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Publication Number
US-12083189-B2
Publication Date
2024-09-10
Expiration Date
Abstract
Tail-tail RNA conjugates translatable by eukaryotic ribosomes, and pharmaceutically acceptable compositions including the same are provided. Methods for preparing such molecules, using them for treating and preventing diseases are described. In addition, processes for producing desired polypeptides using the molecules are also described.
Core Innovation
The invention provides a tail-tail RNA conjugate comprising two RNA molecules. Each RNA molecule includes operably connected elements comprising a 5’ untranslated region, a coding region, a 3’ untranslated region, and a terminal 3’ nucleotide. One RNA molecule comprises the sequence of SEQ ID NO: 5, and the other RNA molecule comprises the sequence of SEQ ID NO: 6.
The two RNA molecules are connected to one another through the terminal 3’ nucleotide of each RNA molecule. The described conjugates are formed as 3’-to-3’ RNA conjugates in which terminal 3’ nucleotides covalently link the RNA molecules. The disclosed embodiments further support linkage optionally via chemically reactive moieties and/or linkers such as bifunctional, trifunctional, or tetrafunctional PEG/peptide/nucleic-acid linkers.
The disclosure further relates to related compositions and methods including preparing the conjugates by transcription and subsequent linkage of terminal 3’ reactive nucleotides, expressing encoded polypeptides in host cells, and administering therapeutically effective amounts to treat or prevent diseases. The document includes rationale for improved stability via protection from 3’ exonuclease degradation and improved or extended protein expression in cell-based examples.
Claims Coverage
The independent claim defines one core inventive feature: a tail-tail RNA conjugate using two defined RNA sequences and a 3’-to-3’ terminal nucleotide connection.
Tail-tail RNA conjugate with terminal 3’ nucleotide linkage between two defined RNAs
A tail-tail RNA conjugate comprising two RNA molecules, each having operably connected elements including a 5’ UTR, a coding region, a 3’ UTR, and a terminal 3’ nucleotide; one RNA molecule comprises the sequence of SEQ ID NO: 5 and the other RNA molecule comprises the sequence of SEQ ID NO: 6; each RNA molecule is connected to the other through the terminal 3’ nucleotide.
Claim coverage is limited to a tail-to-tail 3’-to-3’ RNA conjugate formed by connecting two RNA molecules through their respective terminal 3’ nucleotides, where the RNAs are specifically defined by SEQ ID NO: 5 and SEQ ID NO: 6.
Stated Advantages
Improved stability via protection from 3’ exonuclease degradation.
Improved or extended protein expression.
Documented Applications
Treating or preventing diseases by administering therapeutically effective amounts of the described compositions.
Host cell expression of polypeptides encoded by the conjugate RNAs.
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