	US 11,674,154 B2
	Gene therapeutics for fibrodysplasia ossificans progressiva
Jae-Hyuck Shim, Westborough, MA (US); Guangping Gao, Westborough, MA (US); Jun Xie, Shrewsbury, MA (US); Yeon-Suk Yang, Westborough, MA (US); Jung min Kim, Worcester, MA (US); and Sachin Chaugule, Shrewsbury, MA (US)
Assigned to University of Massachusetts, Boston, MA (US)
Filed by University of Massachusetts, Boston, MA (US)
Filed on Dec. 2, 2021, as Appl. No. 17/541,141.
Claims priority of provisional application 63/251,822, filed on Oct. 4, 2021.
Claims priority of provisional application 63/121,221, filed on Dec. 3, 2020.
Prior Publication US 2022/0177915 A1, Jun. 9, 2022
Int. Cl. C12N 15/86 (2006.01); A61P 19/08 (2006.01); A61K 38/45 (2006.01); A61K 48/00 (2006.01); C12N 9/22 (2006.01); C12N 15/11 (2006.01)

CPC C12N 15/86 (2013.01) [A61K 38/45 (2013.01); A61K 48/0066 (2013.01); A61P 19/08 (2018.01); C12N 9/22 (2013.01); C12N 15/111 (2013.01); C12Y 207/1103 (2013.01); C12N 2310/141 (2013.01); C12N 2310/20 (2017.05); C12N 2320/34 (2013.01); C12N 2750/14143 (2013.01); C12N 2830/008 (2013.01)]

12 Claims

1. A recombinant adeno-associated virus (rAAV) comprising:

(i) an isolated nucleic acid comprising a transgene comprising a promoter operably linked to a codon optimized nucleic acid sequence encoding an activin receptor type-1 (ACVR1) protein, wherein the codon optimized nucleic acid sequence comprises the sequence of SEQ ID NO: 1; and

(ii) at least one AAV capsid protein.