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Editas (EDIT) Submits Application to Start Study on EDIT-301

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Zacks Equity Research
·3 min read
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Leading genome editing company Editas Medicine, Inc. EDIT announced that it submitted an Investigational New Drug (IND) application to the FDA for the initiation of a phase I/II study of EDIT-301.

EDIT-301 is an experimental, autologous cell therapy medicine under investigation for the treatment of sickle cell disease. It comprises sickle patient CD34+ cells genetically modified using a highly specific and efficient CRISPR/Cas12a (also known as Cpf1) ribonucleoprotein (RNP) to edit the HBG1/2 promoter region in the beta-globin locus.

The FDA previously granted a Rare Pediatric Disease designation to EDIT-301.

Sickle cell disease is caused by a mutation in the beta-globin gene that causes polymerization of the sickle hemoglobin protein (HbS).

Meanwhile, the company identified a lead principal investigator and engaged a Clinical Research Organization (CRO).

Editas has no approved product in its portfolio at the moment. Therefore, its pipeline development remains in key focus.

The stock has rallied 70.9% in the year so far compared with the industry’s growth of 5.9%.

Particularly, in the last three days, shares of the company appreciated significantly after the company announced preclinical data on EDIT-301 and the successful development of its large-scale manufacturing process on Dec 5. The data was released at the 62nd Annual Meeting and Exposition of the American Society of Hematology (ASH).

The outcomes showed that high levels of editing in CD34+ cells from normal donors and sickle cell patients were achieved with CRISPR/Cas12a at the HBG1 and HBG2 promoters, leading to robust fetal hemoglobin (HbF) induction in their erythroid progeny in a pan-cellular fashion. Red blood cells derived from edited sickle cell patient CD34+ cells showed a remarkable correction of sickle cell disease phenotypes including a reduction in sickling and improved rheological properties when deoxygenated.

Earlier, another gene editing company CRISPR Therapeutics AG CRSP and partner Vertex Pharmaceuticals VRTX also announced new positive results from an evaluation conducted on a total of 10 patients treated with the investigational CRISPR/Cas9-based gene-editing therapy CTX001. Findings established a consistent and sustained response to treatment in patients with transfusion-dependent beta thalassemia (TDT) as well as those with sickle cell disease. Treatment with CTX001 showed that all seven patients with TDT remained transfusion independent until the last follow-up while all three patients with sickle cell disease were free of vaso-occlusive crises through the last follow-up.

Editas currently carries a Zacks Rank #3 (Hold). A better-ranked stock in the biotech space is Halozyme Therapeutics, Inc. HALO, which carries a Zacks Rank #2 (Buy), presently. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Halozyme’s earnings estimates have grown 21 cents for 2020 in the past 60 days.

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