公开(公告)号：US20240350661A1

公开(公告)日：2024-10-24

申请号：US18597488

申请日：2024-03-06

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

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：US12059478B2

公开(公告)日：2024-08-13

申请号：US16302120

申请日：2017-05-19

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

Inventor： Anna Christina Kajaste-Rudnitski ,  Francesco Piras

IPC: A61K48/00 ,  A61P7/06 ,  A61P43/00 ,  C07K14/47 ,  C12N15/00 ,  C12N15/86

CPC classification number: A61K48/0058 ,  A61K48/0008 ,  A61K48/0066 ,  A61K48/0091 ,  A61P7/06 ,  A61P43/00 ,  C07K14/4746 ,  C12N15/86 ,  C12N2750/14132 ,  C12N2750/14141

Abstract: An inhibitor of p53 activation for use in haematopoietic stem and/or progenitor cell gene therapy, preferably wherein the inhibitor is an inhibitor of p53 phosphorylation, more preferably an inhibitor of p53 Serine 15 phosphorylation.

公开(公告)号：US20240218330A1

公开(公告)日：2024-07-04

申请号：US18288768

申请日：2022-04-26

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

Inventor： Anna Christina Kajaste-Rudnitski ,  Erika Valeri ,  Giulia Unali ,  Francesco Piras

IPC: C12N5/0789 ,  A61K31/513 ,  A61K31/7072 ,  A61K35/28 ,  A61K38/13 ,  C12N15/86

CPC classification number: C12N5/0647 ,  A61K31/513 ,  A61K31/7072 ,  A61K35/28 ,  A61K38/13 ,  C12N15/86 ,  C12N2500/40 ,  C12N2501/04 ,  C12N2510/00 ,  C12N2740/15043

Abstract: A combination of: (a) at least one deoxyribonucleoside (dN) or a derivative thereof and cyclosporin H (CsH) or a derivative thereof: or (b) at least one pyrimidine precursor and cyclosporin H (CsH) or a derivative thereof.

公开(公告)号：US20230365996A1

公开(公告)日：2023-11-16

申请号：US18030711

申请日：2021-10-12

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

Inventor： Anna Villa ,  Pietro Genovese ,  Pietro Genovese ,  Nicolo Sacchetti ,  Maria Carmina Castiello ,  Samuele Ferrari

IPC: C12N15/90 ,  C12N9/10 ,  C12N9/22 ,  C12N5/0789

CPC classification number: C12N15/907 ,  C12N5/0647 ,  C12N9/104 ,  C12N9/22 ,  A61K48/00 ,  C12N2750/14143

Abstract: The present invention relates to an isolated polynucleotide comprising from 5′ to 3′: a first homology region, a splice acceptor sequence, a nucleotide sequence encoding a RAG1 polypeptide, and a second homology region for use in treating a RAG-deficient immunodeficiency.

公开(公告)号：US20230134843A1

公开(公告)日：2023-05-04

申请号：US17910971

申请日：2021-03-11

Applicant: OSPEDALE SAN RAFFAELE S.R.L.

Inventor： Giulio FERRARI ,  Paolo RAMA

IPC: A61K31/675 ,  A61K45/06 ,  A61P43/00

Abstract: The present invention refers to an inhibitor of substance P and/or of its receptor for use in the treatment and/or prevention of stem cell deficiency. Preferably the inhibitor is an NK1 antagonist. Preferably the stem cell deficiency is a corneal epithelial stem cell deficiency. The invention also refers to pharmaceutical compositions containing the inhibitor for use in the treatment and/or prevention of stem cell deficiency.

公开(公告)号：US20220251161A1

公开(公告)日：2022-08-11

申请号：US17614268

申请日：2020-05-28

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

Inventor： Luigi Naldini ,  Alessio Cantore ,  Matteo Iannacone ,  Luca Guidotti ,  Renato Ostuni

IPC: C07K14/55 ,  C12N15/86 ,  C12N15/113 ,  A61K35/17 ,  A61K38/17 ,  A61P31/20

Abstract: An interleukin which binds to IL-2 receptor (IL-2R), or a nucleotide sequence encoding therefor, wherein the interleukin or nucleotide sequence is adapted to be targeted to the liver.

公开(公告)号：US20220031765A1

公开(公告)日：2022-02-03

申请号：US17298837

申请日：2019-12-02

Applicant: OSPEDALE SAN RAFFAELE S.R.L.

Inventor： Matteo Maria Salvatore BELLONE ,  Arianna BREVI ,  Arianna CALCINOTTO ,  Roberto FERRARESE ,  Filippo CANDUCCI

IPC: A61K35/74 ,  A61K35/741 ,  A61K45/06 ,  A61P35/00

Abstract: The present invention relates to the field of bacterial strain to be used in medicine. In particular, it relates to the prevention and/or treatment of cancer or diabetes, bacterial strain is Prevotella melaninogenica.

公开(公告)号：US20210290646A1

公开(公告)日：2021-09-23

申请号：US17261928

申请日：2019-07-22

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

Inventor： Attilio BONDANZA ,  Monica CASUCCI ,  Beatrice GRECO

IPC: A61K31/7004 ,  A61K35/17 ,  A61K39/395 ,  A61K31/145 ,  A61K31/437 ,  A61K31/7008 ,  A61K31/7072 ,  A61K31/407 ,  A61K45/06 ,  A61P35/00 ,  A61K38/17 ,  A61P35/02 ,  C12N5/0783

Abstract: The present invention relates to at least one glycosylation inhibitor for use in combination with CAR cell therapy. Preferably, the glycosylation inhibitor improves the therapeutic potential of the CAR cell therapy. The invention also relates to a pharmaceutical composition and to population or subpopulation of CAR cell that has been contacted with at least one glycosylation inhibitor.

公开(公告)号：US20210277354A1

公开(公告)日：2021-09-09

申请号：US17253301

申请日：2019-06-19

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

Inventor： Andrea ANNONI ,  Silvia Adriana GREGORI

IPC: C12N5/0784 ,  C12N5/0783 ,  A61K35/15 ,  C12N15/85 ,  A61K45/06

Abstract: The present disclosure relates to a genetically modified dendritic cell or precursor thereof expressing at least one anti-gen-derived peptide and at least one immuno-modulatory molecule, its medical use and method of preparation. The invention also relates to an in vitro method to produce IL-10-producing CD49b+LAG-3+ Tr1 cells or antigen-specific FOXP3+ T cells and relative medical uses and pharmaceutical compositions.

公开(公告)号：US20210222197A1

公开(公告)日：2021-07-22

申请号：US17055151

申请日：2019-05-16

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

Inventor： Alessio Cantore ,  Andrea Annoni ,  Michela Milani ,  Luigi Naldini

IPC: C12N15/86 ,  C12N5/0783 ,  C12N7/00

Abstract: An enveloped viral particle producer or packaging cell, wherein the cell is genetically engineered to decrease expression of CD47 on the surface of the cell.