Patents Assigned to Prevail Therapeutics, Inc.
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Publication number: 20240100132Abstract: Aspects of the disclosure relate to compositions and methods for positively or negatively regulating the expression of a gene therapeutic (e.g., a therapeutic protein expressed from an AAV vector). The disclosure is based, in part, on certain nucleic acids, for example antisense oligonucleotides (ASOs), configured to bind specific regions of an expression cassette (or an mRNA transcribed from such an expression cassette).Type: ApplicationFiled: January 24, 2022Publication date: March 28, 2024Applicant: Prevail Therapeutics, Inc.Inventors: Benjamin Shykind, Asa Abeliovich, E. David Litwack, Kumar Anindya Sen
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Patent number: 11903985Abstract: 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.Type: GrantFiled: April 10, 2020Date of Patent: February 20, 2024Assignee: Prevail Therapeutics, Inc.Inventors: Asa Abeliovich, Laura Heckman, Herve Rhinn, Franz Hefti
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Publication number: 20230405149Abstract: The disclosure relates, in some aspects, to compositions and methods for treatment of neurodegenerative disease, for example Alzheimer's disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding an APOE Christchurch (e.g., APOE3ch and/or APOE2ch) protein isoform or a portion thereof, an inhibitory nucleic acid targeting an APOE gene or a portion thereof, or any combination of the foregoing. In some embodiments, the disclosure provides methods of treating Alzheimer's disease by administering an expression construct to a subject in need thereof.Type: ApplicationFiled: November 24, 2021Publication date: December 21, 2023Applicant: Prevail Therapeutics, Inc.Inventors: Asa Abeliovich, Sitharthan Kamalakaran, Benjamin Shykind, Edmund c. Schwartz, Anindya Kumar Sen
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Patent number: 11807849Abstract: 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.Type: GrantFiled: October 3, 2018Date of Patent: November 7, 2023Assignee: Prevail Therapeutics, Inc.Inventors: Asa Abeliovich, Laura Heckman, Herve Rhinn
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Patent number: 11802294Abstract: 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.Type: GrantFiled: October 3, 2018Date of Patent: October 31, 2023Assignee: Prevail Therapeutics, Inc.Inventors: Asa Abeliovich, Laura Heckman, Herve Rhinn
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Publication number: 20230304036Abstract: The disclosure relates, in some aspects, to compositions and methods for delivery of transgenes to a subject. In some embodiments, the disclosure provides expression constructs (e.g., vectors containing an expression construct) comprising a transgene encoding human Parkin or a portion thereof. In some embodiments, the disclosure provides methods of treating a neurodegenerative disease (e.g., Parkinson’s disease) by administering such expression constructs to a subject in need thereof.Type: ApplicationFiled: August 3, 2021Publication date: September 28, 2023Applicant: Prevail Therapeutics, Inc.Inventors: Asa Abeliovich, Benjamin Shykind
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Patent number: 11661585Abstract: 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.Type: GrantFiled: April 6, 2020Date of Patent: May 30, 2023Assignee: Prevail Therapeutics, Inc.Inventors: Asa Abeliovich, Laura Heckman, Herve Rhinn
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Patent number: 11655460Abstract: 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.Type: GrantFiled: June 18, 2020Date of Patent: May 23, 2023Assignee: Prevail Therapeutics, Inc.Inventors: Asa Abeliovich, Laura Heckman, Herve Rhinn
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Patent number: 11613737Abstract: 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.Type: GrantFiled: April 6, 2020Date of Patent: March 28, 2023Assignee: Prevail Therapeutics, Inc.Inventors: Asa Abeliovich, Laura Heckman, Herve Rhinn
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Publication number: 20220211871Abstract: The disclosure relates, in some aspects, to compositions and methods for treatment of central nervous system (CNS) diseases, for example Parkinson's disease (PD) and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding one or more CNS disease-associated gene products and/or one or more an inhibitory nucleic acids targeting a CNS disease-associated gene or gene product. In some embodiments, the disclosure provides methods of treating CNS diseases by administering such expression constructs to a subject in need thereof.Type: ApplicationFiled: April 10, 2020Publication date: July 7, 2022Applicant: Prevail Therapeutics, Inc.Inventors: Asa Abeliovich, Laura Heckman, Herve Rhinn
