公开(公告)号：US20200338131A1

公开(公告)日：2020-10-29

申请号：US16827708

申请日：2020-03-24

Applicant: Ospedale San Raffaele S.R.L. ,  Fondazione Telethon

Inventor： Luigi Naldini ,  Bernhard Rudolf Gentner ,  Erika Zonari ,  Francesco Boccalatte

IPC: A61K35/28 ,  C12N5/0789 ,  C12N15/86 ,  A61K48/00

Abstract: A method of preparing a therapeutic cell population for clinical use from a starting population of cells comprising haematopoietic stem cells, said method comprising separating a population of cells that substantially do not express CD38 but which express CD34 from the starting population of cells, and transducing the separated cell population with a vector to obtain the therapeutic cell population.

公开(公告)号：US20200078408A1

公开(公告)日：2020-03-12

申请号：US16604484

申请日：2018-04-20

Applicant: Ospedale San Raffaele S.r.l. ,  Fondazione Telethon

Inventor： Luigi Naldini ,  Giulia Escobar ,  Bernhard Rudolf Gentner ,  Adele Mucci

IPC: A61K35/28 ,  C12N5/0789 ,  C12N5/0786 ,  A61K35/15 ,  A61K38/21 ,  A61K35/17 ,  A61K39/395 ,  A61P35/00 ,  A61P35/02 ,  A61K31/405 ,  C07K16/28 ,  C12N15/86 ,  C12N7/00 ,  A61K48/00

Abstract: A hematopoietic stem cell (HSC), a hematopoietic progenitor cell (HPC) a myeloid/monocyte-committed progenitor cell, a macrophage or a monocyte comprising a vector wherein the vector comprises at least one mir-130a and/or mir-126 target sequence operably linked to a nucleotide sequence encoding interferon-alpha (IFNα) for use in treating or preventing a cancer in a patient, wherein the HSC, the HPC, the myeloid/monocyte-committed progenitor cell, the macrophage or the monocyte is used in combination with an immune checkpoint inhibitor and/or a tumor associated antigen (TAA)-specific T-cell.

公开(公告)号：US09951328B2

公开(公告)日：2018-04-24

申请号：US15693112

申请日：2017-08-31

Applicant: Fondazione Telethon ,  Ospedale San Raffaele S.r.l.

Inventor： Alessandra Biffi ,  Bernhard Rudolf Gentner ,  Luigi Naldini

IPC: C12N15/63 ,  C12N15/113 ,  C12N15/86 ,  C12N15/11 ,  A61K48/00

CPC classification number: C12N15/111 ,  A61K48/00 ,  C12N2310/141 ,  C12N2310/51 ,  C12N2320/32 ,  C12N2330/51 ,  C12N2799/027

Abstract: A gene vector for use in gene therapy comprising at least one miRNA sequence target operably linked to a nucleotide sequence having a corresponding miRNA in a hematopoietic progenitor cell (HSPC) or hematopoietic stem cell (HSC) which prevents or reduces expression of the nucleotide sequence in a HSPC or HSC but not in a differentiated cell.

公开(公告)号：US11964027B2

公开(公告)日：2024-04-23

申请号：US16606633

申请日：2018-04-20

Applicant: Ospedale San Raffaele S.R.L ,  Fondazione Telethon

Inventor： Anna Christina Kajaste-Rudnitski ,  Carolina Petrillo ,  Bernhard Rudolf Gentner ,  Luigi Naldini ,  Pietro Genovese ,  Giulia Schiroli

IPC: A61K48/00 ,  C07K7/64 ,  C12N5/0789 ,  C12N15/69 ,  C12N15/86

CPC classification number: A61K48/0008 ,  C07K7/645 ,  C12N5/0647 ,  C12N15/69 ,  C12N15/86 ,  C12N2740/15043

Abstract: Use of cyclosporin H (CsH) or a derivative thereof for increasing the efficiency of transduction of an isolated population of cells by a viral vector and/or increasing the efficiency of gene editing of an isolated population of cells when transduced by a viral vector.

公开(公告)号：US11407996B2

公开(公告)日：2022-08-09

申请号：US16384571

申请日：2019-04-15

Applicant: Ospedale San Raffaele S.r.l. ,  Fondazione Telethon

Inventor： Alessandra Biffi ,  Bernhard Rudolf Gentner ,  Luigi Naldini

IPC: C12N15/11 ,  A61K48/00

Abstract: A gene vector for use in gene therapy comprising at least one miRNA sequence target operably linked to a nucleotide sequence having a corresponding miRNA in a hematopoietic progenitor cell (HSPC) or hematopoietic stem cell (HSC) which prevents or reduces expression of the nucleotide sequence in a HSPC or HSC but not in a differentiated cell.

公开(公告)号：US20220333103A1

公开(公告)日：2022-10-20

申请号：US17855135

申请日：2022-06-30

Applicant: Fondazione Telethon ,  Ospedale San Raffaele S.r.l.

Inventor： Alessandra Biffi ,  Bernhard Rudolf Gentner ,  Luigi Naldini

IPC: C12N15/11

Abstract: A gene vector for use in gene therapy comprising at least one miRNA sequence target operably linked to a nucleotide sequence having a corresponding miRNA in a hematopoietic progenitor cell (HSPC) or hematopoietic stem cell (HSC) which prevents or reduces expression of the nucleotide sequence in a HSPC or HSC but not in a differentiated cell.

公开(公告)号：US20180044670A1

公开(公告)日：2018-02-15

申请号：US15693112

申请日：2017-08-31

Applicant: Ospedale San Raffaele S.R.L.

Inventor： Alessandra Biffi ,  Bernhard Rudolf Gentner ,  Luigi Naldini

IPC: C12N15/11 ,  A61K48/00

CPC classification number: C12N15/111 ,  A61K48/00 ,  C12N2310/141 ,  C12N2310/51 ,  C12N2320/32 ,  C12N2330/51 ,  C12N2799/027

Abstract: A gene vector for use in gene therapy comprising at least one miRNA sequence target operably linked to a nucleotide sequence having a corresponding miRNA in a hematopoietic progenitor cell (HSPC) or hematopoietic stem cell (HSC) which prevents or reduces expression of the nucleotide sequence in a HSPC or HSC but not in a differentiated cell.

公开(公告)号：US20160256492A1

公开(公告)日：2016-09-08

申请号：US15031169

申请日：2014-10-24

Applicant: OSPEDALE SAN RAFFAELE S.R.L. ,  FONDAZIONE TELETHON

Inventor： Luigi Naldini ,  Bernhard Rudolf Gentner ,  Erika Zonari ,  Francesco Boccalatte

IPC: A61K35/28 ,  C12N15/86 ,  C12N7/00 ,  C12N5/0789

CPC classification number: A61K35/28 ,  A61K48/0091 ,  C12N5/0647 ,  C12N7/00 ,  C12N15/86 ,  C12N2510/00 ,  C12N2740/15043

Abstract: A method of preparing a therapeutic cell population for clinical use from a starting population of cells comprising haematopoietic stem cells, said method comprising separating a population of cells that substantially do not express CD38 but which express CD34 from the starting population of cells, and transducing the separated cell population with a vector to obtain the therapeutic cell population.

Abstract translation: 一种从包含造血干细胞的起始原始细胞群制备用于临床使用的治疗细胞群的方法，所述方法包括分离基本上不表达CD38但从起始细胞群体表达CD34的细胞群，并转导 用载体分离细胞群以获得治疗细胞群体。