C12Y306/03044

Immune modulator for immunotherapy and vaccine formulation

A recombinant Flagrp170 protein and pharmaceutical compositions comprising a Flagrp170 protein and related molecules encoding same, and cells presenting such a protein are provided. The Flagrp170 protein comprises an NF-.sub.KB-activating domain of Flagellin and an ATP-binding domain truncated Grp170. The pharmaceutical compositions of the invention can be used for the treatment or prevention of cancer or infectious disease.

Stable Gene Transfer to Proliferating Cells
20170216456 · 2017-08-03 ·

Provided herein are methods for facilitating or inducing stable transgene integration and expression in a proliferating cell, comprising administering to the cell (i) a recombinant AAV (rAAV) vector comprising the transgene flanked by transposon-derived inverted terminal repeat sequences, which sequences are in turn flanked by AAV-derived inverted terminal repeat regions, and (ii) a source of a transposase that recognises said transposon-derived inverted terminal repeat sequences and directs the genomic integration of the transgene into the genome of the proliferating cell. Also provided are methods and transgene delivery systems for the treatment or prevention of diseases affecting, associated with or characterised by proliferating cells, including paediatric liver diseases, bone marrow diseases and cancer.

Stable gene transfer to proliferating cells

Provided herein are methods for facilitating or inducing stable transgene integration and expression in a proliferating cell, comprising administering to the cell (i) a recombinant AAV (rAAV) vector comprising the transgene flanked by transposon-derived inverted terminal repeat sequences, which sequences are in turn flanked by AAV-derived inverted terminal repeat regions, and (ii) a source of a transposase that recognises said transposon-derived inverted terminal repeat sequences and directs the genomic integration of the transgene into the genome of the proliferating cell. Also provide are methods and transgene delivery systems for the treatment or prevention of diseases affecting, associated with or characterised by proliferating cells.

A NOVEL THERAPY FOR ERYTHROPOIETIC PROTOPORPHYRIA (EPP) AND X-LINKED PROTOPORPHYRIA (XLP)
20220249473 · 2022-08-11 ·

Disclosed are novel compositions and methods of using the same for the treatment of Erythropoiefic protoporphyria (EPP) and X-linked protoporphyria (XLP). Disclosed are methods and compositions related to treating Erythropoietic protoporphyria and X-linked protoporphyria. Also described are therapeutic agents that can inhibit ABCG2. For example, provided herein are therapeutic agents defined by Formula I.

STABLE GENE TRANSFER TO PROLIFERATING CELLS
20200016278 · 2020-01-16 ·

Provided herein are methods for facilitating or inducing stable transgene integration and expression in a proliferating cell, comprising administering to the cell (i) a recombinant AAV (rAAV) vector comprising the transgene flanked by transposon-derived inverted terminal repeat sequences, which sequences are in turn flanked by AAV-derived inverted terminal repeat regions, and (ii) a source of a transposase that recognises said transposon-derived inverted terminal repeat sequences and directs the genomic integration of the transgene into the genome of the proliferating cell. Also provide are methods and transgene delivery systems for the treatment or prevention of diseases affecting, associated with or characterised by proliferating cells.

Oligopeptidic compounds and uses thereof

Disclosed is A method of treatment of a disorder or condition where it is desirable to inhibit the growth of cells, or a method of treatment which involves cytostatic therapy by administering an oligopeptidic compound to a subject in need thereof. The oligopeptidic compound is capable of interacting with proliferating cell nuclear antigen (PCNA) The compound comprises a PCNA interacting motif which is: which is: [K/R]-[F/Y/W]-[L/I/V/A/M]-[L/I/V/A/M]-[K/R] (SEQ ID NO. 28). The oligopeptidic compound has 9-70 subunits and at least one signal sequence. The signal sequence is a nuclear localization signal sequence and/or a cell penetrating signal sequence. In the compound a PCNA interacting motif is N-terminal to a signal sequence.

IMMUNE MODULATOR FOR IMMUNOTHERAPY AND VACCINE FORMULATION

A recombinant Flagrp170 protein and pharmaceutical compositions comprising a Flagrp170 protein and related molecules encoding same, and cells presenting such a protein are provided. The Flagrp170 protein comprises an NF-?B-activating domain of Flagellin and an ATP-binding domain truncated Grp170. The pharmaceutical compositions of the invention can be used for the treatment or prevention of cancer or infectious disease.

OLIGOPEPTIDIC COMPOUNDS AND USES THEREOF
20170246248 · 2017-08-31 ·

Disclosed is A method of treatment of a disorder or condition where it is desirable to inhibit the growth of cells, or a method of treatment which involves cytostatic therapy by administering an oligopeptidic compound to a subject in need thereof. The oligopeptidic compound is capable of interacting with proliferating cell nuclear antigen (PCNA) The compound comprises a PCNA interacting motif which is: which is: [K/R]-[F/Y/W]-[L/I/V/A/M]-[L/I/V/A/M]-[K/R] (SEQ ID NO. 28).

The oligopeptidic compound has 9-70 subunits and at least one signal sequence. The signal sequence is a nuclear localization signal sequence and/or a cell penetrating signal sequence. In the compound a PCNA interacting motif is N-terminal to a signal sequence.

Oligopeptidic compounds and uses thereof

Disclosed is an oligopeptidic compound capable of interacting with proliferating cell nuclear antigen (PCNA), wherein the compound comprises a PCNA interacting motif which is: TABLE-US-00001 (SEQIDNO.28) [K/R]-[F/Y/W]-[L/I/V/A/M]-[L/I/V/A/M]-[K/R];
wherein the oligopeptidic compound has 9-70 subunits and comprises at least one signal sequence, wherein the signal sequence is a nuclear localization signal sequence and/or a cell penetrating signal sequence and wherein in said compound a PCNA interacting motif is N-terminal to a signal sequence. Also disclosed are methods of treatment of a disorder or condition where it is desirable to inhibit the growth of cells, or a method of treatment which involves cytostatic therapy, or a method of treatment of inflammation said method comprising administering the oligopeptidic compound to a subject in need thereof.

Therapy for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP)

Disclosed are novel compositions and methods of using the same for the treatment of Erythropoiefic protoporphyria (EPP) and X-linked protoporphyria (XLP). Disclosed are methods and compositions related to treating Erythropoietic protoporphyria and X-linked protoporphyria. Also described are therapeutic agents that can inhibit ABCG2. For example, provided herein are therapeutic agents defined by Formula I.