公开(公告)号：US20210010032A1

公开(公告)日：2021-01-14

申请号：US17024117

申请日：2020-09-17

Applicant: Prevail Therapeutics, Inc.

Inventor： Asa ABELIOVICH ,  Laura HECKMAN ,  Herve RHINN

IPC: C12N15/861 ,  A61K48/00 ,  C12N15/113 ,  C07K14/435 ,  C07K14/705 ,  C12N7/00 ,  C12N9/24 ,  C12N15/86

Abstract: The disclosure relates, in some aspects, to compositions and methods for treatment of diseases associated with aberrant lysosomal function, for example Parkinson's disease and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding beta-Glucocerebrosidase (GBA) or a portion thereof, Lysosomal Membrane Protein 2 (LIMP2), Prosaposin, or any combination of the foregoing. In some embodiments, the disclosure provides methods of Parkinson's disease by administering such expression constructs to a subject in need thereof

公开(公告)号：US20240123003A1

公开(公告)日：2024-04-18

申请号：US18392002

申请日：2023-12-21

Applicant: Prevail Therapeutics, Inc.

Inventor： Asa ABELIOVICH ,  Laura HECKMAN ,  Herve RHINN ,  Franz HEFTI

IPC: A61K35/761 ,  A61K9/00 ,  A61K38/16 ,  A61P25/28 ,  A61P43/00 ,  C12N15/86

CPC classification number: A61K35/761 ,  A61K9/0085 ,  A61K38/162 ,  A61P25/28 ,  A61P43/00 ,  C12N15/86 ,  A61K9/0019 ,  C12Y302/01045

Abstract: The disclosure relates to compositions and methods for treatment of diseases associated with aberrant lysosomal function, such as Parkinson's disease and Gaucher disease. The disclosure provides expression constructs comprising a transgene encoding beta-glucocerebrosidase, an inhibitory RNA targeting alpha-Synuclein, or a combination of the foregoing. The disclosure further provides methods of treating Gaucher disease, Parkinson's disease or other synucleinopathies by administering such expression constructs to a subject in need thereof.

公开(公告)号：US20210332385A1

公开(公告)日：2021-10-28

申请号：US17369168

申请日：2021-07-07

Applicant: Prevail Therapeutics, Inc.

Inventor： Asa ABELIOVICH ,  Laura HECKMAN ,  Herve RHINN

IPC: C12N15/861 ,  A61K48/00 ,  C12N15/113 ,  C07K14/435 ,  C07K14/705 ,  C12N7/00 ,  C12N9/24 ,  C12N15/86

Abstract: The disclosure relates, in some aspects, to compositions and methods for treatment of diseases associated with aberrant lysosomal function, for example Parkinson's disease and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding beta-Glucocerebrosidase (GBA) or a portion thereof, Lysosomal Membrane Protein 2 (LIMP2), Prosaposin, or any combination of the foregoing. In some embodiments, the disclosure provides methods of Parkinson's disease by administering such expression constructs to a subject in need thereof.

公开(公告)号：US20200231954A1

公开(公告)日：2020-07-23

申请号：US16753016

申请日：2018-10-03

Applicant: Prevail Therapeutics, Inc.

Inventor： Asa ABELIOVICH ,  Laura HECKMAN ,  Herve RHINN

IPC: C12N9/24 ,  C07K14/435 ,  C07K14/705 ,  C12N15/113 ,  C12N15/86 ,  C12N7/00

Abstract: The disclosure relates, in some aspects, to compositions and methods for treatment of diseases associated with aberrant lysosomal function, for example Parkinson's disease and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding beta-Glucocerebrosidase (GBA) or a portion thereof, Lysosomal Membrane Protein 2 (LIMP2), Prosaposin, or any combination of the foregoing. In some embodiments, the disclosure provides methods of Parkinson's disease by administering such expression constructs to a subject in need thereof.

公开(公告)号：US20240287471A1

公开(公告)日：2024-08-29

申请号：US18646957

申请日：2024-04-26

Applicant: Prevail Therapeutics, Inc.

