Process Development of sgRNA in CRISPR/CAS Therapeutics

Sungwon Kim, Ph.D. Head of Oligonucleotide R&D at ST Pharm

Process Development of sgRNA in CRISPR/CAS Therapeutics

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Sungwon Kim, Ph.D., heads Oligonucleotide R&D at ST Pharm, with over 15 years of experience in process development and manufacturing of oligonucleotide APIs. While working at ST Pharm, he has expanded his capabilities in developing diverse types of oligonucleotides. Furthermore, he played an essential role in implementing microbial control for GMP production which require stringent control. Recently, he took the lead in establishing the process for developing and manufacturing long oligonucleotides, e.g., single guide RNA (sgRNA). He holds a Ph.D. in bioengineering.

The CRISPR/Casx system is widely recognized as a breakthrough technology for precisely editing DNA sequences, allowing for the removal, addition, or alteration of genetic material. Comprising two crucial elements, the system features the enzymatic scissor, Casx, and the guide RNA, known as single guide RNA (sgRNA), tasked with precision in genome targeting. sgRNA can be generated through cell transcription, in vitro transcription (IVT), or solid-supported synthesis. The growing demand for solid-supported synthesis, particularly for longmers (>100mer), to meet therapeutic needs presents unique challenges compared to ASO or siRNA synthesis. This presentation will delve into the outcomes of process optimization for longmer preparation, shedding light on the impact of key parameters.