Patents

US 12139709 B2: Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene

Granted
2024-11-12
Filed
2021-06-09 application 17343447
Type
Utility · 16 claims
Elsewhere
Google Patents ↗ · USPTO patent document ↗ · USPTO Patent Center record ↗

Abstract

The present disclosure relates to modified antisense oligonucleotides. The nucleotides described herein are of 10 to 40 nucleobases and include a targeting sequence complementary to a target region within intron 1 of a pre-mRNA of the human alpha glucosidase (GAA) gene. The target region includes at least one additional nucleobase compared to the targeting sequence, wherein the at least one additional nucleobase has no complementary nucleobase in the targeting sequence, and wherein the at least one additional nucleobase is internal to the target region.

Source: PVGPATDIS, file of 2026-04-10 (ipg241112.xml).

Assignees

AssigneeTypePlaceAlso on furp.orgSource
SAREPTA THERAPEUTICS, INC. 126 patentsUS company or organizationCambridge, MASarepta Therapeutics, Inc. Form D issuer EXACT high confidence: identical full name and state
SAREPTA THERAPEUTICS, INC. SEC filer EXACT medium confidence: identical full name, one of each nationally
PVGPATDIS, file of 2026-04-10

Inventors

InventorPlacePatentsPerson pageSource
Frederick Joseph SCHNELLCambridge, MA10PVGPATDIS, file of 2026-04-10

CPC categories

SubclassCategoryFirst groupSource
C12NMicroorganisms or enzymes; compositions thereof; propagating, preserving, or maintaining microorganisms; mutation or genetic engineering; culture mediaC12N15/113PVGPATDIS, file of 2026-04-10
A61KPreparations for medical, dental or toiletry purposesA61K9/0019PVGPATDIS, file of 2026-04-10
A61PSpecific therapeutic activity of chemical compounds or medicinal preparationsA61P3/00PVGPATDIS, file of 2026-04-10
C07HSugars; derivatives thereof; nucleosides; nucleotides; nucleic acidsC07H21/02PVGPATDIS, file of 2026-04-10
C12YEnzymesC12Y302/0102PVGPATDIS, file of 2026-04-10
Y02PClimate change mitigation technologies in the production or processing of goodsY02P20/582PVGPATDIS, file of 2026-04-10

Cooperative Patent Classification, as currently assigned (one row per subclass).

Assignments and security interests

RecordedWhatFromToReel/frameSource
2025-05-07Security interest Security interest
Security interest
SAREPTA THERAPEUTICS, INC. signed 2025-02-13JPMORGAN CHASE BANK, N.A. AS ADMINISTRATIVE AGENT CHICAGO, IL71218/445PASDL, file of 2026-10-15

Documents recorded with the USPTO Assignment Recordation Branch that list this patent. A security interest is a lender’s claim on the patent as collateral; a release ends it.

USPTO bulk data: PatentsView granted patents, disambiguated (PVGPATDIS) for patents, assignees, inventors, places and CPC categories; the USPTO Patent Assignment Dataset (ECORSEXC) for assignments and security interests. Patents filed in the last 20 years. No claims or full text. Links to other records are by name (and state): EXACT means the identical full name and state; FUZZY means a looser name match, with the confidence shown.