Enhanced prime editing system with increased editing efficiency for more effective gene-based therapies
Opportunity
Prime editing is a CRISPR-based genome editing technology that holds tremendous promise for the treatment of monogenic diseases through the permanent correct of disease-causing mutations in the genome. It represents the most versatile genome editing tool, with its capability to mediate targeted insertions, deletions, and base-to-base conversions, without the need for double-stranded breaks.
The global prime editing and CRISPR market was valued at US$2.69 B in 2020, and is projected to reach US$24.5B by 2030, growing at a CAGR of 24.3% from 2021 to 2030. This is driven by the increasing interest in development of gene therapies as a novel therapeutic modality as well as its application to disease indications with larger patient populations.
However, the technology is still in its infancy and has several limitations. In particular, the low editing efficiency remains a critical challenge.
Technology
Prime editing system comprises of the prime editor enzyme and a pegRNA (prime editing guide RNA) that specifies the target site and encodes the desired edit.
The NUS research team has generated an improved version of the prime editor with enhanced editing efficiency, with 1.3-1.5 fold higher editing efficiency than Prime Editor 2 (PE2).
In addition, the team has also identified a new targeting strategy using paired pegRNAs in trans, which leads to significantly higher editing efficiency. The dual pegRNA strategy provides a 1.8-2 fold increase over single pegRNA, and a 1.5 fold over PE3 system.
There is ongoing studies to validate the system in vivo.
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