PATENT CLAIM ANALYSIS

Application Number: 15969374
Application Type: Utility
Filing Date: 2018-05
Publication Date: 2018-11
Patent Classification: ["514", "419000"]

Abstract:
A repeat expansion in C9ORF72 causes frontotemporal dementia and amyotrophic lateral sclerosis (c9FTD/ALS). RNA of the expanded repeat (r(GGGGCC)exp) forms nuclear foci or undergoes repeat-associated non-ATG (RAN) translation producing “c9RAN proteins”. Since neutralizing r(GGGGCC) exp  could inhibit these potentially toxic events, we sought to identify small molecule binders of r(GGGGCC) exp . Chemical and enzymatic probing of r(GGGGCC) 8  indicate it adopts a hairpin structure in equilibrium with a quadruplex structure. Using this model, bioactive small molecules targeting r(GGGGCC) exp  were designed and found to significantly inhibit RAN translation and foci formation in cultured cells expressing r(GGGGCC) 66  and neurons trans-differentiated from fibroblasts of repeat expansion carriers. Finally, we show that poly(GP) c9RAN proteins are specifically detected in c9ALS patient cerebrospinal fluid. Our findings highlight r(GGGGCC) exp -binding small molecules as a possible c9FTD/ALS therapeutic, and suggest c9RAN proteins could potentially serve as a pharmacodynamic biomarker to assess efficacy of therapies that target r(GGGGCC) exp .

Claim (Index 2):
A method of inhibiting repeat-associated non-ATG (RAN) translation and foci formation in cultured cells expressing r(GGGGCC)66 and neurons trans-differentiated from fibroblasts of repeat expansion carriers, comprising contacting the cells with an effective amount of a compound of  claim 1 , or a pharmaceutically acceptable salt thereof.

Metadata:
- Claim Count in Document: 3.0
- Percentile: 93.0
- Lexical Diversity: 1.77876
- Patent Class: 514.0
- Transitional Phrase Type: open
- Component Type: 1
- Foreign Priority: False
- Related Applications: ['14762623', '14775278', '15518824', '14162570', '15508343']

Analysis Scores:
- 35 USC 101 Eligibility (BERT): 0.7513139464969315
- 35 USC 102 Novelty (BERT): 0.5154112366213843
- Combined Prediction Score: 0.7277236755093769
- Mean Citation Score: 243.318358
- Max Citation Score: 275.6588
- Similarity Product: 169.72342069745062

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