公开(公告)号：US20230066287A1

公开(公告)日：2023-03-02

申请号：US17423029

申请日：2020-01-16

Applicant: OSPEDALE SAN RAFFAELE S.R.L.

Inventor： Francesco TREVISANI ,  Alessandra CINQUE ,  Alessandro LARCHER ,  Luca RAMPOLDI ,  Domenico FICHERA ,  Francesco RIPA

IPC: C12Q1/6886

Abstract: The present invention relates to methods for the screening, diagnosis and/or prognosis of renal cell carcinoma. The methods of the invention are based on the determination of expression profiles of sets of miRNAs representative for renal cell carcinoma optionally associated with genotype analysis, in particular of uromodulin SNP variants. The invention also refers to a kit to perform such methods.

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：US20220056484A1

公开(公告)日：2022-02-24

申请号：US17284160

申请日：2019-10-11

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

Inventor： Pietro Genovese ,  Samuele Ferrari ,  Angelo Leone Lombardo ,  Luigi Naldini ,  Martina Fiumara

IPC: C12N15/90 ,  A61K35/28 ,  A61K38/46 ,  A61K48/00 ,  C12N15/86

Abstract: A method for selecting genome edited cells and/or for enrichment of genome edited cells in a population of cells comprising: (a) introducing into a cell or a population of cells at least one first component, at least one second component and at least one third component; and (b) selecting the genome edited cells which transiently express or transiently upregulate a nucleotide sequence encoding a selector.

公开(公告)号：US20210388072A1

公开(公告)日：2021-12-16

申请号：US17281874

申请日：2019-10-02

Applicant: OSPEDALE SAN RAFFAELE S.R.L. ,  ENTHERA S.R.L.

Inventor： Paolo FIORINA ,  Francesca D'ADDIO ,  Francesca ZAGARI

IPC: C07K16/22

Abstract: The present invention relates to an antibody or antigen binding fragment thereof that binds specifically to IGFBP3 and does not displace the binding of IGF-I to IGFBP3. The antibody inhibits or reduces the binding of IGFBP3 to the TMEM219 receptor. The invention also relates to methods for their production, pharmaceutical compositions containing said antibodies, and uses thereof.

公开(公告)号：US20210346489A1

公开(公告)日：2021-11-11

申请号：US17143953

申请日：2021-01-07

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

Inventor： Alessio Cantore ,  Angelo Leone Lombardo ,  Luigi Naldini

IPC: A61K39/12 ,  C12N7/04 ,  C12N15/86

Abstract: An enveloped viral particle producer or packaging cell, wherein the cell is genetically engineered to decrease expression of MHC-I on the surface of the cell.

公开(公告)号：US20210276967A1

公开(公告)日：2021-09-09

申请号：US17261223

申请日：2019-07-19

Applicant: OSPEDALE SAN RAFFAELE S.R.L. ,  IRBM S.P.A. ,  PROMIDIS S.R.L. ,  ISTITUTO NAZIONALE DI GENETICA MOLECOLARE - INGM

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

IPC: C07D305/08 ,  A61K45/06 ,  C07D207/48 ,  C07D213/42 ,  C07D205/04 ,  C07D213/73 ,  C07D211/96 ,  C07C311/16

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 said compounds.

公开(公告)号：US20210260216A1

公开(公告)日：2021-08-26

申请号：US17255362

申请日：2019-06-25

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

Inventor： Pietro Genovese ,  Giulia Schiroli ,  Luigi Naldini ,  Aurelien Jacob ,  Samuele Ferrari

IPC: A61K48/00 ,  C12N15/90 ,  C12N15/86 ,  C07K14/005

Abstract: An agent which promotes homology directed DNA repair for use in haematopoietic stem and/or progenitor cell gene therapy, wherein said haematopoietic stem cells are gene edited. Use of an agent which promotes homology directed DNA repair, for increasing the survival and/or engraftment of gene edited haematopoietic stemand/or progenitorcells or for increasing the efficiency of gene editing of haematopoietic stem and/or progenitor cells.

公开(公告)号：US20200237962A1

公开(公告)日：2020-07-30

申请号：US16457740

申请日：2019-06-28

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

Inventor： Anna Kajaste-Rudnitski ,  Eugenio Montini

IPC: A61L27/38 ,  C12N5/0789 ,  C07K7/64 ,  A61K35/28 ,  A61L27/36 ,  C12N15/86

Abstract: Use of cyclosporin A (CsA) or a derivative thereof for increasing the efficiency of transduction of an isolated population of human haematopoietic stem and/or progenitor cells by a vector derived from HIV-1, HIV-2, FIV, BIV, EIAV, CAEV or visna lentivirus.

公开(公告)号：US20200087630A1

公开(公告)日：2020-03-19

申请号：US16505774

申请日：2019-07-25

Applicant: Ospedale San Raffaele S.r.l. ,  Istituto Zooprofilattico Sperimentale delle Venezie

Inventor： Lorenzo Piemonti ,  Ilaria Capua

IPC: C12N7/00 ,  A61K45/06 ,  A61K31/351 ,  A61K31/215 ,  A61K39/12 ,  C07K14/005 ,  A61K39/145

Abstract: Type 1 diabetes mellitus is characterized by loss of pancreatic insulin-producing beta cells, resulting in insulin deficiency. The usual cause of this beta cell loss is autoimmune destruction. The inventors provide the first evidence of a causal link between influenza virus infection and the development of type 1 diabetes and/or pancreatitis. This causal link between infection and type 1 diabetes and/or pancreatitis provides various therapeutic, prophylactic and diagnostic opportunities.

公开(公告)号：US10391201B2

公开(公告)日：2019-08-27

申请号：US15306682

申请日：2015-04-24

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

Inventor： Anna Kajaste-Rudnitski ,  Eugenio Montini

IPC: A61K35/28 ,  A61L27/38 ,  C07K7/64 ,  C12N5/0789 ,  C12N15/86 ,  A61L27/36

Abstract: Use of cyclosporin A (CsA) or a derivative thereof for increasing the efficiency of transduction of an isolated population of human haematopoietic stem and/or progenitor cells by a vector derived from HIV-1, HIV-2, FIV, BIV, EIAV, CAEV or visna lentivirus.