Inventor： Asa ABELIOVICH ,  Laura HECKMAN ,  Herve RHINN ,  Li Chin WONG ,  Hsuan-Ni LIN ,  Franz HEFTI

IPC: C12N7/00 ,  A61K48/00 ,  A61P25/28 ,  C07K14/435 ,  C12N9/02 ,  G01N33/68

CPC classification number: C12N7/00 ,  A61K48/0075 ,  A61K48/0083 ,  A61P25/28 ,  C07K14/435 ,  C12N9/0071 ,  C12Y114/18001 ,  G01N33/6803 ,  C12N2750/14132 ,  C12N2750/14133 ,  C12N2750/14143 ,  G01N2333/435

Abstract: The disclosure relates to compositions and methods for treatment of diseases associated with aberrant lysosomal function, such as fronto-temporal dementia (FTD). The disclosure also provides expression constructs comprising a transgene encoding progranulin or a portion thereof. The disclosure provides methods of treating FTD by administering such expression constructs to a subject in need thereof.

公开(公告)号：US20230287358A1

公开(公告)日：2023-09-14

申请号：US18298529

申请日：2023-04-11

Applicant: Prevail Therapeutics, Inc.

Inventor： Asa ABELIOVICH ,  Laura HECKMAN ,  Herve RHINN

IPC: C12N7/00 ,  C07K14/47 ,  A61P25/28 ,  A61K9/00 ,  A61K35/76 ,  A61K48/00 ,  C07K14/005 ,  C07K14/61 ,  C07K14/705 ,  A61P25/16 ,  C07K14/435 ,  A61K35/761 ,  C12N15/861

CPC classification number: C12N7/00 ,  C07K14/47 ,  A61P25/28 ,  A61K9/0019 ,  A61K9/0085 ,  A61K35/76 ,  A61K48/0058 ,  C07K14/005 ,  C07K14/61 ,  C07K14/70503 ,  A61P25/16 ,  A61K48/0075 ,  C07K14/435 ,  A61K35/761 ,  C12N15/861 ,  C12N2750/14121 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N15/86

Abstract: The disclosure relates, in some aspects, to compositions and methods for treatment of diseases associated with aberrant lysosomal function, for example Parkinson's disease (PD) and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding beta-Glucocerebrosidase (GBA) or a portion thereof alone or in combination with one or more PD-associated genes. In some embodiments, the disclosure provides methods of Parkinson's disease by administering such expression constructs to a subject in need thereof.

公开(公告)号：US20200332265A1

公开(公告)日：2020-10-22

申请号：US16846065

申请日：2020-04-10

Applicant: Prevail Therapeutics, Inc.

Inventor： Asa ABELIOVICH ,  Laura HECKMAN ,  Herve RHINN ,  Li Chin WONG ,  Hsuan-Ni LIN ,  Franz HEFTI

IPC: C12N7/00 ,  C07K14/435 ,  G01N33/68 ,  A61K48/00 ,  C12N9/02 ,  A61P25/28

Abstract: The disclosure relates to compositions and methods for treatment of diseases associated with aberrant lysosomal function, such as fronto-temporal dementia (FTD). The disclosure also provides expression constructs comprising a transgene encoding progranulin or a portion thereof. The disclosure provides methods of treating FTD by administering such expression constructs to a subject in need thereof.

公开(公告)号：US20200308554A1

公开(公告)日：2020-10-01

申请号：US16904909

申请日：2020-06-18

Applicant: PREVAIL THERAPEUTICS, INC.

Inventor： Asa ABELIOVICH ,  Laura HECKMAN ,  Herve RHINN

IPC: C12N7/00 ,  C07K14/47

Abstract: The disclosure relates, in some aspects, to compositions and methods for treatment of diseases associated with aberrant lysosomal function, for example Parkinson's disease (PD) and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding beta-Glucocerebrosidase (GBA) or a portion thereof alone or in combination with one or more PD-associated genes. In some embodiments, the disclosure provides methods of Parkinson's disease by administering such expression constructs to a subject in need thereof.

公开(公告)号：US20200231970A1

公开(公告)日：2020-07-23

申请号：US16753018

申请日：2018-10-03

Applicant: Prevail Therapeutics, Inc.

Inventor： Asa ABELIOVICH ,  Laura HECKMAN ,  Herve RHINN

IPC: C12N15/113 ,  C12N9/24 ,  C07K14/005 ,  C12N7/00 ,  C12N15/86 ,  A61K9/00

Abstract: The disclosure relates, in some aspects, to compositions and methods for treatment of diseases associated with aberrant lysosomal function, for example Parkinson's disease and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding one or more inhibitory nucleic acids targeting SCNA or a portion thereof, TMEM106B or a portion thereof, or any combination of the foregoing. In some embodiments, the disclosure provides methods of Parkinson's disease by administering such expression constructs to a subject in need thereof.