Harvard University announced on the 14th that it will grant Beam Therapeutics (BT) a global patent license to develop and commercialize a set of revolutionary DNA base editing technologies that can be used to treat human diseases.
BT announced on the same day that it had raised up to $87 million in Series A financing led by F-Prime Capital and ARCH Ventures. BT was jointly founded by Liu Ruqian, Zhang Feng and Keith Yang, leaders in gene editing technology.
Vivien Boleyn, director of business development at Harvard’s Technology Development Office, said: “Base editing represents a powerful platform for addressing a wide range of genetic diseases that are difficult to solve with other genome editing methods.â€
The reporter learned that the licensed technology platform includes base editing and techniques related to enhanced base editing targeting, opening the door to the use of gene editing technology to treat a range of human genetic diseases.
CRISPR gene editing: It is artifact but it also has short board
In the human genome, tens of thousands of mutations in known DNA base (A, C, G, T) sequences can cause disease. Most disease-related human DNA variants consist of site mutations, or a single base pair in the genome is displaced by another base pair. This mutation has been demonstrated in the study of various genetic diseases such as neurodegenerative diseases, metabolic diseases, hematological diseases, vision or hearing loss.
The use of the CRISPR platform in combination with the genome editing techniques of the Cas9 and Cpfl enzymes has shown great promise for the regulation of genes by insertion or deletion of multiple nucleotides, but this technique is difficult to correct single nucleotides cleanly and efficiently.
Existing genome editing methods use CRISPR as a molecular scissors for double-stranded breaks, and then rely on the introduced DNA template for guided correction to attempt to correct point mutations.
However, intracellular double-strand breaks trigger the process of reconnecting the broken ends and result in the negative effects of random insertions and deletions.
Therefore, accurate correction of point mutations often must compete with these unwanted by-products. In addition, precise correction using CRISPR/Cas9 typically relies on cellular components, and these cells are missing in non-actively dividing cells, the majority of cells in the body.
Base Editing Techniques: Point-to-Point Accurate Targeting
The base-editing technology multi-functional platform was invented by Liu Ruqian, a professor of chemistry and chemical biology at Harvard University. Base-editing techniques are not precisely correcting disease-causing mutations in specific genes, but rather cutting target sites that disrupt genes or create mutations. The programmable molecular machine developed by Liu Ruqian team can access the target site in the selected cell genomic DNA and directly convert one base into another without simultaneously producing a double-strand break in the DNA.
This technique uses an engineered multi-component protein containing modified Cas9 to unwind the targeting portion of the DNA helix, opening a small window for manipulation on a single base without the need to cause double strand breaks in the DNA. Base editing then directly converts the target gene from the mutated form to the correct form, and in some cases, it also needs to add a protein component to prevent the cell from undoing the correction.
At the same time, the improved Cas9 cuts the unedited DNA strand, causing the cell to repair the second strand with a base that can complement the modified base. The result is that double swapping will permanently change the entire base pair (eg, A·T) to a different base pair (eg, G·C).
In the past one and a half years, Liu Ruqian team has greatly expanded the scope of the base editing technology. Expanding the targeting range, increasing targeted DNA specificity and creating new base editors will have a major impact on the treatment of genetic diseases.
Ultimate goal: fundamentally change medicine
Liu Ruqian said: "The ultimate goal is to change DNA bases to another DNA base in an unmodified organism, whether human, plant or animal, at will, cleanly and efficiently."
Bolin said: “Our goal is to develop this innovative technology as the most transformative treatment for human diseases. Licensing a startup company for commercial development will ensure rapid mobilization of resources and full development and use of new technologies in this area. ."
John Evans, chief executive of BT, pointed out that base editing technology can complete single-base modification with high efficiency and unprecedented control, and BT will fuse the key technologies of base editing to produce a wide range of accurate gene drugs. The pipeline repairs mutations in pathogenic sites, writes protective genetic variants, or regulates the expression or function of disease-causing genes, ultimately making base editing a treatment option for human diseases.
Isaac Kohlberg, a senior assistant dean and chief technology development officer at Harvard University, said: "The genome editing and base editing technologies fully reflect Harvard University researchers' continuous and important contributions to biomedical innovation, and this progress is Opportunities for economic development and social benefits. This opportunity not only focuses on the treatment of diseases, but also fundamentally changes the practice of medicine.†(Reporter Feng Weidong)
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