With the orientation on human health and Healthy China Movement, GIBH focuses on cutting-edge scientific issues and the pathogenesis of major diseases and strives to be a world-class R&D institute of biomedicine and health as well as a high-level incubator for innovative talents. It undertakes the mission of producing original fundamental theories, procuring technology breakthroughs and providing systematic solutions for health and disease prevention and control, with the expectation to optimize the rapid commercialization of scientific and technological achievements, to meet the national strategic and regional economic and social needs, to promote the biopharmaceutical industry, and to support national strategic science and technology programs.

GIBH is now mainly engaged in the research of stem cells and regenerative medicine, chemical biology, infection and immunity, public health, and scientific equipment R&D, Currently, the institute contains five research units including Center for Cell Lineage Atlas, Center for Cell Lineage Technology and Engineering, Center for Biomedical Digital Science, Institute of Development and Regeneration, as well as Institute of Drug Discovery. In addition, there are five research facilities at GIBH, including Analytical Instrumentation Core, Laboratory Animal Core, Biomedical Data Analytics and Supercomputing Core,Therapeutic Cell Manufacturing and Research Core, and Featured Support Platforms.

GlBH gathers and cultivates a group of committed talents. At present, there are 418 employees and 554 students, including 529 graduate students. Scientists from GIBH have been awarded with many high academic honors such as the National Talent Program, the National Science Fund for Distinguished Young Scholars, the National Science Fund for Outstanding Young Scholars, the National High-Level Foreign Expert Introduction Program, the Chinese Government Friendship Award, the CAS Talent Introduction Program, the CAS Young Talent Program, the South Guangdong Innovation Award, as well as the Guangdong Provincial Talent Plan, etc.

GIBH not only provides doctoral programs in Biology, Basic Medicine as well as Medicinal Chemistry, but also offers master programs in Materials and Chemical Engineering, as well as Biology and Medicine. The institute is also running several education programs in collaboration with multiple top universities in China, including University of Science and Technology of China, Jilin University, Guangzhou Medical University, Shenyang Pharmaceutical University and Shandong First Medical University, covering undergraduate, master and doctoral levels. Those educational and training programs have been attracting students from all over the world, such as Spain, India, Pakistan and Nepal.

Up till now, more than 1400 projects have been funded by China's Ministry of Science and Technology, National Natural Science Foundation, CAS, Guangdong provincial and Guangzhou municipal government. 2285 papers have been published. 2227 of them have been accepted by SCI. Many awards and honors have been received, including two second prizes of National Natural Science Award, one Outstanding Scientific and Technological Achievement Award of CAS, two South Guangdong Outstanding Contribution and Innovation Award, seven first prizes of Guangdong Science and Technology Award, and three first prizes of Guangzhou Science and Technology Award.

GIBH is committed to promoting local industries through science and technology, and attaches great importance to local engagements for the translation and commercialization of research findings. So far, 958 patents (722 domestic and 139 international) and 97 PCT patents have been applied for. 471 of them have been licensed (410 domestic and  61 international) and 4 of which have been granted with China Patent Excellence Award and Guangdong Patent Excellence Award. In addition,three self-developed drugs have been approved for clinical trials by China's Medical Products Administration (NMPA), including GZD824 (a first-in-class anti-leukemia drug), ASK120067(a first-in-class anti-tumor drug), as well as AD16 (a first-in-class anti-Alzheimer drug). Moreover, GZD824, also known as Olverembatinib Tablets, has been authorized for marketing on November 24, 2021. ASK120067 has been submitted to the Center for Drug Evaluation of NMPA. AD16 has been approved to enter Phase l clinical study.

GIBH has co-established the State Key Laboratory of Respiratory Diseases with Guangzhou Institute of Respiratory Health, the Key Laboratory of Regenerative Biology of CAS, the Guangdong Provincial Key Laboratory of Stem Cell and Regenerative Medicine as well as Guangdong Provincial Key Laboratory of Biomedical Computing, and has supported the foundation of Guangzhou Bio-island Lab as well as the Stem Cell innovation Center of CAS's Hong Kong Institute of Science and Innovation.

GIBH has forged close bonds with international research institutions and universities, including Germany's Max Planck Society and the University of Auckland in New Zealand. In collaboration with top universities and research institutes worldwide, GIBH have established International Joint Research Center for Biomedicine and Health, International Science Cooperation Base for Stem Cell, and Regenerative Medicine and Max Planck-GIBH Research Center. Moreover, GIBH has successfully organized Guangzhou International Stem Cell and Regenerative Medicine Annual Meeting for 14 years, in which several Nobel Laureates were invited as keynote speakers.