일본 최대 제약회사 다케다제약에서 분사하여 설립된 일본 최초의 신약 솔루션 제공업체인 Axcelead Drug Discovery Partners (ADDP)와 KRCC, SEKISUI MEDICAL이 공동주최하는 웨비나를 개최합니다.
주제： How to proceed GLP toxicity study without cynomolgus monkeys
일시：2023년 9월 20일(수요일) 오후 16시 ~ 17시
위치：온라인 (zoom meeting)
1. Knock-in animals are useful in place of cynos in GLP toxicity study
As the number of approved biopharmaceuticals (new modality drugs) has increased, sales of biopharmaceuticals (new modality drugs) have increased in Japan as well as the United States. As the development of biopharmaceuticals expands, the diversification of modalities is accelerating. The development of new modality drugs has been increasingly driven by academia or drug discovery venture companies. New modality drugs are peptide drugs, antibody drugs, nucleic acid drugs, gene therapy products, and cell therapies. There are also a wide range of exosome preparations.
Species selection is important in non-clinical safety studies of new modality drugs. This is because regulatory authorities are required to “evaluate on-target toxicity in non-clinical toxicity studies.” Therefore, non-clinical safety studies of new modality drugs, which are known to be less likely to induce off target toxicity, require study in animals that show efficacy. Cynomolgus monkeys (cynos) have high homology and crossover with humans, and their pharmacological effects often only appear in cynos, so cynos are selected as appropriate animal species for non-clinical safety studies. However, since the end of last year, the price of monkeys has soared, import restrictions have made it difficult to conduct non-clinical safety studies using cynos, and many projects seem to have been canceled or suspended. Knock-in mice/rats may solve the problem of high prices and supply shortages in cynos.
2. Knock-in mice/rats in Axcelead: Key Features and uniqueness
Human DNA KI (Knock-In) mice or rats are useful for evaluation of on-target toxicity of drugs with species differences including new modality drugs. Human DNA KI mice were successfully obtained by CRISPR/Cas9 gRNA mixture with targeting vector plasmid by pronuclear injection. Long-DNA KI mice that retain the exon-intron structure are useful for the evaluation of nucleic acid drugs. It has been succeeded in deleting the endogenous mouse gene and replacing with a human gene with exon/intron structure (40Kbp) by homologous recombination using ES cells.
We would like to discuss the usefulness of human gene KI mice or rats.
Hideo Fukui, PhD, DABT, DJSOT
Axcelead Drug Discovery Partners Inc, Applied Regulatory Science Senior Director/Senior Consultant
1989 M.S. in Agriculture (Major: Nutritional Biochemistry), Nagoya University, Nagoya, Japan
1989 Research Scientist, Toxicologist, Drug Safety Research
Laboratories (DSRL), Takeda Chemical Industries, Ltd., Osaka, Japan
1994 Ph.D. in Pharmacological Science, Kyoto University, Kyoto, Japan
2000 Visiting Scientist, Edward S. Harkness Eye Institute, Department
of Ophthalmology, Columbia University Medical Center, NY, USA
2002 Diplomate, Japanese Society of Toxicology (DJSOT), certified
2004 Diplomate, American Board of Toxicology (DABT), certified
2014 Drug Safety Therapeutic Area Lead for GI (Gastro-Intestine) in
Global Drug Safety Research and Evaluation, Takeda
Pharmaceutical Company Limited, Japan
2017 Director, Nonclinical Safety Research, Partnership Research
Center, Research, Takeda Pharmaceutical Company Limited, Japan
2018 Director, Nonclinical Safety Research, Axcelead Drug Discovery
Partners, Inc., Japan
2020 Senior director/Senior consultant, Nonclinical Development
Strategy, Applied Regulatory Science, Axcelead Drug Discovery
Partners, Inc., Japan
2021 President of the 48th Japanese Society of Toxicology(JSOT) annual meeting in 2021 (Kobe, Japan)
Executive board member of the JSOT (2016-2020),
Observer of executive board of the JSOT (2020-2022)
Michiyasu Takeyama, PhD
Axcelead Drug Discovery Partners Inc, Integrated & Translational Research Director
1991 PhD in Engineering, Osaka University
1991 Biotechnology Research Laboratories, Takeda Chemical Industries, Ltd.
2006 Biological Research Laboratories
2017 Axcelead Drug Discovery Partners, Inc.