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US 12,351,834 B2Granted
Compositions and methods for the treatment of ornithine transcarbamylase deficiency
AssigneeArcturus Therapeutics, Inc.
InventorsPadmanabh Chivukula, Priya Prakash Karmali, Kiyoshi Tachikawa, Suezanne E. Parker, Marciano Rodriguez Sablad, Pattraranee Limphong, Yanjie Bao, Jerel Boyd Lee Vega
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Abstract
The present disclosure describes compositions and methods for treating ornithine transcarbamylase (OTC) deficiency. The compositions include a lipid formulation and messenger RNA (mRNA) encoding an OTC enzyme. The lipid formulations can comprise an ionizable cationic lipid in a lipid nanoparticle encapsulating the mRNA.
Claims37
A composition comprising:
- a. an mRNA encoding an enzyme comprising a sequence having at least 95% identity to a sequence of SEQ ID NO:
- 3 or SEQ ID NO:
- 4 and having ornithine transcarbamylase (OTC) activity;
- and
- b. a lipid formulation comprising an ionizable cationic lipid selected from
2 more elements
Dependencies are inferred from the claim text.