Intellia Therapeutics, Inc.

Shobu Odate

Reynald Michael Lescarbeau

Walter R Strapps

Jessica Lynn Seitzer

Arti Mahendra Prakash Kanjolia

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.

Compositions and methods for kallikrein (KLKB1) gene editing

Compositions and methods for introducing double-stranded breaks within the SERPINA1 gene are provided. Compositions and methods for reducing and eliminating mutant forms of α1-antitrypsin (AAT), such as seen in subjects having α1-antitrypsin deficiency (AATD), are provided.

Compositions and methods for treating alpha-1 antitrypsin deficiency

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.

Compositions and methods for TTR gene editing and treating ATTR amyloidosis

Compositions and methods for introducing double-stranded breaks within the SERPINA1 gene are provided. Compositions and methods for reducing and eliminating mutant forms of &#x3b1;1-antitrypsin (AAT), such as seen in subjects having &#x3b1;1-antitrypsin deficiency (AATD), are provided.

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Arlington, MA, United States of America

Shobu Odate