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EDIT-301, Experimental Cell Therapy for SCD, Earns Rare Pediatric Disease Designation

Joana Carvalho, PhD avatar

by Joana Carvalho, PhD |

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The U.S. Food and Drug Administration (FDA) has given rare pediatric disease designation to EDIT-301, a potential cell therapy for the treatment of sickle cell disease (SCD).

This designation is given to therapies that aim to treat serious or life-threatening disorders that affect fewer than 200,000 people in the U.S., primarily those younger than 18. Companies whose medications are approved after receiving this designation may be eligible to receive a priority review voucher to be used on a different product.

Editas Medicine, the company developing EDIT-301, also stated that it is planning to submit an investigational new drug (IND) application to the FDA before the end of this year, so that EDIT-301 can enter clinical testing.

“The Editas team has a bold vision to unlock the potential of CRISPR to design and develop game-changing medicines. We are making tremendous progress towards this vision with the continued development of EDIT-301, a potentially transformative medicine for the treatment of sickle cell disease, and we are pleased to receive Rare Pediatric Disease designation from the FDA for this program,” Cynthia Collins, CEO of Editas Medicine, said in a press release.

“We know patients are counting on us, and this designation is a significant milestone for the program that highlights the serious, life-threatening manifestations of sickle cell disease,” Collins said.

EDIT-301 is an investigational cell therapy made of red blood cell progenitors containing the CD34 protein — a marker of blood cell precursors — on their surface.

After being isolated from patients, these blood cell precursors are genetically modified — using the CRISPR/Cas12a (also known as Cpf1) gene-editing tool — to change a portion of the DNA sequence of the HBB gene, which is mutated in people with SCD.

Such alteration in the HBB gene ensures that red blood cells generated from these progenitor cells are able to produce high levels of fetal hemoglobin — a version of hemoglobin found in newborns that is more efficient at transporting oxygen than its adult counterpart. Also, fetal hemoglobin is able to inhibit the clumping of sickled hemoglobin, which blocks blood flow and causes pain crises in people with SCD.

By increasing the production of fetal hemoglobin, EDIT-301 is intended to provide long-term benefits to people with SCD, according to Editas.

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