The US Food and Drug Administration (FDA) has provided Labeling of orphan drugs in the experimental gene editing therapy of Editas Medicine, EDIT-301, for the treatment of beta thalassemia.

EDIT-301 includes CD34 + hematopoietic stem and progenitor cells derived from patients and edited in the promoters of the gamma globin gene (HBG1 and HBG2) by AsCas12a nuclease.

It is being analyzed for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

The company intends to launch a phase I / II clinical trial of EDIT-301 in patients with transfusion-dependent beta thalassemia with dosing expected by the end of this year.

James Mullen, chairman, president and CEO of Editas Medicine, said: “Beta thalassemia is a devastating disease that leads to severe anemia, organ failure and premature death.

“Preparations for the launch of the Phase I / II clinical trial of EDIT-301, a potentially transforming drug for people living with beta thalassemia, are underway and we look forward to dosing the first patient in this year’s clinical trial.

Beta thalassemia is a common autosomal recessive condition and its mutations reduce or inhibit the expression of beta globin.

Inadequate production of beta globin causes inefficient production of red blood cells, compensatory extramedullary hematopoiesis and chronic hemolytic anemia due to destruction of red blood cells.

Earlier, EDIT-301 received a designation for rare pediatric diseases from the FDA for the treatment of beta thalassemia and SCD.

It is currently being analyzed in the RUBY clinical trial, which includes patients with severe sickle cell anemia.

In April 2019, Editas and BlueRock Therapeutics collaborated to discover, develop and manufacture new drugs in oncology, neurology, cardiology and immunology.

The coverage for cell and gene therapy in pharmaceutical technology is maintained by Cytiva.

Editorial content is produced independently and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.

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