PATENT CLAIM ANALYSIS

Application Number: 15754782
Application Type: Utility
Filing Date: 2018-02
Publication Date: 2019-01
Patent Classification: ["514", "04400A"]

Abstract:
The present disclosure relates to modified antisense oligomers and related compositions and methods for increasing the expression of functional SMN protein and methods for treating spinal muscular atrophy and relates to inducing inclusion of exon 7 in SMN2 mRNA.

Claim (Index 23):
A pharmaceutical composition, comprising a compound of formula (V): or a pharmaceutically acceptable salt thereof, wherein:\n each Nu is a nucleobase which taken together forms a targeting sequence; \n Z is an integer from 6 to 38; \n each Y is independently selected from O and \u2014NR 4 , wherein each R 4  is independently selected from H, C 1 -C 6  alkyl, \n aralkyl, \u2014C(\u2550NH)NH 2 , \u2014C(O)(CH 2 ) n NR 5 C(\u2550NH)NH 2 , \u2014C(O)(CH 2 ) 2 NHC(O)(CH 2 ) 5 NR 5 C(\u2550NH)NH 2 , and G, wherein R 5  is selected from H and C 1 -C 6  alkyl and n is an integer from 1 to 5;\n T is selected from OH and a moiety of the formula: \n wherein:\n A is selected from \u2014OH, \u2014N(R 7 ) 2 R 8 , and R 1  wherein: \n each R 7  is independently selected from H and C 1 -C 6  alkyl, and \n R 8  is selected from an electron pair and H, and \n R 6  is selected from OH, \u2014N(R 9 )CH 2 C(O)NH 2 , and a moiety of the formula: \n wherein:\n R 9  is selected from H and C 1 -C 6  alkyl; and \n R 10  is selected from G, \u2014C(O)\u2014R 11 OH, acyl, trityl, 4-methoxytrityl, \u2014C(\u2550NH)NH 2 , \u2014C(O)(CH 2 ) m NR 12 C(\u2550NH)NH 2 , and \u2014C(O)(CH 2 ) 2 NHC(O)(CH 2 ) 5 NR 12 C(\u2550NH)NH 2 , wherein:\n m is an integer from 1 to 5, \n R 11  is of the formula \u2014(O-alkyl) y - wherein y is an integer from 3 to 10 and\n each of the y alkyl groups is independently selected from C 2 -C 6  alkyl; and \n \n R 12  is selected from H and C 1 -C 6  alkyl; \n \n each instance of R 1  is independently selected from:\n \u2014N(R 13 ) 2 R 14  wherein each R 13  is independently selected from H and C 1 -C 6  alkyl, and R 14  is selected from an electron pair and H; \n a moiety of formula (II): \n wherein:\n R 15  is selected from H, G, C 1 -C 6  alkyl, \u2014C(\u2550NH)NH 2 , \u2014C(O)(CH 2 ) q NR 18 C(\u2550NH)NH 2 , and \u2014C(O)(CH 2 ) 2 NHC(O)(CH 2 ) 5 NR 18 C(\u2550NH)NH 2 , wherein:\n R 18  is selected from H and C 1 -C 6  alkyl; and \n q is an integer from 1 to 5, \n \n R 16  is selected from an electron pair and H; and \n each R 17  is independently selected from H and methyl; and \n a moiety of formula (III): wherein:\n R 19  is selected from H, C 1 -C 6  alkyl, \u2014C(\u2550NH)NH 2 , \u2014C(O)(CH 2 ) 11 NR 22 C(\u2550NH)NH 2 , \u2014C(O)CH(NH 2 )(CH 2 ) 3 NHC(\u2550NH)NH 2 , \u2014C(O)(CH 2 ) 2 NHC(O)(CH 2 ) 5 NR 22 C(\u2550NH)NH 2 , \u2014C(O)CH(NH 2 )(CH 2 ) 4 NH 2 , and G wherein:\n R 22  is selected from H and C 1 -C 6  alkyl; and \n r is an integer from 1 to 5, \n \n R 20  is selected from H and C 1 -C 6  alkyl; and \n R 21  is selected from an electron pair and H; and \n R 2  is selected from G, H, acyl, trityl, 4-methoxytrityl, and 01-06 alkyl, wherein G is a cell penetrating peptide (\u201cCPP\u201d) and linker moiety selected from \u2014C(O)(CH 2 ) 5 NH\u2014CPP, \u2014C(O)(CH 2 ) 2 NH\u2014CPP, \u2014C(O)(CH 2 ) 2 NHC(O)(CH 2 ) 5 NH\u2014CPP, and \u2014C(Q)CH 2 NH\u2014CPP, or G is of the formula: wherein the CPP is attached to the linker moiety by an amide bond at the CPP carboxy terminus, with the proviso that up to one instance of G is present, wherein the targeting sequence: (a) comprises a sequence selected from SEQ ID NOS: 4 to 16, (b) is selected from SEQ ID NOS: 4 to 16 and 33 to 45, (c) is a fragment of at least 8 contiguous nucleotides of a targeting sequence selected from SEQ ID NOS: 4 to 16 and 33 to 45, or (d) is a variant having at least 90% sequence identity to a targeting sequence selected from SEQ ID NOS: 4 to 16 and 33 to 45, wherein each X is independently selected from uracil (U) and thymine (T), and wherein each Y is independently selected from cytosine (C) and 5-Methylcytosine (5mC), and wherein at least one of the following conditions is present:\n c) at least one R 1  is of formula (H) or of formula (III), or \n d) R 2  is G or T is:

Metadata:
- Claim Count in Document: 11.0
- Percentile: 88.0
- Lexical Diversity: 1.25
- Patent Class: 514.0
- Transitional Phrase Type: open
- Component Type: 1
- Foreign Priority: False
- Related Applications: ['14360895', '14713857', '14714104', '11801885', '15727483']

Analysis Scores:
- 35 USC 101 Eligibility (BERT): 0.7606506998655483
- 35 USC 102 Novelty (BERT): 0.5485530977582714
- Combined Prediction Score: 0.7394409396548207
- Mean Citation Score: 368.839766
- Max Citation Score: 393.62296
- Similarity Product: 343.5153811996508

Labels:
- Claim Label 101: 1
- Claim Label 102: 1
- Claim Label 103: 0
- Claim Label 112: 1
- Combined Label: 1
- Label 101 Adjusted: 1

Dataset: test