Abstract: Compositions and methods for editing, e.g., altering a DNA sequence, within a LAG3 gene are provided. Compositions and methods for immunotherapy are provided.
Abstract: Compositions and methods for editing, e.g., altering a DNA sequence, within a 2B4 gene are provided. Compositions and methods for immunotherapy are provided.
Abstract: Compositions and methods for editing, e.g., altering a DNA sequence, within a TIM3 gene are provided. Compositions and methods for immunotherapy are provided.
Abstract: Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene are provided. Compositions and methods for treating subjects having amyloidosis associated with transthyretin (ATTR), are provided.
Type:
Grant
Filed:
December 9, 2022
Date of Patent:
October 24, 2023
Assignee:
Intellia Therapeutics, Inc.
Inventors:
Arti Mahendra Prakash Kanjolia, Shobu Odate, Jessica Lynn Seitzer, Reynald Michael Lescarbeau, Walter Strapps
Abstract: Compositions and methods for editing, e.g., introducing double-stranded breaks, within the KLKB1 gene are provided. Compositions and methods for treating subjects having hereditary angioedema (HAE), are provided.
Abstract: This disclosure relates to the field of poly-adenylated (poly-A) tails. In some embodiments, a DNA encodes a poly-A tail located 3? to nucleotides encoding a protein of interest, wherein the poly-A tail comprises one or more non-adenine nucleotide.
Abstract: Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene are provided. Compositions and methods for treating subjects having amyloidosis associated with transthyretin (ATTR), are provided.
Type:
Application
Filed:
December 9, 2022
Publication date:
August 17, 2023
Applicant:
Intellia Therapeutics, Inc.
Inventors:
Arti Mahendra Prakash Kanjolia, Shobu Odate, Jessica Lynn Seitzer, Reynald Michael Lescarbeau, Walter Strapps
Abstract: Compositions and methods for gene editing. In some embodiments, a polynucleotide encoding Cas9 is provided that can provide one or more of improved editing efficiency, reduced immunogenicity, or other benefits.
Type:
Grant
Filed:
March 24, 2020
Date of Patent:
July 11, 2023
Assignee:
Intellia Therapeutics, Inc.
Inventors:
Christian Dombrowski, Jonathan Douglas Finn, Amy Madison Rhoden Smith, Seth C. Alexander
Abstract: Compositions and methods for introducing double-stranded breaks within the SERPINA1 gene are provided. Compositions and methods for reducing and eliminating mutant forms of ?1-antitrypsin (AAT), such as seen in subjects having ?1-antitrypsin deficiency (AATD), are provided.
Type:
Application
Filed:
December 9, 2022
Publication date:
July 6, 2023
Applicant:
Intellia Therapeutics, Inc.
Inventors:
Shobu Odate, Walter Strapps, Reynald Michael Lescarbeau
Abstract: Compositions and methods for multiplex delivery and gene editing in vitro are provided.
Type:
Application
Filed:
October 27, 2022
Publication date:
June 15, 2023
Applicant:
Intellia Therapeutics, Inc.
Inventors:
Pooja Kyatsandra Narendra, Sean Michael Burns, Paula Gutierrez Martinez, Arti Mahendra Prakash Kanjolia, Anthony Monti, Aaron Prodeus, Mohamed Simo Arredouani, Özgün Kiliç, Reed Walker LaRiviere, Palak Sushil Sharma, Eleni Stampouloglou, Qingzhan Zhang
Abstract: This disclosure relates to the field of poly-adenylated (poly-A) tails. In some embodiments, a DNA encodes a poly-A tail located 3? to nucleotides encoding a protein of interest, wherein the poly-A tail comprises one or more non-adenine nucleotide.
Abstract: Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene are provided. Compositions and methods for treating subjects having amyloidosis associated with transthyretin (ATTR), are provided.
Type:
Application
Filed:
December 9, 2022
Publication date:
April 20, 2023
Applicant:
Intellia Therapeutics, Inc.
Inventors:
Arti Mahendra Prakash Kanjolia, Shobu Odate, Jessica Lynn Seitzer, Reynald Michael Lescarbeau, Walter Strapps
Abstract: Provided herein are compositions and methods for increasing editing efficiency of a target nucleic acid. A composition may comprise a guide nucleic acid, a Cas9 nickase, or a reverse transcriptase. The reverse transcriptase may be fused to the Cas9 nickase. The reverse transcriptase may heterodimerize with the Cas9 nickase. The reverse transcriptase may bind to a guide nucleic acid. The reverse transcriptase may be engineered to increase processivity. The guide nucleic acid may be engineered to facilitate synthesis or editing of a sequence. The guide nucleic acid, Cas9 nickase, and reverse transcriptase may be engineered to fit within AAV vectors. The guide nucleic acid may comprise a region that binds to another region on the guide nucleic acid to improve gene editing.
Abstract: Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene are provided. Compositions and methods for treating subjects having amyloidosis associated with transthyretin (ATTR), are provided.
Type:
Application
Filed:
September 27, 2021
Publication date:
February 9, 2023
Applicant:
Intellia Therapeutics, Inc.
Inventors:
Yong Chang, Seth C. Alexander, Kristy M. Wood, Arti Mahendra Prakash Kanjolia, Shobu Odate, Jessica Lynn Seitzer, Reynald Michael Lescarbeau, Walter Strapps
Abstract: Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene in combination with administration of a corticosteroid are provided. Compositions and methods for treating subjects having amyloidosis associated with transthyretin (ATTR), in which a guide RNA and a corticosteroid are administered, are provided.
Type:
Application
Filed:
September 27, 2021
Publication date:
February 2, 2023
Applicant:
Intellia Therapeutics, Inc.
Inventors:
Yong CHANG, Seth C. ALEXANDER, Kristy M. WOOD, Arti Mahendra Prakash KANJOLIA, Shobu ODATE, Jessica Lynn SEITZER, Reynald Michael LESCARBEAU, Walter STRAPPS
Abstract: Compositions and methods for gene editing. In some embodiments, a polynucleotide encoding Cas9 is provided that can provide one or more of improved editing efficiency, reduced immunogenicity, or other benefits.
Type:
Application
Filed:
September 27, 2021
Publication date:
January 19, 2023
Applicant:
Intellia Therapeutics, Inc.
Inventors:
Bradley Andrew Murray, Christian Dombrowski, Seth C. Alexander
Abstract: Compositions and methods for introducing double-stranded breaks within the SERPINA1 gene are provided. Compositions and methods for reducing and eliminating mutant forms of ?1-antitrypsin (AAT), such as seen in subjects having ?1-antitrypsin deficiency (AATD), are provided.
Type:
Grant
Filed:
June 20, 2019
Date of Patent:
January 10, 2023
Assignee:
Intellia Therapeutics, Inc.
Inventors:
Shobu Odate, Walter Strapps, Reynald Michael Lescarbeau