Editas Medicine Announces Submission of IND Application for EDIT-301 with the FDA
- Category: DNA RNA and Cells
- Published on Thursday, 10 December 2020 18:13
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EDIT-301 is in development as a best-in-class, durable medicine for people living with sickle cell disease
CAMBRIDGE, MA, USA I December 09, 2020 I Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced it submitted an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for the initiation of a Phase 1/2 clinical trial of EDIT-301, an experimental CRISPR/Cas12a gene editing medicine in development for the treatment of sickle cell disease. The Company previously received Rare Pediatric Disease designation from the FDA for EDIT-301.
“This IND submission is a key milestone for Editas as we continue to advance several ex vivo cell therapy medicines. This submission brings us one step closer to entering the clinic with our potentially best-in-class, transformative, and durable medicine for people living with sickle cell disease,” said Cynthia Collins, Chief Executive Officer, Editas Medicine. “This moment is very exciting for the Editas team. We know patients are counting on us, and we look forward to next steps for the clinical development of EDIT-301, including dosing sickle cell disease patients.”
Editas Medicine continues to prepare for a Phase 1/2 clinical trial evaluating EDIT-301 for the treatment of sickle cell disease. The Company has identified a lead principal investigator and engaged a Clinical Research Organization (CRO). Clinical trial materials are being manufactured by Editas Medicine.
About Sickle Cell Disease
Sickle cell disease is caused by a mutation in the beta-globin gene that leads to polymerization of the sickle hemoglobin protein (HbS). Fetal hemoglobin (HbF) protects against sickle cell disease by inhibiting HbS polymerization. Individuals with high levels of HbF are protected from sickle cell disease. EDIT-301 is an experimental, autologous cell therapy comprising CD34+ cells genetically modified using a Cas12a ribonucleoprotein (RNP) that targets the HBG1/2 promoter in the beta-globin gene to stimulate HbF production.
EDIT-301 is an experimental, autologous cell therapy medicine under investigation for the treatment of sickle cell disease. EDIT-301 is comprised of 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. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in fetal hemoglobin (HbF) production, which has the potential to provide a durable treatment benefit for people living with sickle cell disease.
About Editas Medicine
As a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a) genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit www.editasmedicine.com.
SOURCE: Editas Medicine