FDA grants rare pediatric disease designation to cell therapy for sickle cell disease
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The FDA granted rare pediatric disease designation to EDIT-301, an investigational autologous cell therapy for the treatment of sickle cell disease, according to the agent’s manufacturer.
EDIT-301 (Editas Medicine) is designed to modify red blood cells to increase fetal hemoglobin production.
EDIT-301 consists of the patient’s CD34-positive cells genetically modified using a CRISPR/Cas12a technique that edits the HBG1/HBG2 promoter region in the beta-globin locus.
Elevated levels of fetal hemoglobin have been associated with fewer vaso-occlusive crises and fewer hospitalizations among patients with sickle cell disease.
“The Editas team has a bold vision to unlock the potential of CRISPR to design and develop game-changing medicines,” Cynthia Collins, CEO of Editas Medicine, said in a company-issued press release. “We are making tremendous progress toward 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.
“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,” she added.
The FDA defines a rare pediatric disease as a disease or condition that is serious or life-threatening and affects fewer than 200,000 children in the United States.
The designation makes Editas Medicine eligible for an FDA priority review voucher for EDIT-301 if the agency approves the investigational therapy’s biologics license application.
Editas expects to submit an investigational new drug application for EDIT-301 to the FDA by the end of this year, according to the release.
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