Gene therapy for treating propionic acidemia
AssigneeUltragenyx Pharmaceutical Inc.
InventorsMatthew Scott Fuller, Samuel Wadsworth, Kelly Reed Clark, Sean Christopher Daugherty, Stewart Craig
Life of this patent
Estimating the term…
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).
Claims3
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.
Dependencies are inferred from the claim text.