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1.发明申请公开(公告)号：US20190300906A1
公开(公告)日：2019-10-03
申请号：US16302341
申请日：2017-05-18
Applicant: MODERNATX, INC.
Inventor： Paolo MARTINI , Stephen HOGE , Kerry BENENATO , Vladimir PRESNYAK , Iain McFADYEN , Ellalahewage Sathyajith KUMARASINGHE , Ding AN , Staci SABNIS
Abstract: The invention relates to mRNA therapy for the treatment of galactosemia type 1 (Gal-1). mRNAs for use in the invention, when administered in vivo, encode human galactose-1-phosphate uridylyltransferase (GALT), isoforms thereof, functional fragments thereof, and fusion proteins comprising GALT. 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 GALT expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GALT activity in subjects, namely galactose-1-phosphate (Gal-1-P).
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公开(公告)号：US20190298657A1
公开(公告)日：2019-10-03
申请号：US16302298
申请日：2017-05-18
Applicant: MODERNATX, INC.
Inventor： Paolo MARTINI , Stephen HOGE , Kerry BENENATO , Vladimir PRESNYAK , Iain McFADYEN , Ellalahewage Sathyajith KUMARASINGHE , Evan Lockwood RACHLIN , Staci SABNIS
Abstract: The invention relates to mRNA therapy for the treatment of VLCADD. mRNAs for use in the invention, when administered in vivo, encode human acyl-CoA dehydrogenase, very longchain (ACADVL), isoforms thereof, functional fragments thereof, and fusion proteins comprising ACADVL. 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 ACADVL expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient ACADVL activity in subjects, namely acylcarnitine and acylcarnitine metabolites.
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3.发明申请公开(公告)号：US20190298658A1
公开(公告)日：2019-10-03
申请号：US16302370
申请日：2017-05-18
Applicant: MODERNATX, INC.
Inventor： Kerry BENENATO , Stephen HOGE , Iain McFADYEN , Vladimir PRESNYAK , Paolo MARTINI , Ellalahewage Sathyajith KUMARASINGHE
Abstract: The invention relates to mRNA therapy for the treatment of cystic fibrosis. mRNAs for use in the invention, when administered in vivo, encode cystic fibrosis transmembrane conductance regulator (CFTR), isoforms thereof, functional fragments thereof, and fusion proteins comprising CFTR. 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 CFTR expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient CFTR activity in subjects.
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