2022-04-27| Trials & Approvals

CRISPR Biotech Editas Obtains FDA Rare Pediatric Disease Label

by Fujie Tham
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Cambridge, MA-based Editas Medicine recently announced that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease designation to EDIT-301, an investigational, gene-edited medicine for treating beta thalassemia. Beta thalassemia is a blood disorder that reduces hemoglobin production, the protein responsible for carrying oxygen.

Following their first designation for treating sickle cell disease, this second FDA label will lead to initiation of Phase I/II study of EDIT-301 in transfusion-dependent beta thalassemia patients this year.

Related Article: Landmark CRISPR Trial Shows Promise Against Fighting Blindness, but Is It a Cure?


Editas’ EDIT-301


EDIT-301 is an experimental cell therapy medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). This therapeutic consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin genes (HBG1, HBG2) promoters, with potential to provide a one-time, persistent treatment benefit for people living with severe sickle cell disease and transfusion-dependent beta thalassemia.

Currently, Editas boasts 10 pipelines revolving around in-vivo gene-edited medicines and ex-vivo gene-edited cell medicines, with research and development efforts focused on diseases which there are currently few or no treatments available. Under its Ex Vivo Gene Edited Cell Medicines catalog, Editas is in partnership with Bristol-Myers Squibb to develop the next generation of ex-vivo gene-edited cell medicines for cancer immunotherapy. 

Editas Medicine was featured as one of GeneOnline’s 10 top gene therapy companies to look out for in 2021.

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