公开(公告)号：US20230313207A1

公开(公告)日：2023-10-05

申请号：US18330356

申请日：2023-06-06

申请人： CureVac SE

发明人： Andreas THESS ,  Thomas SCHLAKE ,  Stefanie GRUND

IPC分类号： C12N15/67 ,  A61K39/12 ,  C12N15/85 ,  A61K39/145 ,  A61K39/205 ,  C12N15/68

CPC分类号： C12N15/67 ,  A61K39/12 ,  C12N15/85 ,  A61K39/145 ,  A61K39/205 ,  C12N15/68 ,  A61K48/00

摘要： The invention relates to an artificial nucleic acid molecule comprising an open reading frame and a 3′-UTR comprising at least one poly(A) sequence or a polyadenylation signal. The invention further relates to a vector comprising the artificial nucleic acid molecule comprising an open reading frame and a 3′-UTR comprising at least one poly(A) sequence or a polyadenylation signal, to a cell comprising the artificial nucleic acid molecule or the vector, to a pharmaceutical composition comprising the artificial nucleic acid molecule or the vector and to a kit comprising the artificial nucleic acid molecule, the vector and/or the pharmaceutical composition. The invention also relates to a method for increasing protein production from an artificial nucleic acid molecule and to the use of a 3′-UTR for a method for increasing protein production from an artificial nucleic acid molecule. Moreover, the invention concerns the use of the artificial nucleic acid molecule, the vector, the kit or the pharmaceutical composition as a medicament, as a vaccine or in gene therapy.

公开(公告)号：US20240294928A1

公开(公告)日：2024-09-05

申请号：US18415477

申请日：2024-01-17

申请人： CureVac SE

发明人： Stefanie GRUND ,  Thomas SCHLAKE

IPC分类号： C12N15/68 ,  A61K48/00 ,  C12N15/67 ,  C12N15/85

CPC分类号： C12N15/68 ,  A61K48/0066 ,  C12N15/67 ,  C12N15/85 ,  C12N2830/50 ,  C12N2840/105

摘要： The invention relates to an artificial nucleic acid molecule comprising at least one open reading frame and at least one 3′-untranslated region element (3′-UTR element) and/or at least one 5′-untranslated region element (5′-UTR element), wherein the at least one 3′-UTR element and/or the at least one 5′-UTR element prolongs and/or increases protein production from said artificial nucleic acid molecule and wherein the at least one 3′-UTR element and/or the at least one 5′-UTR element is derived from a stable mRNA. The invention further relates to the use of such an artificial nucleic acid molecule in gene therapy and/or genetic vaccination. Furthermore, methods for identifying a 3′-UTR element and/or a 5′-UTR derived from a stable mRNA element are disclosed.

公开(公告)号：US20240101608A1

公开(公告)日：2024-03-28

申请号：US18528065

申请日：2023-12-04

申请人： CureVac SE

发明人： Patrick BAUMHOF ,  Susanne RAUCH ,  Aleksandra KOWALCZYK ,  Johannes LUTZ ,  Edith JASNY ,  Benjamin PETSCH ,  Andreas THESS ,  Thomas SCHLAKE ,  Mariola FOTIN-MLECZEK ,  Regina HEIDENREICH ,  Sandra LAZZARO ,  Fatma FUNKNER ,  Wolfgang GROSSE

IPC分类号： C07K14/005 ,  A61K39/12 ,  A61K39/145 ,  A61P31/16 ,  C07K14/205 ,  C12N15/00

CPC分类号： C07K14/005 ,  A61K39/12 ,  A61K39/145 ,  A61P31/16 ,  C07K14/205 ,  C12N15/00 ,  A61K2039/53

摘要： The present invention provides optimized nucleic acid molecules, methods for optimization of nucleic acid molecules and uses of optimized nucleic acid molecules. A modular design principle is provided that is suitable to generate a nucleic acid, particularly mRNA, which is tailored for a respective application. The nucleic acid molecules of the present invention can be obtained by the versatile combination of multiple modules on nucleic acid level. Such nucleic acid, e.g. mRNA, can be tailored by combining one or more modules, comprising (i) a nucleic acid moiety encoding a polypeptide of interest (e.g. a protein potentially producing a therapeutic outcome) and (ii) at least one further coding or non-coding nucleic acid moiety, e.g. selected among nucleic acid moieties encoding a polypeptide element, such as a secretory signal peptide (SSP), a multimerization element (dimerization, trimerization, tetramerization and oligomerization), a virus like particle (VLP) forming element, a transmembrane element, a dendritic cell targeting element, an immunological adjuvant element, an element promoting antigen presentation; a 2A peptide; a peptide linker element, elements that extend protein half-life, and/or any other polypeptide or protein. Non-coding nucleic acid moieties may be selected e.g. from the group comprising 3′-UTR, 5′-UTR, IRES element, miRNA moiety, histone stem loop, poly(C) sequence, polyadenylation signal, polyA-sequence. The optimized nucleic acid molecule can further be characterized by the presence of at least one modified nucleoside. The versatility of the present invention allows for rational design of a large variety of different nucleic acid molecules with desired properties.

公开(公告)号：US20230399649A1

公开(公告)日：2023-12-14

申请号：US18320187

申请日：2023-05-18

申请人： CureVac SE

发明人： Stefanie GRUND ,  Thomas SCHLAKE

IPC分类号： C12N15/68 ,  C12N15/85 ,  C12N15/67

CPC分类号： C12N15/68 ,  C12N15/85 ,  C12N15/67 ,  C12N2800/22 ,  C12N2830/00 ,  C12N2800/107 ,  C12N2840/203 ,  A61K39/00

摘要： The invention relates to an artificial nucleic acid molecule comprising at least one open reading frame and at least one 3′-untranslated region element (3′-UTR element) comprising a nucleic acid sequence which is derived from the 3′-UTR of a FIG4 gene or from a variant of the 3′-UTR of a FIG4 gene. The invention further relates to the use of such an artificial nucleic acid molecule in gene therapy and/or genetic vaccination. Furthermore, the invention relates to the use of a 3′-UTR element comprising a nucleic acid sequence which is derived from the 3′-UTR of a FIG4 gene or from a variant of the 3′-UTR of a FIG4 gene for the stabilization and/or prolongation of protein expression from a nucleic acid sequence comprising such 3′-UTR element.

公开(公告)号：US20230332163A1

公开(公告)日：2023-10-19

申请号：US18346686

申请日：2023-07-03

申请人： CureVac SE

发明人： Frédéric CHEVESSIER-TÜNNESEN ,  Marion POENISCH ,  Thomas SCHLAKE

IPC分类号： C12N15/67 ,  A61K31/7088 ,  A61K48/00 ,  C12N9/22 ,  C12N15/113

CPC分类号： C12N15/67 ,  A61K31/7088 ,  A61K48/0066 ,  C12N9/22 ,  C12N15/113 ,  C12N2310/20 ,  C12N2310/531 ,  C12N15/62

摘要： The present invention relates to the field of biomedicine, and in particular to the field of therapeutic nucleic acids. The present invention provides artificial nucleic acids, in particular RNAs, encoding CRISPR-associated proteins. A (pharmaceutical) composition and kit-of-parts comprising the same are also provided. Furthermore, the present invention relates to the artificial nucleic acid, (pharmaceutical) composition, or kit-of-parts for use in medicine, and in particular in the treatment and/or prophylaxis of diseases amenable to treatment with CRISPR-associated proteins.