09:45 AM EDT, 04/02/2024 (MT Newswires) -- Rocket Pharmaceuticals ( RCKT ) said Tuesday that the European Medicines Agency has accepted its marketing authorization application for RP-L102, an investigational gene therapy for Fanconi Anemia, a rare genetic disorder.

The company said its application was based on positive data from a phase 1/2 clinical trial where RP-L102 "demonstrated sustained genetic correction, comprehensive phenotypic correction, and hematologic stabilization." The product candidate was also well tolerated, with no significant safety signals, the company added.

Rocket Pharmaceuticals ( RCKT ) said it remains on track to submit the drug candidate's biologics license application for Fanconi Anemia to the US Food and Drug Administration in the first half of the year.

Shares of the company retreated almost 4% in recent trading activity.

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