公开(公告)号：US20220204974A1

公开(公告)日：2022-06-30

申请号：US17576130

申请日：2022-01-14

申请人： University of Massachusetts

发明人： Terence Flotte ,  Christian Mueller ,  Phillip D. Zamore

IPC分类号： C12N15/113 ,  C12N15/67 ,  A61K48/00 ,  A61K35/12 ,  C12N7/00 ,  C12N15/86 ,  A61K31/713 ,  A61K38/57 ,  C07K14/81

摘要： The invention relates to isolated nucleic acids and rAAV-based compositions, methods and kits useful for treating genetic diseases (e.g., alpha-1 antitrypsin deficiency).

公开(公告)号：US20220170062A1

公开(公告)日：2022-06-02

申请号：US17600844

申请日：2020-04-02

申请人： KYOTO UNIVERSITY

发明人： Hirohide SAITO ,  Hirohisa OHNO ,  Sae AKAMINE

IPC分类号： C12P19/34 ,  C12N15/67 ,  C07H21/02 ,  C07H1/00

摘要： An RNA capping method including a step of reacting a compound (1) represented by the following general formula (1) with an RNA in the presence of a Vaccinia virus capping enzyme (Rb1 represents an oxo group, an alkoxy group, or a halogen atom; Rb2 is either absent or represents an alkyl group; Rb3 represents an amino group or a hydrogen atom; Rb4 is either absent or represents a hydrogen atom or an alkyl group; Rr1 represents a hydroxy group, an alkoxy group having 1 to 3 carbon atoms, an amino group, an azide group, —OR1C≡CH, or —R2R3; Rr2 represents a hydrogen atom, a hydroxy group, a halogen atom, an alkoxy group, an amino group, an azide group, —OR1C≡CH, or —R2R3; and A1 represents an oxygen atom or a sulfur atom).

公开(公告)号：US11345920B2

公开(公告)日：2022-05-31

申请号：US17542430

申请日：2021-12-05

申请人： CureVac AG

发明人： Andreas Thess ,  Thomas Schlake ,  Stefanie Grund

IPC分类号： C12N15/67 ,  A61K39/205 ,  A61K39/12 ,  A61K39/145 ,  C12N15/85 ,  C12N15/68 ,  A61K39/00 ,  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 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 as a medicament, as a vaccine or in gene therapy.

公开(公告)号：US11339397B2

公开(公告)日：2022-05-24

申请号：US16498570

申请日：2018-03-30

申请人： Nanjing Bestzyme Bio-Engineering Co., Ltd.

发明人： Aixi Bai ,  Feng Li ,  Furong Bian ,  Jidong Zhu ,  Yan Sun ,  Hong Xu

IPC分类号： C12N15/67 ,  C12N9/16 ,  C12N15/80

摘要： Disclosed herein is a method for expressing phytase in a filamentous fungus by using an optimized Escherichia coli phytase gene having a nucleotide sequence as shown in SEQ ID NO. 7 and a signal peptide having a nucleotide sequence as shown in SEQ ID NO. 12.

公开(公告)号：US20220073944A1

公开(公告)日：2022-03-10

申请号：US17404283

申请日：2021-08-17

申请人： TRANSLATE BIO, INC.

发明人： Frank DeRosa ,  Michael Heartlein ,  Daniel Crawford ,  Shrirang Karve

IPC分类号： C12N15/85 ,  C07K14/505 ,  C12N9/10 ,  C12N9/02 ,  C07K14/705 ,  A61K48/00 ,  A61K38/17 ,  A61K38/44 ,  A61K38/45 ,  A61K38/18 ,  A61K31/7115 ,  A61K31/7125 ,  C07H21/02 ,  C12N15/67 ,  C12P21/00

摘要： The present invention provides, among other things, multimeric coding nucleic acids that exhibit superior stability for in vivo and in vitro use. In some embodiments, a multimeric coding nucleic acid (MCNA) comprises two or more encoding polynucleotides linked via 3′ ends such that the multimeric coding nucleic acid compound comprises two or more 5′ ends.

