Intellia Therapeutics, Inc.

Novartis Ag

Reynald Michael Lescarbeau

Walter R Strapps

Shobu Odate

Jessica Lynn Seitzer

Arti Mahendra Prakash Kanjolia

Morag Helen Stewart

Yi Yang

Celeste Richardson

Craig Stephen Mickanin

Glenn Dranoff

Melissa Deck

Ming-Wei Chen

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

The present invention is directed to genome editing systems, reagents and methods for immunooncology.

Compositions and methods for immunooncology

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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Medford, MA, United States of America

Reynald Michael Lescarbeau