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US 2026/0226462 A1Published application
Synthetic mrna lacking a polya tail or having a short adenine homopolymer and methods of use and production thereof
AssigneeElixirgen Therapeutics, Inc.
InventorMinoru S.H. KO
Life of this patent
Estimating the term…
Abstract
The present disclosure relates to mRNA molecules that include a 3′-untranslated region (3′-UTR) of a positive-sense, single-stranded RNA (+ssRNA) of a virus, a coding sequence for a protein that is heterologous to the virus, and a 5′-untranslated region (5′-UTR). In particular, the present disclosure relates to mRNA encoding a protein of interest but lacking a poly(A) tail. Furthermore, the present disclosure relates to such mRNAs that permit the addition of an adenine homopolymer at their 3′ ends.
Claims22
1-48. (canceled)
An RNA molecule comprising from 5′ to 3′:
- a 5′-untranslated region (5′-UTR), at least one coding sequence (CDS) for at least one protein, and a 3′-untranslated region (3′-UTR), wherein
- the 3′-UTR comprises a virus 3′-UTR or fragment thereof, wherein the fragment is at least 40 nucleotides in length,
- the CDS encodes a full-length human dystrophin or a full-length human collagen type VII alpha-1 (VII) chain (COL7A1) protein, and replaces at least a portion of an open reading frame of the virus, and
- the virus is a positive-sense, single-stranded RNA (+ssRNA) virus.
1 more element
An RNA molecule comprising from 5′ to 3′:
- a 5′-untranslated region (5′-UTR), at least one coding sequence (CDS) for at least one protein, and a 3′-untranslated region (3′-UTR), wherein
- the 5′-UTR is a 5′-UTR of a Dengue virus or fragment thereof, and the 3′-UTR is a 3′-UTR of the Dengue virus or fragment thereof, wherein the fragment is at least 40 nucleotides in length and
- the CDS is heterologous to the Dengue virus and replaces an open reading frame of the Dengue virus such that a portion of the open reading frame that interacts with a complementary sequence of the 3′-UTR to form a circular conformation remains in the RNA molecule.
61-97. (canceled)
Dependencies are inferred from the claim text.