Priyam Singh

US Patent:

20230112920, Apr 13, 2023

Filed:

Apr 15, 2022

Appl. No.:

17/721765

Inventors:

- Carlsbad CA, US
Frank Rigo - Carlsbad CA, US
Priyam Singh - San Diego CA, US

Assignee:

Ionis Pharmaceuticals, Inc. - Carlsbad CA

International Classification:

C12N 15/113
G01N 33/68

Abstract:

Disclosed herein are compositions and methods for reducing expression of C9ORF72 mRNA and protein in an animal with C9ORF72 specific inhibitors. Also disclosed herein are compositions and methods of selectively inhibiting a C9ORF72 pathogenic associated mRNA variant by administering an antisense compound targeting the region beginning at the start site of exon 1A to the start site of exon 1B of a C9ORF72 pre-mRNA. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C9ORF72 specific inhibitors include antisense compounds.

US Patent:

20210346421, Nov 11, 2021

Filed:

May 21, 2021

Appl. No.:

17/327469

Inventors:

- Carlsbad CA, US
Punit P. Seth - Carlsbad CA, US
Michael L. McCaleb - La Jolla CA, US
Susan M. Freier - San Diego CA, US
Priyam Singh - San Diego CA, US

International Classification:

A61K 31/713
A61P 27/02
A61K 31/475
A61K 31/704
C07K 14/705

Abstract:

The present embodiments provide methods, compounds, and compositions for treating, preventing, ameliorating, or slowing progression of retinitis pigmentosa (RP), such as autosomal dominant retinitis pigmentosa (AdRP) by administering a P23H rhodopsin specific inhibitor to a subject. The present embodiments provided herein are directed to compounds and compositions useful for treating, preventing, ameliorating, or slowing progression of retinitis pigmentosa (RP), such as autosomal dominant retinitis pigmentosa (AdRP). In certain embodiments, P23H rhodopsin inhibitors provided herein are allele-specific antisense compounds targeted to a P23H mutant allele that are capable of selectively inhibiting expression of P23H rhodopsin mutant protein to a greater extent than wild-type protein. In certain embodiments, administration of the allele specific antisense compounds in a subject having AdRP results in selective inhibition of P23H rhodopsin and allows the normal protein produced from the wild-type allele to maintain rod survival and function in the subject.

US Patent:

20200392494, Dec 17, 2020

Filed:

Nov 9, 2018

Appl. No.:

16/759698

Inventors:

- Carlsbad CA, US
Priyam Singh - San Diego CA, US
Susan M. Freier - San Diego CA, US
Tracy A. Cole - Encinitas CA, US

Assignee:

Ionis Pharmaceuticals, Inc. - Carlsbad CA

International Classification:

C12N 15/113
A61P 25/16

Abstract:

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SNCA mRNA in a cell or animal, and in certain instances reducing the amount of alpha-synuclein protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include motor dysfunction, aggregation of alpha-synuclein, neurodegeneration, cognitive decline and dementia. Such neurodegenerative diseases include Parkinson's disease, dementia with Lewy bodies, diffuse Lewy body disease, pure autonomic failure, multiple system atrophy, neuronopathic Gaucher's disease and Alzheimer's disease.

US Patent:

20200069722, Mar 5, 2020

Filed:

Aug 28, 2019

Appl. No.:

16/553799

Inventors:

- Carlsbad CA, US
Punit P. Seth - Carlsbad CA, US
Michael L. McCaleb - La Jolla CA, US
Susan M. Freier - San Diego CA, US
Priyam Singh - San Diego CA, US

International Classification:

A61K 31/713
C07K 14/705
A61K 31/704
A61K 31/475
A61P 27/02

Abstract:

The present embodiments provide methods, compounds, and compositions for treating, preventing, ameliorating, or slowing progression of retinitis pigmentosa (RP), such as autosomal dominant retinitis pigmentosa (AdRP) by administering a P23H rhodopsin specific inhibitor to a subject. The present embodiments provided herein are directed to compounds and compositions useful for treating, preventing, ameliorating, or slowing progression of retinitis pigmentosa (RP), such as autosomal dominant retinitis pigmentosa (AdRP). In certain embodiments, P23H rhodopsin inhibitors provided herein are allele-specific antisense compounds targeted to a P23H mutant allele that are capable of selectively inhibiting expression of P23H rhodopsin mutant protein to a greater extent than wild-type protein. In certain embodiments, administration of the allele specific antisense compounds in a subject having AdRP results in selective inhibition of P23H rhodopsin and allows the normal protein produced from the wild-type allele to maintain rod survival and function in the subject.

