The US Food and Drug Administration (FDA) Accept Designation of an orphan drug for EDIT-301, an experimental gene editing therapy for Editas Medicine to treat beta-thalassemia.
EDIT-301 is composed of CD34 + hematopoietic stem cells and progenitor cells obtained from the patient and is edited by the AsCas12a nuclease at the gamma globin gene (HBG1 and HBG2) promoters.
It has been analyzed to treat severe sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).
The company plans to begin Phase I / II clinical trials of EDIT-301 in transfusion-dependent beta-thalassemia patients who are expected to be on medication by the end of this year.
James Mullen, Chairman, President and CEO of Editas Medicine, said: “Beta thalassemia is a catastrophic disease that leads to severe anemia, organ failure and premature death.
“Preparations are underway to begin Phase I / II clinical trials of EDIT-301, a potentially transformative drug for people with beta-thalassemia, and administration to the first patients in this year’s clinical trials. I’m looking forward to it. “
Beta thalassemia is a common autosomal recessive disorder whose mutations reduce or inhibit beta-globin expression.
Inadequate production of beta globin causes ineffective production of red blood cells, compensatory extramedullary hematopoiesis, and chronic hemolytic anemia due to red blood cell destruction.
Previously, EDIT-301 was designated by the FDA as a rare childhood disease to treat beta-thalassemia and SCD.
It is currently being analyzed in a RUBY clinical trial in patients with severe sickle cell disease.
April 2019, Editas and BlueRock Therapeutics collaborated Discover, develop and manufacture new drugs in the fields of oncology, neurology, cardiology and immunology.
The range of cell and gene therapies related to pharmaceutical technology is supported by Cytiva..
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https://www.pharmaceutical-technology.com/news/fda-orphan-drug-editas/ FDA grants Editors beta thalassemia therapy an orphan drug designation