JP-2025516527-A5 -

Dates

Publication Date

20260508

Application Date

20230509

Description

All patents, patent applications, publications, test methods, product descriptions, literature, and other materials cited herein are incorporated herein by reference in their entirety for any purpose as if they were physically present herein. Finally, preferred embodiments of the present invention are described in separate sections. [Embodiment 1] A system for producing antibodies or antigen-binding fragments thereof within the body of a target, wherein the system is: a) A first component comprising a polynucleotide molecule, wherein the polynucleotide molecule comprises a sequence encoding the antibody or its antigen-binding fragment; and b) A second component comprising a gene editing molecule, or a polynucleotide molecule containing a sequence encoding the gene editing molecule. A system that includes this. [Embodiment 2] The system according to Embodiment 1, wherein by administering the first component and the second component to the subject, the sequence encoding the antibody or the antigen-binding fragment thereof is incorporated into the DNA of the subject's B cells and/or hematopoietic stem cells (HSCs), thereby causing the production of the antibody or the antigen-binding fragment within the subject's body. [Embodiment 3] The system according to Embodiment 1, wherein by administering the first component and the second component ex vivo to B cells and/or hematopoietic stem cells (HSCs), the sequence encoding the antibody or its antigen-binding fragment is incorporated into the DNA of the cells, thereby producing modified B cells or modified HSCs, and thereby, when the modified B cells or modified HSCs are administered to the subject, the production of the antibody or its antigen-binding fragment is induced in the subject's body. [Embodiment 4] The system according to any one of embodiments 1 to 3, wherein the antibody or its antigen-binding fragment binds to an antigen associated with a disease or disorder. [Embodiment 5] The system according to Embodiment 4, wherein the disease or disorder is an infectious disease, cancer, autoimmune disease, cardiovascular disease, musculoskeletal disease, or neurodegenerative disease. [Embodiment 6] The system according to Embodiment 5, wherein the infectious disease is a viral infection, a bacterial infection, a fungal infection, or a parasitic infection. [Embodiment 7] The system according to any one of embodiments 4 to 6, wherein the antigen is a viral antigen, a bacterial antigen, a fungal antigen, a parasitic antigen, or a tumor-associated antigen (TAA) antigen. [Embodiment 8] The system according to any one of embodiments 1 to 7, wherein the gene editing molecule is a Cas nuclease. [Embodiment 9] The system according to embodiment 8, wherein the Cas nuclease is Cas9 nuclease. [Embodiment 10] The system according to any one of embodiments 1 to 9, wherein the first component or the second component further comprises a guide RNA (gRNA) molecule or a sequence encoding the gRNA molecule. [Method 11] The system according to Embodiment 10, wherein the first component comprises the polynucleotide molecule comprising the sequence encoding the antibody or its antigen-binding fragment and the sequence encoding the gRNA. [Embodiment 12] The system according to Embodiment 10, wherein the first component comprises (i) a first polynucleotide molecule containing the sequence encoding the antibody or its antigen-binding fragment; and (ii) a second polynucleotide molecule containing the sequence encoding the gRNA. [Embodiment 13] The system according to Embodiment 10, wherein the first component comprises (i) a first polynucleotide molecule containing the sequence encoding the antibody or its antigen-binding fragment; and (ii) the gRNA. [Embodiment 14] The system according to embodiment 10, wherein the second component includes the gRNA molecule or the sequence encoding the gRNA molecule. [Embodiment 15] The system according to any one of embodiments 10 to 14, wherein the gRNA is complementary to the sequence of the IgH locus, the J chain locus, or the Igκ locus. [Embodiment 16] The system according to embodiment 15, wherein the gRNA is complementary to the sequence in the fourth exon of the J-chain locus. [Embodiment 17] The system according to embodiment 15, wherein the gRNA is complementary to the sequence in the first intron of the J-chain locus. [Embodiment 18] The system according to any one of embodiments 1 to 17, wherein the sequence encoding the antibody or its antigen-binding fragment includes a sequence encoding the variable region of the antibody's light chain and, optionally, the constant region of the light chain. [Embodiment 19] The system according to any one of embodiments 1 to 18, wherein the sequence encoding the antibody or its antigen-binding fragment includes a sequence encoding the heavy chain variable region of the antibody. [Embodiment 20] The system according to any one of embodiments 1 to 20, wherein the sequence encoding the antibody or its antige