US Patent

US7838657 — Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences

Composition of Matter · Assigned to University of Massachusetts Amherst · Expires 2027-07-11 · 1y remaining

Vulnerability score 17/100 Ironclad — strong claim type, well-established, deep family

What this patent protects

This patent protects methods and compositions that block an inhibitory sequence in the SMN2 gene to modulate the splicing of pre-mRNA and elevate SMN protein expression.

USPTO Abstract

The present invention is directed to methods and compositions capable of blocking the inhibitory effect of a newly-identified intronic inhibitory sequence element, named ISS-N1 (for “intronic splicing silencer”), located in the SMN2 gene. The compositions and methods of the instant invention include oligonucleotide reagents (e.g., oligoribonucleotides) that effectively target the SMN2 ISS-N1 site in the SMN2 pre-mRNA, thereby modulating the splicing of SMN2 pre-mRNA to include exon 7 in the processed transcript. The ISS-N1 blocking agents of the invention cause elevated expression of SMN protein, thus compensating for the loss of SMN protein expression commonly observed in subjects with spinal muscular atrophy (SMA).

Drugs covered by this patent

Patent Metadata

Patent number
US7838657
Jurisdiction
US
Classification
Composition of Matter
Expires
2027-07-11
Drug substance claim
Yes
Drug product claim
No
Assignee
University of Massachusetts Amherst
Source
FDA Orange Book + USPTO grounding via Google Patents

Bibliographic data sourced from FDA Orange Book + USPTO public records. Plain-English summary generated by AI grounded in source text. Patent term extensions (PTR, SPC, pediatric) may shift the effective expiry. Not legal advice.

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