Editas up after unveiling novel gene editing to treat hemoglobinopathies

 

• Editas Medicine (NASDAQ:EDIT) joins the gene-editing bandwagon as the company showcases promising pre-clinical data and successful development of a large-scale manufacturing process for EDIT-301, the company’s gene-editing treatment for sickle cell disease and beta-thalassemia.
• The data announced over the weekend at the 62nd Annual Meeting and Exposition of the American Society of Hematology demonstrate high levels of editing in CD34+ cells from normal donors and sickle cell patients, leading to robust fetal hemoglobin induction. The company expects an IND filing for the treatment by the end of 2020, and shares surged +13.2%.
• Sickle cell disease, an inherited blood disorder impacting an estimated 100,000 people in the United States, leads to polymerization of the sickle hemoglobin protein, which is inhibited by fetal hemoglobin.
• "These findings are very encouraging and further support our novel approach to developing and manufacturing EDIT-301 as a best-in-class and durable medicine for the potential treatment of sickle cell disease and beta-thalassemia,” said Charles Albright, Ph.D., Executive Vice President, and Chief Scientific Officer, Editas Medicine.
• Additionally, the company announced that its large-scale manufacturing process for edited CD34+ cells from normal donors was consistent and robust.
• During the ASH meeting, CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals (NASDAQ:VRTX) also demonstrated promising data from Phase 1/2 clinical studies for CTX001, an investigational therapy for patients suffering from TDT (transfusion-dependent beta-thalassemia) and severe SCD (sickle cell disease)currently undergoing Phase 1/2 clinical trials.
• https://seekingalpha.com/news/3642287-editas-medicine-surges-13-after-unveiling-novel-approach-to-gene-editing-therapies-to-treat