Search International and National Patent Collections

1. (WO2017201328) POLYNUCLEOTIDES ENCODING α-GALACTOSIDASE A FOR THE TREATMENT OF FABRY DISEASE

Pub. No.:    WO/2017/201328    International Application No.:    PCT/US2017/033398
Publication Date: Fri Nov 24 00:59:59 CET 2017 International Filing Date: Fri May 19 01:59:59 CEST 2017
IPC: C12N 15/67
Applicants: MODERNATX, INC.
Inventors: MARTINI, Paolo
HOGE, Stephen
BENENATO, Kerry
PRESNYAK, Vladimir
MCFADYEN, Iain
KUMARASINGHE, Ellalahewage Sathyajith
ZHU, Xuling
GUEY, Lin Tung
SABNIS, Staci
Title: POLYNUCLEOTIDES ENCODING α-GALACTOSIDASE A FOR THE TREATMENT OF FABRY DISEASE
Abstract:
The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the α-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.