PATENT CLAIM ANALYSIS

Application Number: 15921518
Application Type: Utility
Filing Date: 2018-03
Publication Date: 2018-09
Patent Classification: ["514", "044000"]

Abstract:
The invention relates to the use of an antisense compound for inducing exon inclusion as a treatment for Spinal Muscle Atrophy (SMA). More particularly it relates to inducing inclusion of exon 7 to restore levels of Survival Motor Neuron (SMN) protein encoded by the Survival Motor Neuron (SMN) gene.

Claim (Index 18):
An antisense oligonucleotide comprising a sequence selected from 14 to 21 nucleotides, from 15 to 25 nucleotides, and from 20 to 30 nucleotides in length which specifically hybridizes to a region within the SMN2 pre-mRNA selected from within exon 7, intron 7, exon 8, or a portion of intron 7 and exon 8 of the SMN2 pre-mRNA, such that the level of exon 7-containing SMN2 mRNA relative to exon 7-deleted SMN2 mRNA in the cell is enhanced, wherein the antisense oligonucleotide is a morpholino oligonucleotide comprising:\n at least one nucleotide comprising an internucleoside linkage that is positively charged at physiological pH, wherein the at least one nucleotide is of the formula: wherein Nu is a nucleobase; R x  is selected from the group consisting of HO\u2014, a nucleotide, a cell penetrating peptide moiety, and piperazinyl; R y  is selected from the group consisting of hydrogen, a C 1 -C 6  alkyl, a nucleotide, a peptide moiety, an amino acid, a formamidinyl moiety, and acyl; and, R z  is selected from the group consisting of null, hydrogen, a C 1 -C 6  alkyl, and acyl; and pharmaceutically acceptable salts thereof, wherein the sequence is selected from: AT T \u2003CAC\u2003TTT\u2003CAT\u2003AAT\u2003GC T \u2003GG;   ATT\u2003CAC\u2003T T T\u2003CA T \u2003AA T \u2003GCT\u2003GG;   AT T \u2003CAC\u2003T T T\u2003CAT\u2003AA T \u2003GC T \u2003GG;   AT T \u2003CAC\u2003T T T\u2003CA T \u2003AA T \u2003GC T \u2003GG;   AT T \u2003CAC\u2003T T T\u2003CA T \u2003AA T \u2003GC T \u2003GG;   AT T \u2003C A C\u2003T T T\u2003CAT\u2003AAT\u2003GCT\u2003GG;   AT T \u2003C A C\u2003T T T\u2003 C AT\u2003AA T \u2003GC T \u2003GG;   CAC\u2003 T TT\u2003CAT\u2003AAT\u2003GCT\u2003GG;   CAC\u2003 T TT\u2003CA T \u2003AAT\u2003GCT\u2003GG;   CAC\u2003 T T T \u2003CA T \u2003AAT\u2003GCT\u2003GG;   C A C\u2003 T T T \u2003CA T \u2003AA T \u2003GC T \u2003GG;   C A C\u2003 T T T \u2003CA T \u2003 A A T \u2003GC T \u2003GG;   C A C\u2003 T T T \u2003 C AT\u2003 A A T \u2003GC T \u2003GG;   C A C\u2003 T T T \u2003 C AT\u2003 A AT\u2003 G C T \u2003GG;   CAC\u2003TTT\u2003C A T\u2003 A A T \u2003GCT\u2003GG;   C A C\u2003TTT\u2003CAT\u2003AAT\u2003GC T \u2003GG;   T T T\u2003CA T \u2003AAT\u2003GC T \u2003GG;   T T T\u2003CA T \u2003AA T \u2003GC T \u2003GG;   T T T\u2003CA T \u2003 A A T \u2003GC T \u2003GG;   T T T\u2003 C A T \u2003 A A T \u2003GC T \u2003GG;   TTT\u2003 C A T \u2003 A A T \u2003 G C T \u2003GG;   TTT\u2003 C A T \u2003 A A T \u2003GC T \u2003 G G;   C\u2003 T AG\u2003 T AT\u2003TTC\u2003C T G\u2003CAA\u2003A T G\u2003AG;   C\u2003 T AG\u2003 T AT\u2003 T TC\u2003C T G\u2003CAA\u2003A T G\u2003AG;   C\u2003 T AG\u2003 T AT\u2003 T TC\u2003C T G\u2003C A A\u2003A T G\u2003AG;   C\u2003 T AG\u2003 T AT\u2003 T TC\u2003C T G\u2003C A A\u2003A T G\u2003 A G;   C \u2003 T AG\u2003 T