US 12,351,611 B2
Molecules for organelle-specific protein delivery
Joan David Bettoun, Elkins Park, PA (US); and Rebecca Wissner, Wayne, PA (US)
Assigned to Larimar Therapeutics, Inc., Bala Cynwyd, PA (US)
Filed by Larimar Therapeutics, Inc., Bala Cynwyd, PA (US)
Filed on Feb. 5, 2024, as Appl. No. 18/432,505.
Application 18/432,505 is a continuation of application No. 17/214,757, filed on Mar. 26, 2021, granted, now 11,891,420.
Claims priority of provisional application 63/000,138, filed on Mar. 26, 2020.
Prior Publication US 2024/0254176 A1, Aug. 1, 2024
This patent is subject to a terminal disclaimer.
Int. Cl. C07K 14/47 (2006.01); A61K 38/00 (2006.01); A61K 47/64 (2017.01); A61P 25/00 (2006.01); A61P 25/16 (2006.01); A61P 25/28 (2006.01); C07K 14/005 (2006.01); C12N 9/02 (2006.01); C12N 9/10 (2006.01)
CPC C07K 14/47 (2013.01) [A61K 47/64 (2017.08); C07K 14/005 (2013.01); C12N 9/104 (2013.01); C12Y 203/02 (2013.01); A61K 38/00 (2013.01); C07K 2319/02 (2013.01); C07K 2319/07 (2013.01); C07K 2319/50 (2013.01); C12N 2740/16322 (2013.01)] 13 Claims
 
1. A fusion protein, comprising:
a protein of interest to be delivered to a cell;
a cell penetrating peptide (CPP); and
a target enhancing sequence (TES);
wherein:
the protein of interest is Frataxin (FXN) comprising an amino acid sequence having at least 85% sequence identity to SEQ ID NO: 1;
the CPP is HIV-TAT comprising an amino acid sequence having at least 85% sequence identity to SEQ ID NO: 11; and
the TES is a nuclear export sequence (NES), wherein the NES comprises an amino acid sequence having at least 85% sequence identity to an amino acid sequence selected from the group consisting of SEQ ID NO: 36, SEQ ID NO: 37, SEQ ID NO: 38, SEQ ID NO: 39, SEQ ID NO: 40, SEQ ID NO: 41, SEQ ID NO: 42 and SEQ ID NO: 43.