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USPTO Patent Application for Modulating SMN2 RNA and Protein

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Published March 26th, 2026
Detected March 27th, 2026
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Summary

The USPTO has published a patent application (US20260085313A1) detailing compounds, methods, and pharmaceutical compositions for modulating SMN2 RNA and protein. This application, filed on May 2, 2025, aims to ameliorate symptoms of neurodegenerative disorders.

What changed

The United States Patent and Trademark Office (USPTO) has published patent application US20260085313A1, titled 'COMPOUNDS AND METHODS FOR MODULATING SMN2'. The application, filed on May 2, 2025, by inventors including Frank Rigo, describes novel compounds and methods for modulating SMN2 RNA and protein, intended to treat neurodegenerative disorders. The publication date is March 26, 2026.

This is a patent application, not a regulatory rule or guidance. It does not impose new compliance obligations on regulated entities. However, it signifies potential future intellectual property and market exclusivity for specific therapeutic approaches in the biotechnology and pharmaceutical sectors. Companies operating in this space should be aware of this filing for competitive intelligence and potential licensing or collaboration opportunities.

Source document (simplified)

← USPTO Patent Applications

COMPOUNDS AND METHODS FOR MODULATING SMN2

Application US20260085313A1 Kind: A1 Mar 26, 2026

Inventors

Frank Rigo, Thazha P. Prakash, Kar Yun Karen Ling, W. Brad Wan, William John Drury, III

Abstract

Provided are compounds, methods, and pharmaceutical compositions for modulating SMN2 RNA and/or protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a neurodegenerative disorder. Such symptoms include reduced muscle strength; inability or reduced ability to sit upright, to stand, and/or walk; reduced neuromuscular activity; reduced electrical activity in one or more muscles; reduced respiration; inability or reduced ability to eat, drink, and/or breathe without assistance; loss of weight or reduced weight gain; and/or decreased survival.

CPC Classifications

C12N 15/113 C12N 2310/11 C12N 2310/315 C12N 2310/321 C12N 2310/322 C12N 2310/3341

Filing Date

2025-05-02

Application No.

19197566

View original document →

Named provisions

Inventors Abstract CPC Classifications

Classification

Agency
USPTO
Published
March 26th, 2026
Instrument
Notice
Legal weight
Non-binding
Stage
Final
Change scope
Minor
Document ID
US20260085313A1

Who this affects

Applies to
Drug manufacturers Pharmaceutical companies
Industry sector
3254 Pharmaceutical Manufacturing 3254.1 Biotechnology
Activity scope
Drug Development Biotechnology Research
Geographic scope
United States US

Taxonomy

Primary area
Pharmaceuticals
Operational domain
Legal
Compliance frameworks
FDA 21 CFR Part 11 GxP
Topics
Biotechnology Medical Research

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