公开(公告)号：US20240218370A1

公开(公告)日：2024-07-04

申请号：US18191611

申请日：2023-03-28

Applicant: OSPEDALE SAN RAFFAELE S.R.L. ,  Fondazione Telethon

Inventor： Luigi Naldini ,  Brian Brown

IPC: C12N15/113 ,  C12N15/63 ,  C12N15/86

CPC classification number: C12N15/1137 ,  C12N15/635 ,  C12N15/86 ,  A61K48/00

Abstract: A gene vector comprising a miRNA sequence target.

公开(公告)号：US20220273712A1

公开(公告)日：2022-09-01

申请号：US17615766

申请日：2020-06-05

Applicant: THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY ,  OSPEDALE SAN RAFFAELE S.R.L. ,  FONDAZIONE TELETHON

Inventor： Rosa Bacchetta ,  Maria Grazia Roncarolo ,  Yohei Sato ,  Luigi Naldini ,  Laura Passerini

IPC: A61K35/17 ,  C12N5/0783

Abstract: Engineered Treg-like cells, CD4LVFOXP3 T cells, and their use in cellular therapy to promote immune tolerance are disclosed. In particular, CD4LVFOXP3 T cells are produced by transduction of CD4+ T cells with a lentiviral vector expressing FOXP3 under the control of a constitutive promoter. Transduced cells express FOXP3 at high and persistent levels and acquire immune suppressive characteristics resembling naturally occurring Treg cells.

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：US20190032049A1

公开(公告)日：2019-01-31

申请号：US15521294

申请日：2015-10-23

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

Inventor： Luigi Naldini ,  Angelo Leone Lombardo ,  Angelo Amabile ,  Alessandro Migliara

IPC: C12N15/113 ,  C07K14/47 ,  C12N9/10 ,  C12N9/22

Abstract: A product comprising two or more artificial transcription repressors (ATRs), or polynucleotides encoding therefor, selected from groups (a), (b), (c) or (d): (a) an ATR comprising a DNA-binding domain operably linked to a KRAB domain or homologue thereof; (b) an ATR comprising a DNA-binding domain operably linked to a DNMT3A, DNMT3B or DNMT1 domain or homologue thereof; (c) an ATR comprising a DNA-binding domain operably linked to a DNMT3L domain or homologue thereof; and (d) an ATR comprising a DNA-binding domain operably linked to a SETDB1 domain or homologue thereof, wherein at least two of the ATRs are selected from different groups (a), (b), (c) or (d).

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：US12152240B2

公开(公告)日：2024-11-26

申请号：US15521294

申请日：2015-10-23

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

Inventor： Luigi Naldini ,  Angelo Leone Lombardo ,  Angelo Amabile ,  Alessandro Migliara

IPC: C12N15/113 ,  A61K35/12 ,  A61K38/00 ,  A61K38/17 ,  A61K38/45 ,  A61K48/00 ,  C07K14/47 ,  C12N9/10 ,  C12N9/22

Abstract: A product comprising two or more artificial transcription repressors (ATRs), or polynucleotides encoding therefor, selected from groups (a), (b), (c) or (d): (a) an ATR comprising a DNA-binding domain operably linked to a KRAB domain or homologue thereof; (b) an ATR comprising a DNA-binding domain operably linked to a DNMT3A, DNMT3B or DNMT1 domain or homologue thereof; (c) an ATR comprising a DNA-binding domain operably linked to a DNMT3L domain or homologue thereof; and (d) an ATR comprising a DNA-binding domain operably linked to a SETDB1 domain or homologue thereof, wherein at least two of the ATRs are selected from different groups (a), (b), (c) or (d).

公开(公告)号：US11753643B2

公开(公告)日：2023-09-12

申请号：US16004394

申请日：2018-06-10

Applicant: OSPEDALE SAN RAFFAELE S.R.L. ,  Fondazione Telethon

Inventor： Luigi Naldini ,  Brian Brown

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

CPC classification number: C12N15/1137 ,  C12N15/635 ,  C12N15/86 ,  A61K48/00 ,  C12N2310/141 ,  C12N2740/15043 ,  C12N2740/15045 ,  C12N2760/20222 ,  C12N2810/6081

Abstract: A gene vector comprising a miRNA sequence target.

公开(公告)号：US20230174622A1

公开(公告)日：2023-06-08

申请号：US17998447

申请日：2021-05-14

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

Inventor： Luigi Naldini ,  Pietro Genovese ,  Valentina Vavassori

IPC: C07K14/71 ,  C12N15/86 ,  C07K16/28 ,  A61P37/06

CPC classification number: C07K14/71 ,  C12N15/86 ,  C07K16/2863 ,  A61P37/06 ,  C07K2319/03

Abstract: A polynucleotide comprising a nucleotide sequence encoding an epidermal growth factor receptor (EGFR) extracellular epitope operably linked to: (a) a NGFR or GMS SFR alpha signal peptide; (b) a EGFR or NGFR transmembrane domain; and/or (c) a NGFR or EGFR cytosplasmic tail.

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：US20190032059A1

公开(公告)日：2019-01-31

申请号：US16004394

申请日：2018-06-10

Applicant: OSPEDALE SAN RAFFAELE S.R.L. ,  Fondazione Telethon

Inventor： Luigi Naldini ,  Brian Brown

IPC: C12N15/113 ,  C12N15/63 ,  C12N15/86

Abstract: A gene vector comprising a miRNA sequence target.