Gene editing is a group of technologies that allow genetic material to be added, removed, or altered at desired locations in the genome. Among the gene editing technologies developed, CRISPR-Cas9 has become the fundamental gene editing technology because it is faster, cheaper, more accurate, and more efficient than other genome editing methods. Despite the above advantages, the double strand breaks (DSB) induced by CRISPR-Cas9 could result in chromosomal truncation, large DNA fragment deletion and other chromosomal aberrations. To reduce such potential risks, base editing and prime editing technologies were developed to edit genome without generating DSB. (Figure 1).
Figure 1 overview of gene editing technologies (Cell (2022), https://doi.org/10.1016/j.cell.2022.03.045)
Two major classes of base editors have been developed: cytidine base editors or CBEs allowing C>T conversions and adenine base editors or ABEs allowing A>G conversions (Figure 2).
Figure 2 Cas-deaminase–base editors can generate single-nucleotide changes in DNA (Science (2019), doi: 10.1126/science.aax1827)
Base Therapeutics develops the next generation base editor (BE). Given that base editing both ABEs and CBEs can create substantial off-target edits on RNA, whereas CBEs additionally has off-target effects on DNA, Base Therapeutics developed cas-embeded base editors (ceBEs). Our patented ceBE has higher editing efficiency than the most commonly used editing tools. Additionally, it has nearly zero off-target effect, which offers great safety measures when editing human cells. The ceBEs has broad applications in basic research, treatment of genetic disease, autoimmune disease, cancer, agriculture, and veterinary medicine.
Prime editing is a ‘search-and-replace’ genome editing technology in molecular biology by which the genome of living organisms may be modified. The technology directly writes new genetic information into a targeted DNA site. It mediates targeted insertions, deletions, and base-to-base conversions without the need for double strand breaks (DSBs) or donor DNA templates. The technology is an early-stage, experimental genome editing method that has received mainstream press attention due to its potential uses in medical genetics. Our enhanced prime editor (“ePE”) is the most efficient PE in the current industry.
Base Therapeutics’ first product NK510 is a first-in-class, peripheral blood-derived base-edited Super-NK cell product and is currently in three IIT trials. Our Super-iNK is a first-in-class iPSC-derived, base-edited and site-specific integrated Super-CAR-NK cell product.
The best science and the best medicines come from the combined effort and expertise and resources of the right people and organization. We are looking for partners to establish research collaborations, co-development and license agreements for our ceBE base editor in oncology and genetic disease, as well as our Super-NK & Super-iNK products.