Editas Medicine EDIT

Editas Medicine Inc is a gene editing company focused on developing genomic medicines using CRISPR technology to treat serious diseases by editing DNA in vivo and ex vivo to upregulate normal gene expression and protein function.

The company generates revenue through collaboration agreements licensing fees milestone payments and royalties from partnerships with pharmaceutical companies such as Bristol Myers Squibb Vertex Pharmaceuticals and Immatics as well as upfront payments and annual license fees from agreements like the Vertex License Agreement and the DRI Agreement.

The company operates through the following segments: Cardiovascular Hemoglobinopathies Other Cells/Tissues.

• The Cardiovascular segment focuses on developing EDIT-401 an in vivo gene editing medicine designed to significantly reduce LDL cholesterol by upregulating the LDL receptor gene using Cas9 and dual guide RNAs to treat hyperlipidemia and heterozygous familial hypercholesterolemia with preclinical data showing approximately 90% LDL-C reduction in non-human primates.
• The Hemoglobinopathies segment develops in vivo gene editing therapies targeting hematopoietic stem cells to treat sickle cell disease and transfusion-dependent beta thalassemia by using AsCas12a to edit the HBG1/2 promoter site and increase fetal hemoglobin production independent of erythropoietic stress.
• The Other Cells/Tissues segment aims to develop in vivo gene editing medicines for additional cell types using the company's proprietary lipid nanoparticle targeting platform to substitute targeting ligands for delivery to extrahepatic tissues with proof-of-concept demonstrated in humanized mice for two non-hematopoietic stem cell types.

Editas Medicine Inc holds a differentiated position in the gene editing industry due to its proprietary platform that includes natural and engineered variants of Cas9 and Cas12a enabling targeting of over 95% of the human genome and its functional upregulation strategy that edits non-coding regulatory sequences to increase normal gene expression without altering the protein sequence distinguishing it from gene knockdown and correction approaches.

The company serves patients with serious genetic diseases including those with hyperlipidemia hemoglobinopathies and other serious conditions as well as collaborates with pharmaceutical and biotechnology companies such as Bristol Myers Squibb Vertex Pharmaceuticals and Immatics to develop gene edited therapies.