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US 12,569,572 B2Granted
Messenger RNA therapy for the treatment of Friedreich's ataxia
AssigneeTranslate Bio, Inc.
InventorsMichael Heartlein, Frank DeRosa, Jonathan Cherry, Paula Lewis, Shrirang Karve, Zarna Patel, Darshan Parekh, Susan Wood, Chi-Sung Chiu, Caroline J. Woo
Life of this patent
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Abstract
The present invention provides methods and compositions of treating Friedreich's ataxia (FRDA) based on administering an mRNA encoding a frataxin protein.
Claims14
A pharmaceutical composition for treating Friedreich's ataxia (FRDA), comprising an mRNA encoding a human frataxin protein and wherein the mRNA is encapsulated within a liposome, wherein the mRNA comprises a polynucleotide sequence that is identical to SEQ ID NO:
- 1, SEQ ID NO:
- 2, SEQ ID NO. 3, or SEQ ID NO:
- 4.
Dependencies are inferred from the claim text.