US 12,357,707 B2
Compositions and methods for treating laminopathies
Sirika Wood, South San Francisco, CA (US); Kartik Ramamoorthi, South San Francisco, CA (US); Stephanie Tagliatela, South San Francisco, CA (US); and Anne Tanenhaus, South San Francisco, CA (US)
Assigned to Encoded Therapeutics, Inc., South San Francisco, CA (US)
Appl. No. 17/433,927
Filed by Encoded Therapeutics, Inc., South San Francisco, CA (US)
PCT Filed Feb. 28, 2020, PCT No. PCT/US2020/020520
§ 371(c)(1), (2) Date Aug. 25, 2021,
PCT Pub. No. WO2020/176896, PCT Pub. Date Sep. 3, 2020.
Claims priority of provisional application 62/812,021, filed on Feb. 28, 2019.
Prior Publication US 2022/0193264 A1, Jun. 23, 2022
Int. Cl. A61K 48/00 (2006.01); C07K 14/78 (2006.01); C12N 15/86 (2006.01)
CPC A61K 48/0058 (2013.01) [C07K 14/78 (2013.01); C12N 15/86 (2013.01); C12N 2750/14143 (2013.01); C12N 2830/008 (2013.01)] 29 Claims
 
1. A nucleic acid construct comprising a nucleotide sequence encoding a lamin A polypeptide and a lamin C polypeptide, wherein said construct comprises introns 8-11 of the wildtype human LMNA gene and lacks introns 1-7 of the wildtype-human LMNA gene, wherein the lamin A polypeptide comprises an amino acid sequence that is at least 95% identical to the amino acid sequence of SEQ ID NO: 12 or SEQ ID NO: 21, and wherein said lamin C polypeptide comprises an amino acid sequence that is at least 95% identical to the amino acid sequence of SEQ ID NO: 13.