Company Overview:
Editas Medicine (EDIT) is a Massachusetts-based genome editing company focused on treating people with genetically defined diseases. Achieving success at Editas Medicine is realizing the promise that was born from the sequencing of the human genome - treating diseases at the DNA level. The company intends on using CRISPR technology to make a meaningful impact across a wide array of disease areas: eye, muscle, blood, lung, liver and cancer. The company's pipeline is comprised of NHEJ and HDR editing mechanisms via AAV, RNP, and LNP delivery. The pipeline is in its infancy with all drug candidates in the discovery phase.
CRISPR:
CRISPR is a technology that uses a protein-RNA-complex comprised of either Cas9 or Cpf1. Both bind to a guide RNA molecule, gRNA, that has been specially designed to recognize a particular DNA sequence. Cas9 and Cpf1 are programmed to different or multiple sites using multiple gRNAs. The engineered variants of Cas9 and Cpf1 enable different types of cuts for gene editing.
The company utilizes two different types of repair mechanisms, NHEJ and HDR. In the cut and revise and cut and remove approaches, problem genes are either modified or eliminated. In both approaches, the company uses NHEJ to complete the editing. When non-homologous end joining is used for repair, it leaves small insertions and deletions at the cut site, known as indels. In the cut and revise process, a single cut is made, and in the cut and remove process, two cuts are made. The approach can be used to delete either a small or large segment of DNA depending on the type of repair desired. In cut and replace, leveraging homology directed repair, the cell uses the provided template to construct reparative DNA, resulting in the replacement of a defective genetic sequence with the correct one.
The company believes that what sets them apart in the space is their genome editing platform that consists of four interrelated elements: directed editing to achieve the right repair, the ability to efficiently editing a wide range of mutations, the ability to tightly control the editing process, and the ability to reach the site of disease using multiple modalitie
