USPTO Patent Application for Modulating SMN2 RNA and Protein
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)
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
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