IP Library › Granted Patent US 12686874
Granted Patent B2
US 12686874 · App. 17/906,902 · Granted Jul 21, 2026

Gene therapy for treating propionic acidemia

Inventors: Matthew Scott Fuller (Millis, MA); Samuel Wadsworth (Monterey, MA); Kelly Reed Clark (Westerville, OH); Sean Christopher Daugherty (Petaluma, CA); Stewart Craig (Cambridge, MA)
Assignee: Ultragenyx Pharmaceutical Inc.
C12N15/86A61P3/00C12N9/93C12Y604/01003A61K48/00C12N2750/14143C12N2800/22C12N2830/50
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Quick Facts
Patent No.
US 12686874
App. No.
17/906,902
Granted
Jul 21, 2026
Kind
B2
Abstract

This present disclosure provides recombinant adeno-associated virus (rAAV) and methods of their use in gene therapy for treating propionic acidemia (PA). Also provided are pharmaceutical compositions comprising a rAAV of the invention and a pharmaceutically acceptable carrier or excipient. These pharmaceutical compositions may be useful in gene therapy for the treatment of PA caused by a mutation in propionyl-CoA carboxylase α-subunit (PCCA) or a mutation in propionyl-CoA carboxylase β-subunit (PCCB).

Claims (3)

1 . A polynucleotide which comprises a nucleic acid sequence at least 90% identical, at least 95% identical, at least 99% identical, or 100% identical to SEQ ID NO: 30 or SEQ ID NO: 31.

2 . An isolated host cell comprising the polynucleotide of claim 1 , wherein the isolated host cell is capable of producing an adeno-associated viral (AAV) vector.

3 . The isolated host cell of claim 2 , wherein the host cell is selected from a HeLa, Cos-7, HEK293, A549, BHK, Vero, RD, HT-1080, ARPE-19, and MRC-5 cell.