Claims for Patent: 7,026,122

Summary for Patent: 7,026,122

Title:	Methods of assaying for compounds that inhibit premature translation termination and nonsense-mediated RNA decay
Abstract:	The present application provides methods of assaying for compounds that inhibit premature translation termination and nonsense mediated RNA decay in cells.
Inventor(s):	Beckmann; Holger (El Cerrito, CA), Learned; Marc (El Granada, CA), Peltz; Stuart (Piscataway, NJ), Czaplinski; Kevin (Summerset, NJ)
Assignee:	PTC Therapeutics, Inc. (South Plainfield, NJ)
Application Number:	10/226,803
Patent Claims:	1. A method of in vitro screening for compounds that modulate premature translation termination and nonsense-mediated mRNA decay, the method comprising the steps of: (i) incubating a translation assay, the assay comprising an in vitro translation cellular extract; a nucleic acid encoding a polypeptide, wherein the nucleic acid comprises an intron and at least two exons and wherein the coding sequence for the polypeptide comprises a premature stop codon; and a candidate modulator compound; and (ii) detecting the polypeptide translated from the nucleic acid. 2. The method of claim 1 wherein the cellular extract is from plants, mammals or amphibians. 3. The method of claim 1, wherein the cellular extract is a eukaryotic reticulocyte lysate or a rabbit reticulocyte lysate. 4. The method of claim 1, wherein the cellular extract is a mammalian tissue culture cell extract. 5. The method of claim 1, wherein the cellular extract is a HeLa cell S100 extract. 6. The method of claim 1, wherein the polypeptide is detected by ELISA, light emission, colorimetric measurements or enzymatic activity. 7. The method of claim 1, wherein the incubating and detecting steps are performed in a well of a microtiter dish. 8. The method of claim 7, wherein the microtiter dish has 96 or 384 wells. 9. The method of claim 7, wherein a plurality of candidate compounds are screened in parallel with each other and steps (i) (ii) are repeated sequentially in a microtiter plate format, and wherein said plurality is between at least about 100 and at least about 6,000 different compounds. 10. The method of claim 7, wherein the incubating and detecting steps are performed in a high throughput integrated system comprising an automatic pipetting station, a robotic armature, and a robotic controller. 11. The method of claim 1, wherein the compound inhibits premature translation termination thereby increasing the amount of full-length polypeptide translated from the nucleic acid. 12. The method of claim 11, wherein the nucleic acid encodes an enzyme or an immunoglobulin. 13. The method of claim 11, wherein the nucleic acid encodes a luciferase, green fluorescent protein, red fluorescent protein, phosphatase, peroxidase, kinase, chloramphenicol transferase, or .beta.-Galactosidase. 14. The method of claim 11, wherein the nucleic acid encodes .alpha.-globin, .beta.-globin, factor VIII, factor IX, vWF, p53, dystrophin, CFTR, Rb, MSH1, MSH2, APC, Wt1, hexosaminidase A, neurofibromin 1, or neurofibromin 2. 15. The method of claim 1, wherein the nucleic acid encodes a chimeric polypeptide. 16. The method of claim 1, wherein the polypeptide contains a detectable moiety. 17. The method of claim 16, wherein the detectable moiety is a radiolabel. 18. The method of claim 1, wherein the polypeptide is detected using radioactivity.

Details for Patent 7,026,122

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Approval Date	Patent No.	Expiredate
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	For Injection	103132	06/04/1986		2019-12-14
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	For Injection	103132			2019-12-14
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	Injection	103132			2019-12-14