公开(公告)号：US20240269169A1

公开(公告)日：2024-08-15

申请号：US18401135

申请日：2023-12-29

Applicant: The Children's Medical Center Corporation ,  Ospedale San Raffaele S.r.l. ,  Politecnico di Milano ,  Fondazione Telethon ETS ,  Dana-Farber Cancer Institute, Inc.

Inventor： Alessandra BIFFI ,  Marco PEVIANI ,  Davide MOSCATELLI ,  Alessia CAPOTONDO ,  Rita MILAZZO ,  Umberto CAPASSO PALMIERO

IPC: A61K35/12 ,  A01K67/0271 ,  A61K9/00 ,  A61K45/06 ,  A61P25/28 ,  C12N15/86

CPC classification number: A61K35/12 ,  A61P25/28 ,  C12N15/86 ,  A01K67/0271 ,  A01K2227/10 ,  A61K9/0019 ,  A61K2035/124 ,  A61K45/06 ,  C12N2740/15043

Abstract: The present invention provides compositions and methods for the treatment or prevention of a neurological disease or disorder of the central nervous system (e.g., a storage disorder, lysosomal storage disorder, neurodegenerative disease, etc.) by reconstitution of brain myeloid cell and microglia upon transplantation of hematopoietic cells enriched in microglia reconstitution potential. The invention also provides compositions and methods for ablating and reconstituting microglia.

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：US20230181719A1

公开(公告)日：2023-06-15

申请号：US17912765

申请日：2021-03-19

Applicant: OSPEDALE SAN RAFFAELE S.R.L.

Inventor： Flavio CURNIS ,  Angelo CORTI ,  Andrès J. M. FERRERI

IPC: A61K39/215 ,  A61P31/14

CPC classification number: A61K39/215 ,  A61P31/14 ,  A61K2039/572

Abstract: The present invention relates to a conjugate comprising a first peptide of sequence CNGRCG (SEQ ID NO: 1) linked to the N-terminus of a protein and a compound X linked to the N-terminus of said peptide and to related medical uses.

公开(公告)号：US20230151027A1

公开(公告)日：2023-05-18

申请号：US17798784

申请日：2021-02-09

Applicant: Ospedale San Raffaele S.r.l. ,  Istituto Nazionale Genetica Molecolare - INGM

Inventor： Romano DI FABIO ,  Vincenzo SUMMA ,  Leda Ivanova BENCHEVA ,  Raffaele DE FRANCESCO ,  Lorena DONNICI ,  Luca GUIDOTTI ,  Matteo IANNACONE ,  Adolfo PRANDI ,  Marilenia DE MATTEO ,  Pietro RANDAZZO

IPC: C07D515/14

CPC classification number: C07D515/14

Abstract: The present invention relates to compounds that are inhibitors of hepatitis B virus (HBV). Compounds of this invention are useful alone or in combination with other agents for treating, ameliorating, preventing or curing HBV infection and related conditions. The present invention also relates to pharmaceutical compositions containing the compounds.

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：US20220098252A1

公开(公告)日：2022-03-31

申请号：US17425359

申请日：2020-01-27

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

Inventor： Davide GABELLINI ,  Roberto GIAMBRUNO ,  Valeria RUNFOLA ,  Claudia CARONNI

IPC: C07K14/47 ,  C12N15/63 ,  A61P35/02 ,  A61P25/00

Abstract: The present invention relates to an inhibitor of DUX4 and its use, in particular in the prevention and/or treatment of a condition associated with an aberrant expression and/or function of at least one DUX4 protein and/or of at least one DUX4 fusion protein. Preferably the inhibitor is MATRIN-3 (MATR3), fragment, variant, fusion, or conjugate thereof. The invention also relates to a pharmaceutical composition comprising such inhibitor, to vector and nucleic acids.

公开(公告)号：US20210317411A1

公开(公告)日：2021-10-14

申请号：US17250050

申请日：2019-05-16

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

Inventor： Claire LATROCHE ,  Luigi NALDINI ,  Maura MANZI

IPC: C12N5/0789 ,  A61K35/12

Abstract: Use of CD47 and/or C-X-C chemokine receptor type 4 (CXCR4) for increasing engraftment by haematopoietic stem C and/or progenitor cells (HSPCs).

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.