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Last Updated: September 27, 2020

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Patent: 9,217,147

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Summary for Patent: 9,217,147
Title:Spinal muscular atrophy treatment via targeting SMN2 catalytic core
Abstract: The present invention is directed to methods and compositions for blocking the effect of the intronic inhibitory splicing region of intron 7 of the SMN2 gene. The compositions and methods of the instant invention include short oligonucleotide reagents (e.g., oligoribonucleotides) that effectively target sites in the SMN2 pre-mRNA, thereby modulating the splicing of SMN2 pre-mRNA to include exon 7 in the processed transcript. The short target regions are 8-mers and 5-mers and also include the identification of a single nucleotide base that is essential for initiating a long distance stearic inhibitory interactions as well as novel targets distant from intron 7 which block the intronic inhibitory splicing of the same. These short target regions and concomitant inhibitory blocking oligonucleotides are less expensive and easier to manufacture and are small enough to cross the blood brain barrier.
Inventor(s): Singh; Ravindra N. (Ames, IA), Singh; Natalia N. (Ames, IA)
Assignee: Iowa State Research Foundation, Inc. (Ames, IA)
Application Number:14/134,057
Patent Claims:see list of patent claims

Details for Patent 9,217,147

Applicant Tradename Biologic Ingredient Dosage Form BLA Number Approval Date Patent No. Assignee Estimated Patent Expiration Status Orphan Source
Schering INTRON A interferon alfa-2b VIAL 103132 001 1986-06-04   Start Trial Iowa State Research Foundation, Inc. (Ames, IA) 2030-04-28 RX search
Schering INTRON A interferon alfa-2b VIAL 103132 002 1986-06-04   Start Trial Iowa State Research Foundation, Inc. (Ames, IA) 2030-04-28 RX search
Schering INTRON A interferon alfa-2b VIAL 103132 003 1986-06-04   Start Trial Iowa State Research Foundation, Inc. (Ames, IA) 2030-04-28 RX search
>Applicant >Tradename >Biologic Ingredient >Dosage Form >BLA >Number >Approval Date >Patent No. >Assignee >Estimated Patent Expiration >Status >Orphan >Source

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