Novel therapeutics and technologies
The COVID-19 pandemic showed the world the power of RNA-based therapeutics. The foundational research that gave rise to the life-saving mRNA vaccines was the result of decades of work from scientists spanning multiple disciplines, such as medicine, engineering, chemistry and biochemistry. At the 平特五不中 Centre for RNA Sciences (MCRS), we鈥檝e adopted this interdisciplinary approach. Our Centre brings together researchers across many fields鈥攆rom genomics to biotechnology to AI鈥攖o both unravel the underlying biology of RNA and harness the power of these molecules as novel therapeutics for a broad range of human ailments: infectious diseases, cancers, and rare diseases.
MCRS investigators will apply this cohesive approach to both the basic and applied aspects of RNA sciences. Our research programs will pursue fundamental insights into the biology and chemistry of RNA and facilitate the development of emerging RNA technologies in the areas of biologics, small molecules, and bioprocessing.
Fundamental insights
RNA is a versatile molecule that performs a multitude of roles with the body, including informational, regulatory, and enzymatic functions. Probing the chemistry and biology of RNA will enable our researchers to better tailor RNA for therapeutic applications, and to reveal potential new avenues for RNA-based treatments.
The projects at MCRS examine the structures, functions, synthesis, and interactions of different types of RNA, such as messenger RNA鈥攖he molecules carrying the information required for protein production鈥攁nd non-coding RNA, which do not carry code for proteins but are involved in other important biological activities. Our experts are also investigating the molecular biology of viral pathogens such as the human immunodeficiency virus (HIV), Zika, Ebola, and SARS-CoV-2 (the virus behind COVID-19) to elucidate the pathways underlying their infectivity and pathogenicity. The knowledge gained from this basic research will enable scientists to design new RNA-based technologies for infections, cancer, and other diseases.
Novel therapeutics and technologies
RNA therapeutics come in many different shapes and sizes. To understand how best to tailor these molecules for use in biotechnology and medicine, the researchers at MCRS are tackling the issue from various angles. These include the identification of new targets for rare diseases and cancer types using methods such as single-cell RNA sequencing; the creation of RNA-based biosensors that can detect diseases or identify toxic compounds in our cells and the environment; the development of novel biophysical tools to better characterize how RNA functions; and the use of computational methods such as artificial intelligence and machine learning to accelerate our understanding of the role of RNA in disease.
Toward the clinic
MCRS and group-led initiatives will facilitate the translation of discoveries and innovation towards the clinic and industry. Designer RNA therapies will be guided from the discovery stage towards clinical translation and commercialization, and innovative RNA technologies will be developed and scaled for industrial applications. MCRS will progressively plan, build, and support innovative technology platforms that will revolve around three main functions:
- RNA precision medicine platforms and technologies for identifying disease-specific transcriptomic signatures and potential therapeutic targets and to test candidate treatment approaches in pre-clinical models.
- Nucleic acid synthesis and chemistry platforms to quickly engineer and generate nucleic acid analogs and inhibitors of RNA-directed processes for structural, biological, and therapeutic assays.
- RNA bioprocessing and delivery platforms to develop and test high-quality RNA-based therapeutics and technologies and their delivery formulations, such as lipid nanoparticles, with capacity for clinical-grade manufacturing at various scales.
Preparing the future generation of leaders
The academic mission of MCRS is to train highly qualified personnel in all areas of the RNA sciences to fulfill the expert capacity demands arising from the rapid growth of the RNA industry. The Centre will develop and implement innovative inter-disciplinary training programs that integrate RNA science, medicine, engineering, machine learning and informatics, capitalizing on the established strengths of 平特五不中 and its clinical and industrial partners.
Trainees will acquire technical proficiency through working with the cutting-edge innovation platforms developed by MCRS and partnering institutions, with supervision through partnerships between core directors and highly qualified research professionals.
Forging partnerships
MCRS will serve as a facilitator for collaboration with leading industrial partners. The Centre will ultimately develop mechanisms to facilitate the commercialization of discoveries made by 平特五不中鈥檚 trainees and investigators through spin-offs, incubators, and partnerships including the licensing of intellectual property.