	US 12,331,331 B2
	Polynucleotides
Amit Nathwani, Rickmansworth (GB); Jenny McIntosh, Epsom (GB); Romuald Corbau, St Albans (GB); Azadeh Kia, Enfield (GB); and Carlos Miranda, Watford (GB)
Assigned to SPUR THERAPEUTICS LIMITED, Stevenage (GB)
Appl. No. 17/428,344
Filed by SPUR THERAPEUTICS LIMITED, Stevenage (GB)
PCT Filed Feb. 4, 2020, PCT No. PCT/GB2020/050251 § 371(c)(1), (2) Date Aug. 4, 2021, PCT Pub. No. WO2020/161483, PCT Pub. Date Aug. 13, 2020.
Claims priority of application No. 1901512 (GB), filed on Feb. 4, 2019; and application No. 1917910 (GB), filed on Dec. 6, 2019.
Prior Publication US 2022/0154159 A1, May 19, 2022
Int. Cl. C12N 9/24 (2006.01); A61K 38/47 (2006.01); A61P 1/16 (2006.01); C12N 15/52 (2006.01)

CPC C12N 9/2402 (2013.01) [A61K 38/47 (2013.01); A61P 1/16 (2018.01); C12N 15/52 (2013.01); C12Y 302/01045 (2013.01)]

7 Claims

1. A non-wild type polynucleotide comprising a beta-glucocerebrosidase (GBA) nucleotide sequence, wherein the GBA nucleotide sequence encodes a β-Glucocerebrosidase (GCase) protein, wherein the GBA nucleotide sequence comprises a sequence that is at least 95% identical to SEQ ID NO: 1, and wherein the GCase protein encoded by the GBA nucleotide sequence has GCase activity and expresses in human liver cells at higher levels compared to a GCase encoded by a wild type GBA nucleotide sequence.