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Publication number: 20220010001Abstract: The disclosure relates, in some aspects, to compositions and methods for treatment of neurodegenerative disease, for example Alzheimer's disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding an APOE protein isoform or a portion thereof, an inhibitory nucleic acid targeting an APOE gene or a portion thereof, or any combination of the foregoing. In some embodiments, the disclosure provides methods of treating Alzheimer's disease by administering an expression construct to a subject in need thereof.Type: ApplicationFiled: November 26, 2019Publication date: January 13, 2022Applicant: Prevail Therapeutics, Inc.Inventors: Asa Abeliovich, Laura Heckman, Herve Rhinn
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Publication number: 20210261981Abstract: The disclosure relates, in some aspects, to compositions and methods for treatment of neurodegenerative diseases (e.g., amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD), Alzheimer's disease, Gaucher disease, Parkinson's disease, Lewy body dementia, or a lysosomal storage disease). In some embodiments, the disclosure provides expression constructs comprising a transgene encoding one or more inhibitory nucleic acids (e.g., targeting C9orf72, TMEM106B, ATNX2, RPS25, etc.), wild-type C9orf72 protein or a portion thereof, or any combination of the foregoing. In some embodiments, the disclosure provides methods of ALS/FTD by administering such expression constructs to a subject in need thereof.Type: ApplicationFiled: October 23, 2018Publication date: August 26, 2021Applicant: Prevail Therapeutics, Inc.Inventors: Asa Abeliovich, Laura Heckman, Herve Rhinn
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Patent number: 11060113Abstract: 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.Type: GrantFiled: September 17, 2020Date of Patent: July 13, 2021Assignee: Prevail Therapeutics, Inc.Inventors: Asa Abeliovich, Laura Heckman, Herve Rhinn
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Patent number: 10837028Abstract: 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.Type: GrantFiled: November 20, 2019Date of Patent: November 17, 2020Assignee: Prevail Therapeutics, Inc.Inventors: Asa Abeliovich, Laura Heckman, Herve Rhinn
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Publication number: 20200318115Abstract: 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.Type: ApplicationFiled: April 2, 2020Publication date: October 8, 2020Applicant: Prevail Therapeutics, Inc.Inventors: Asa Abeliovich, Laura Heckman, Herve Rhinn
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Publication number: 20200283800Abstract: The disclosure relates, in some aspects, to compositions and methods for treatment of neurodegenerative diseases (e.g., amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD), Alzheimer's disease, Gaucher disease, Parkinson's disease, Lewy body dementia, or a lysosomal storage disease). In some embodiments, the disclosure provides expression constructs comprising a trans gene encoding one or more inhibitory nucleic acids (e.g., targeting C9orj72, TMEMI 06B, ATNX2, RPS25, etc.), wild-type C9orf72 protein or a portion thereof, or any combination of the foregoing. In some embodiments, the disclosure provides methods of ALS/FTD by administering such expression constructs to a subject in need thereof.Type: ApplicationFiled: April 24, 2020Publication date: September 10, 2020Applicant: Prevail Therapeutics, Inc.Inventors: Asa Abeliovich, Laura Heckman, Herve Rhinn
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Publication number: 20200282080Abstract: 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.Type: ApplicationFiled: April 6, 2020Publication date: September 10, 2020Applicant: Prevail Therapeutics, Inc.Inventors: Asa Abeliovich, Laura Heckman, Herve Rhinn
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Publication number: 20200276335Abstract: 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.Type: ApplicationFiled: October 3, 2018Publication date: September 3, 2020Applicant: Prevail Therapeutics, Inc.Inventors: Asa Abeliovich, Laura Heckman, Herve Rhinn
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Patent number: 10689625Abstract: 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.Type: GrantFiled: November 21, 2019Date of Patent: June 23, 2020Assignee: Prevail Therapeutics, Inc.Inventors: Asa Abeliovich, Laura Heckman, Herve Rhinn