5 patents
Utility
Compositions and methods for TTR gene editing and treating ATTR amyloidosis
24 Oct 23
Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene are provided.
Arti Mahendra Prakash Kanjolia, Shobu Odate, Jessica Lynn Seitzer, Reynald Michael Lescarbeau, Walter Strapps
Filed: 9 Dec 22
Utility
Polynucleotides, compositions, and methods for genome editing
11 Jul 23
Compositions and methods for gene editing.
Christian Dombrowski, Jonathan Douglas Finn, Amy Madison Rhoden Smith, Seth C. Alexander
Filed: 24 Mar 20
Utility
Compositions and methods for treating alpha-1 antitrypsin deficiency
10 Jan 23
Compositions and methods for introducing double-stranded breaks within the SERPINA1 gene are provided.
Shobu Odate, Walter Strapps, Reynald Michael Lescarbeau
Filed: 20 Jun 19
Utility
Modified guide RNAs
25 Oct 22
This disclosure relates to modified single and dual guide RNAs having improved in vitro and in vivo activity in gene editing methods.
Amy Madison Rhoden Smith, David V. Morrissey, Walter Strapps
Filed: 7 Jun 19
Utility
Compositions and methods for the treatment of hemoglobinopathies
11 Oct 22
The present invention is directed to genome editing systems, reagents and methods for the treatment of hemoglobinopathies.
Craig Stephen Mickanin, Christian Schmedt, Jennifer Snead, Susan Stevenson, Yi Yang
Filed: 5 Feb 18
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