Editas Medicine, Inc. is a clinical-stage genome editing company. The Company is focused on developing potentially transformative genomic medicines to treat a broad range of serious diseases. It has developed a proprietary gene editing platform based on CRISPR technology. CRISPR uses a protein- ribonucleic acid (RNA) complex composed of an enzyme, including either CRISPR associated protein 9 (Cas9) or Cas12a (CRISPR from Prevotella and Francisella 1, also known as Cpf1), bound to a guide ribonucleic acid (RNA) molecule designed to recognize a particular deoxyribonucleic acid (DNA) sequence. It is engaged in the development of vivo administered gene editing medicines, in which the medicine is injected or infused into the patient to edit the cells inside their body. Its lead program, reni-cel, is an experimental ex vivo gene-edited medicine to treat sickle cell disease (SCD), a severe inherited blood disease that causes premature death, and transfusion-dependent beta thalassemia (TDT).
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