drug discovery” that can significantly reduce the time and cost while improving the success probability of drug discovery.
Among the many different stages required for drug development, Biocon focuses on the “identification and validation of novel drug targets”. Although well-validated druggable targets are critical determinants for the success of drug discovery, target discovery is typically a function that pharma or biotech industry launch in-house, because it involves much basic science and there are no standard operation procedures for this process. Therefore, druggable targets have to be provided by the researchers in academia and public sectors. Biocon was established to fill the gap between academia and industry by maturing potentially druggable targets.
The identification of novel targets involves a broad spectrum of research activity from in-depth basic biology to translation. The strength of Biocon is based on the integration of biology and chemistry teams combined with cutting-edge convergence technologies. Through this fully united research teamwork, Biocon is aiming to become the world’s first and best target factory, providing innovative targets to the pharmaceutical industry.
Searching for Novel Targets
The need for new drugs is ever increasing due to the surge in diseases such as cancer, diabetes and dementia that are mainly caused by extended life span. However, the number of newly approved drugs is continuously decreasing, giving a crisis not only for drug industry but also eventually for human health.
In 2010, the Korean Ministry of Education, Science and Technology (MEST) launched the Global Frontier Project, targeted at solving global challenges in major R&D areas. The Medicinal Bioconvergence Research Center (Biocon) was initiated as one of the three projects with the aim of resolving difficulties during the development of novel drug discovery. This project will proceed for an initial nine years from 2010 to 2019, and is divided into three
stages (1st: 2 years, 2nd: 3 years, 3rd: 4 years). With the financial support of about 140 million dollars and a projected time-line of 9 years, Biocon has leveraged a unique capability to discover and validate molecular pathways and targets at a level that is possible at few commercial enterprises.
4 specialist groups + 1 integration core group
Biocon seeks innovation across all of its activities including research, technology and strategy. At its core, Biocon is remodeling the R&D process itself. In the current global drug development process, target discovery, drug design, drug screening and disease model are generally addressed in a linear fashion, much like a factory product assembly line. In this system, the downstream processes totally depend on the upstream, and any errors occurring upstream can be cumulative since there is little communication between the different research functions. This situation can be one of the attributes to the high failure rate of drug discovery. To avoid this problem, Biocon has built a unique “4+1” matrix system.
Specifically, four specialist groups have been established for target identification, drug design, drug screening and disease modeling. The discoveries and inventions generated from these four units are integrated, validated and matured in the integration core unit (ICU). This way, the time and cost for target validation and discovery can be significantly reduced. The team also includes the specialists from clinical and pharma industry to orient target development according to the clinical and industry unmet needs.
In addition, Biocon actively seeks and nurtures technical innovation by adopting cutting-edge technologies into bioscience. To achieve this goal, each project is driven by a under the technical framework that we call S.A.F.E. (standing for Smart, Accurate, Fast, Economic). Through these integrated biology and convergence technologies, Biocon seeks to build up world’s most efficient operational research system for target and lead discovery.
Oncology and other complex diseases
Biocon focuses on human aminoacyl-tRNA synthetases (ARSs) and their interacting proteins as our primary target space of interest, and explores their potential to address diverse human diseases. These enzymes emerged early in evolution and catalytically link their cognate amino acids to tRNAs for protein synthesis.
For this reason, ARSs have been traditionally considered as housekeeping proteins solely dedicated to protein synthesis. However, recent new findings from Biocon and other groups world-wide on the novel functions of ARSs are rapidly changing the classical view of these enzymes. Indeed ARSs are emerging as central coordinators linking cell fate determination processes with protein synthesis.
Translation & Drug Development
Well-matured Leads are Linked to Industry
With excellent research achievements during last five years of Biocon, it is time to put in place a flexible and professional management group that can assess, decide and implement decisions for downstream development that are independent of, but in co-operation with, the underlying scientific mission of Biocon. The action of this group needs to be focused
and operated at the standard of global Pharmas since Biocon’s assets in the pipeline are all “first-in-class”. In light of the imperative to move programs from the academic to a translational setting, Biocon has established a TRAnslation and DEvelopment Group (TRADE), comprised of experienced professionals from the global drug development industry.
The TRADE group, led by the Biocon’s Chief Business Officer, has a mandate to take Biocon’s inventions and capabilities to global drug development partners. Currently, the TRADE group carries out rigorous assessment of internal Biocon programs with a particular emphasis on matching the target discovery and validation progress of Biocon with unmet market needs or opportunities.