Therapeutic agent for glycogen storage disease type IA
US11958878B2 · kind B2 · utility
Assignees
Inventors
Key dates
| Filing date | Mar 5, 2019 |
| Grant date | Apr 16, 2024 |
| Priority date | — |
| Expiry date | Mar 5, 2039 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12Y301/03009
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention establishes a molecular therapy for glycogen storage disease type Ia. The present invention provides an oligonucleotide of 15-30 bases comprising a nucleotide sequence complementary to die cDNA of G6PC gene with c.648G>T mutation, wherein the oligonucleotide comprises a sequence complementary to a region comprising any site between the 82nd to the 92nd nucleotide from the 5′ end of exon 5 of the G6PC gene with c.648C>T mutation, a pharmacologically acceptable salt or solvate thereof. Also provided is a pharmaceutical drug comprising the oligonucleotide, a pharmacologically acceptable salt or solvate thereof (e.g., therapeutic drug for glycogen storage disease type Ia).
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.