US 12,215,138 B2
Compositions and methods for internalizing enzymes
Andrew Baik, Scarsdale, NY (US); Katherine Cygnar, New York, NY (US); Christopher Schoenherr, Piermont, NY (US); Christos Kyratsous, Irvington, NY (US); and Cheng Wang, Beijing (CN)
Assigned to Regeneron Pharmaceuticals, Inc., Tarrytown, NY (US)
Filed by Regeneron Pharmaceuticals, Inc., Tarrytown, NY (US)
Filed on Nov. 17, 2021, as Appl. No. 17/528,828.
Application 17/528,828 is a continuation of application No. 16/001,717, filed on Jun. 6, 2018, granted, now 11,208,458.
Claims priority of provisional application 62/673,098, filed on May 17, 2018.
Claims priority of provisional application 62/574,719, filed on Oct. 19, 2017.
Claims priority of provisional application 62/516,656, filed on Jun. 7, 2017.
Prior Publication US 2022/0195011 A1, Jun. 23, 2022
This patent is subject to a terminal disclaimer.
Int. Cl. C07K 14/705 (2006.01); A61K 38/47 (2006.01); A61K 48/00 (2006.01); A61P 43/00 (2006.01); C07K 16/28 (2006.01); C12N 9/14 (2006.01); C12N 9/24 (2006.01); C12N 9/34 (2006.01)
CPC C07K 14/70596 (2013.01) [A61K 38/47 (2013.01); A61P 43/00 (2018.01); C07K 16/2896 (2013.01); C12N 9/14 (2013.01); C12N 9/24 (2013.01); C12N 9/2402 (2013.01); C12N 9/2428 (2013.01); A61K 48/00 (2013.01); C07K 2317/24 (2013.01); C07K 2317/31 (2013.01); C07K 2317/622 (2013.01); C07K 2317/77 (2013.01); C07K 2317/92 (2013.01); C07K 2319/00 (2013.01); C07K 2319/33 (2013.01); C12N 2710/10043 (2013.01); C12N 2750/14143 (2013.01)] 24 Claims
 
1. A polynucleotide encoding a multidomain therapeutic protein comprising a delivery domain and an enzyme domain,
wherein the delivery domain is an antigen-binding protein that binds to CD63, and wherein the antigen binding protein is a single-chain variable fragment (scFv),
wherein the enzyme domain comprises an alpha-glucosidase (GAA) or a portion thereof, and
wherein the polynucleotide further comprises a virus nucleic acid sequence, wherein the virus nucleic acid sequence is an adeno-associated virus (AAV) nucleic acid sequence.