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.

Free white paper


Provide the cell therapy supply chain from bench to bed

The development of cell therapies is changing healthcare, bringing new hope to thousands of patients around the world. However, the vein-to-vein workflow for these therapies is not without challenges, many of which will increase with scaling to treat more patients. Download this free guide from Cytiva to learn more about the challenges and risks associated with the cryogenic supply chain for cellular therapies and how supply chain disruptions can best be mitigated.

by Cytiva Thematic

By clicking the Download Free White Paper button, you accept the terms and conditions and confirm that your data will be used as described in the Cytiva Thematic privacy policy.

By downloading this White Paper, you acknowledge that we may share your information with our White Paper partners / sponsors, who may contact you directly with information about their products and services.

Visit our privacy policy for more information about our services, how we may use, process and share your personal information, including information about your rights with respect to your personal information and how you may unsubscribe from future marketing communications. Our services are designed for corporate subscribers and you ensure that the email address sent is your corporate email address.

Previous articleHire AMD’s next generation SoC console and GPU team
Next articlePacific Basin and partners sign memorandum of understanding to develop zero-emission vessels