公开(公告)号：US20220073933A1

公开(公告)日：2022-03-10

申请号：US17280369

申请日：2019-09-26

申请人： CASE WESTERN RESERVE UNIVIERSITY ,  WHITEHEAD INSTITUTE FOR BIOMEDICAL RESEARCH

发明人： Jeffery M. Coller ,  Thomas Sweet ,  Harvey Lodish

IPC分类号： C12N15/67 ,  C12N15/11 ,  C12N15/86 ,  A61K48/00 ,  A61K45/06 ,  A61K31/7088 ,  A61P43/00

摘要： A tRNA that hybridizes to a non-optimal codon can be used to increase expression in a mammalian cell of a gene product encoded by a gene containing the non-optimal codon or to treat a haploinsufficiency disorder in a subject having a haploinsufficient gene containing the non-optimal codon.

公开(公告)号：US11261472B2

公开(公告)日：2022-03-01

申请号：US16474805

申请日：2017-12-28

申请人： UNIVERSIDAD DE CHILE

发明人： Juan Alfonso Asenjo De Leuze ,  Barbara Anne Andrews Farrow ,  Vida Rodriguez Gallardo

IPC分类号： C12P21/02 ,  C07K14/46 ,  C12N15/62 ,  C12N15/67

摘要： A peptide sequence of a guide protein for the production of a peptide of interest; a peptide sequence that has a similarity of at least 90% to SEQ. ID. NO 1; a nucleotide sequence encoding the guide protein; an expression vector comprising the nucleotide sequence; a host cell that expresses a fusion protein comprising a peptide of interest; a method of production of a peptide of interest, comprising the steps of A) constructing an expression vector; B) inserting the expression vector into a host cell; C) expressing the fusion protein, culturing the host cell in a culture medium; D) recovering the accumulated fusion protein in the host cell; E) cleaving the fusion protein; F) purifying the peptide of interest.

公开(公告)号：US20220056473A1

公开(公告)日：2022-02-24

申请号：US17222910

申请日：2021-04-05

申请人： Marker Therapeutics, Inc.

发明人： Robert Z. Florkiewicz

IPC分类号： C12N15/85 ,  A61K45/06 ,  C07K14/705 ,  A61K39/00 ,  C12N15/67 ,  C07K14/50 ,  C07K14/82

摘要： The present disclosure relates to nucleic acid vaccine compositions and methods for preventing or treating pathological conditions, such as cancer or infectious disease. Further, the disclosure provides methods for more efficient production of antigens via mRNA containing one or more non-conventional start codons to promote multiplex initiation of translation in eukaryotic cells.

公开(公告)号：US20220054602A1

公开(公告)日：2022-02-24

申请号：US17465287

申请日：2021-09-02

申请人： Translate Bio, Inc.

发明人： Braydon Charles Guild ,  Michael Heartlein ,  Frank DeRosa

IPC分类号： A61K38/47 ,  A61K48/00 ,  C07K14/505 ,  C07K14/61 ,  C12N15/67 ,  C12N9/10 ,  C12N9/40 ,  A61K38/27 ,  A61K38/45 ,  A61K38/18

摘要： Compositions for modulating the expression of a protein in a target cell comprising at least one RNA molecule which comprises at least one modification 5 conferring stability to the RNA, as well as related methods, are disclosed.

公开(公告)号：US11236345B2

公开(公告)日：2022-02-01

申请号：US15557412

申请日：2016-03-09

申请人： CASE WESTERN RESERVE UNIVERSITY

发明人： Jeffery M. Coller ,  Kristian E. Baker

IPC分类号： C12N15/68 ,  C12N15/81 ,  C12N15/67 ,  C12P21/02 ,  C12N15/85

摘要： A synthetic nucleic acid which encodes a protein wherein at least one optimal or non-optimal codon in a wild type nucleic acid encoding the protein has been replaced respectively with one or more non-optimal codons or optimal codons encoding the same amino acid.