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Editas Medicine nabs rare pediatric disease tag for sickle cell candidate

August 24, 2020

Editas Medicine (EDIT +0.1%) announced FDA approval of Rare Pediatric Disease designation for EDIT-301, for the treatment of sickle cell disease and plans to file an IND application by the end of 2020.

“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.” said Cynthia Collins, CEO.

Rare Pediatric Disease Designation provides for the issuance of a rare pediatric disease priority review voucher following FDA approval. The voucher can be used for accelerated approval of a future application or it can be sold to a third party.

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