US Patent:

20200056179, Feb 20, 2020

Filed:

Jul 25, 2019

Appl. No.:

16/522133

Inventors:

- Carlsbad CA, US
Priyam Singh - San Diego CA, US
Frank Rigo - Carlsbad CA, US
Holly Kordasiewicz - San Diego CA, US

Assignee:

Ionis Pharmaceuticals, Inc. - Carlsbad CA

International Classification:

C12N 15/113
A61K 47/02
A61K 47/46

Abstract:

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN2 RNA in a cell or animal, and in certain instances reducing the amount of Ataxin-2 protein in a cell or animal Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include spinocerebellar ataxia type 2 (SCA2), amyotrophic lateral sclerosis (ALS), and parkinsonism.

US Patent:

20190367916, Dec 5, 2019

Filed:

Jan 15, 2019

Appl. No.:

16/248612

Inventors:

- Carlsbad CA, US
Frank Rigo - Carlsbad CA, US
Priyam Singh - San Diego CA, US

Assignee:

Ionis Pharmaceuticals, Inc. - Carlsbad CA

International Classification:

C12N 15/113
G01N 33/68

Abstract:

Disclosed herein are compositions and methods for reducing expression of C9ORF72 mRNA and protein in an animal with C9ORF72 specific inhibitors. Also disclosed herein are compositions and methods of selectively inhibiting a C9ORF72 pathogenic associated mRNA variant by administering an antisense compound targeting the region beginning at the start site of exon 1A to the start site of exon 1B of a C9ORF72 pre-mRNA. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C9ORF72 specific inhibitors include antisense compounds.

US Patent:

20180169131, Jun 21, 2018

Filed:

Feb 26, 2016

Appl. No.:

15/553876

Inventors:

- Carlsbad CA, US
Punit P. SETH - Carlsbad CA, US
Michael L. McCALEB - La Jolla CA, US
Susan M. FREIER - San Diego CA, US
Priyam SINGH - San Diego CA, US

Assignee:

Ionis Pharmaceuticals, Inc. - Carlsbad CA

International Classification:

A61K 31/713
A61K 31/475
A61K 31/704
C07K 14/705
A61P 27/02

Abstract:

The present embodiments provide methods, compounds, and compositions for treating, preventing, ameliorating, or slowing progression of retinitis pigmentosa (RP), such as autosomal dominant retinitis pigmentosa (AdRP) by administering a P23H rhodopsin specific inhibitor to a subject. The present embodiments provided herein are directed to compounds and compositions useful for treating, preventing, ameliorating, or slowing progression of retinitis pigmentosa (RP), such as autosomal dominant retinitis pigmentosa (AdRP). In certain embodiments, P23H rhodopsin inhibitors provided herein are allele-specific antisense compounds targeted to a P23H mutant allele that are capable of selectively inhibiting expression of P23H rhodopsin mutant protein to a greater extent than wild-type protein. In certain embodiments, administration of the allele specific antisense compounds in a subject having AdRP results in selective inhibition of P23H rhodopsin and allows the normal protein produced from the wild-type allele to maintain rod survival and function in the subject.

US Patent:

20170037410, Feb 9, 2017

Filed:

Apr 1, 2015

Appl. No.:

15/301004

Inventors:

- Carlsbad CA, US
Tracy Cole - Carlsbad CA, US
Holly Kordasiewicz - San Diego CA, US
Susan M. Freier - San Diego CA, US
Thomas P. Condon - Singapore, SG
Edward Wancewicz - Poway CA, US
Trisha Lockhart - Seattle WA, US
Timothy Vickers - Oceanside CA, US
Priyam Singh - San Diego CA, US

Assignee:

Ionis Pharmaceuticals, Inc. - Carlsbad CA

International Classification:

C12N 15/113

Abstract:

Disclosed herein are antisense compounds and methods for decreasing SOD-1 mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate SOD-1 associated diseases, disorders, and conditions. Such SOD-1 associated diseases include amyotrophic sclerosis (ALS).