AT\u2003 T TC\u2003C T G\u2003C A A\u2003A T G\u2003AG;   C\u2003 T AG\u2003 T AT\u2003TTC\u2003CTG\u2003CAA\u2003A T G\u2003 A G;   C\u2003 T AG\u2003 T AT\u2003TTC\u2003CTG\u2003CAA\u2003A T G\u2003AG;   C\u2003TAG\u2003TAT\u2003T T C\u2003C T G\u2003CAA\u2003A T G\u2003AG;   C\u2003TAG\u2003TAT\u2003T T C\u2003C T G\u2003C A A\u2003ATG\u2003AG;   C\u2003TAG\u2003TA T \u2003T T C\u2003C T G\u2003C A A\u2003ATG\u2003AG;   C\u2003CAG\u2003CA T \u2003T T C\u2003C T G\u2003CAA\u2003A T G\u2003AG;   C\u2003CAG\u2003CA T \u2003T T C\u2003C T G\u2003C A A\u2003A T G\u2003AG;   C\u2003C A G\u2003CA T \u2003T T C\u2003 C TG\u2003C A A\u2003A T G\u2003AG;   C\u2003CA G \u2003CA T \u2003T T C\u2003 C TG\u2003C A A\u2003A T G\u2003AG;   C\u2003C A G\u2003CA T \u2003TTC\u2003CTG\u2003CAA\u2003A T G\u2003AG;   C\u2003C A G\u2003CA T \u2003TTC\u2003CTG\u2003CAA\u2003A T G\u2003 A G;   C\u2003CAG\u2003CAT\u2003T T C\u2003CTG\u2003C A A\u2003ATG\u2003AG;   C\u2003CAG\u2003CAT\u2003T T C\u2003CTG\u2003C A A\u2003 A TG\u2003AG;   A\u2003 T GC\u2003CAG\u2003CA T \u2003TTC\u2003C T G\u2003CAA\u2003A T G\u2003AGA;   A\u2003 T GC\u2003C A G\u2003CAT\u2003TTC\u2003CTG\u2003C A A\u2003A T G\u2003AGA;   A\u2003 T GC\u2003C A G\u2003CA T \u2003TTC\u2003C T G\u2003C A A\u2003A T G\u2003AGA;   A\u2003 T GC\u2003C A G\u2003 C AT\u2003TTC\u2003C T G\u2003 C AA\u2003A T G\u2003AGA;   A\u2003TGC\u2003C A G\u2003CAT\u2003 T TC\u2003C T G\u2003 C AA\u2003ATG\u2003A G A;   A\u2003 T GC\u2003CAG\u2003CAT\u2003TTC\u2003C T G\u2003CAA\u2003A T G\u2003AGA;   A\u2003 T GC\u2003CAG\u2003CAT\u2003TTC\u2003CTG\u2003CAA\u2003A T G\u2003AGA;   GCT\u2003C T A\u2003TGC\u2003CAG\u2003CAT\u2003TTC\u2003C T G\u2003CAA\u2003A;   GCT\u2003C T A\u2003TGC\u2003CAG\u2003CA T \u2003TTC\u2003C T G\u2003CAA\u2003A;   GCT\u2003C T A\u2003TGC\u2003C A G\u2003CA T \u2003TTC\u2003C T G\u2003CAA\u2003A;   GC T \u2003C T A\u2003TGC\u2003CAG\u2003CAT\u2003TTC\u2003C T G\u2003C A A\u2003A;   GCT\u2003CTA\u2003TG C \u2003C A G\u2003C A T\u2003 T TC\u2003CTG\u2003CAA\u2003A;   GC T \u2003C T A\u2003TGC\u2003CAG\u2003CA T \u2003TTC\u2003C T G\u2003CAA\u2003A;   GC T \u2003C T A\u2003TGC\u2003CAG\u2003CA T \u2003T T C\u2003CTG\u2003CAA\u2003A;   GC T \u2003C T A\u2003TGC\u2003CAG\u2003CA T \u2003T T C\u2003C T G\u2003CAA\u2003A; and   GC T \u2003C T A\u2003TGC\u2003CAG\u2003CA T \u2003T T C\u2003CTG\u2003C A A\u2003A, wherein A represents the nucleobase adenine, T represents the nucleobase thymine, C represents the nucleobase cytosine, and G represents the nucleobase guanine, and where the nucleobase in the sequence is bold indicates the at least one nucleotide.

Metadata:
- Claim Count in Document: 5.0
- Percentile: 90.0
- Lexical Diversity: 1.5
- Patent Class: 514.0
- Transitional Phrase Type: open
- Component Type: 1
- Foreign Priority: False
- Related Applications: ['14360895', '14647952', '14360890', '14134057', '14443367']

Analysis Scores:
- 35 USC 101 Eligibility (BERT): 0.7827375770709157
- 35 USC 102 Novelty (BERT): 0.5863958940440784
- Combined Prediction Score: 0.763103408768232
- Mean Citation Score: 384.646352
- Max Citation Score: 509.5931
- Similarity Product: 425.04984709357024

Labels:
- Claim Label 101: 1
- Claim Label 102: 1
- Claim Label 103: 1
- Claim Label 112: 1
- Combined Label: 1
- Label 101 Adjusted: 1

Dataset: test