US 12,128,109 B2
Muscle targeting complexes and formulations for treating dystrophinopathies
Timothy Weeden, Waltham, MA (US); Scott Hilderbrand, Cambridge, MA (US); Sean Spring, Waltham, MA (US); Peiyi Shen, Waltham, MA (US); Cody A. Desjardins, Waltham, MA (US); Romesh R. Subramanian, Framingham, MA (US); Mohammed T. Qatanani, Waltham, MA (US); Brendan Quinn, Boston, MA (US); and John Najim, Waltham, MA (US)
Assigned to Dyne Therapeutics, Inc., Waltham, MA (US)
Filed by Dyne Therapeutics, Inc., Waltham, MA (US)
Filed on Aug. 24, 2023, as Appl. No. 18/455,351.
Application 18/455,351 is a continuation of application No. PCT/US2022/073540, filed on Jul. 8, 2022.
Claims priority of provisional application 63/220,426, filed on Jul. 9, 2021.
Prior Publication US 2024/0016950 A1, Jan. 18, 2024
This patent is subject to a terminal disclaimer.
Int. Cl. A61K 39/00 (2006.01); A61K 47/54 (2017.01); A61K 47/65 (2017.01); A61K 47/68 (2017.01); A61P 21/00 (2006.01)
CPC A61K 47/6807 (2017.08) [A61K 47/545 (2017.08); A61K 47/548 (2017.08); A61K 47/65 (2017.08); A61K 47/6849 (2017.08); A61P 21/00 (2018.01)] 28 Claims
 
1. A complex comprising a structure of formula (I): [R1]n1—R2,
wherein each R1 comprises a group of the formula (Ic):

OG Complex Work Unit Chemistry
wherein R2 comprises a Fab that binds to transferrin receptor 1 (TfR1), wherein the Fab comprises a heavy chain comprising the amino acid sequence of SEQ ID NO: 19 and a light chain comprising the amino acid sequence of SEQ ID NO: 20;
wherein R1 is covalently linked to R2 at attachment point A; wherein n1 is an integer of 1-5 representing the number of instances of R1, and wherein each instance of R1 is covalently linked to a different amino acid residue of the Fab.