You’re using a public version of DrugPatentWatch with 5 free searches available | Register to unlock more free searches. CREATE FREE ACCOUNT

Last Updated: March 29, 2024

Claims for Patent: 9,267,154


✉ Email this page to a colleague

« Back to Dashboard


Summary for Patent: 9,267,154
Title:Targeted integration into the PPP1R12C locus
Abstract: Disclosed herein are methods and compositions for targeted integration of an exogenous sequence into the human PPP1R12C locus, for example, for expression of a polypeptide of interest.
Inventor(s): DeKelver; Russell (Richmond, CA), Gregory; Philip D. (Richmond, CA), Paschon; David (Richmond, CA), Tam; Phillip (Richmond, CA), Urnov; Fyodor (Richmond, CA)
Assignee: Sangamo BioSciences, Inc. (Richmond, CA)
Application Number:14/447,378
Patent Claims:1. An isolated genetically modified cell comprising an exogenous polynucleotide integrated into an endogenous PPP1R12C (AAVS1) gene using at least one nuclease, wherein the exogenous polynucleotide is integrated into intron 1 of the endogenous AAVS1 gene and further wherein the exogenous polynucleotide does not comprise a wild-type AAV gene.

2. The cell of claim 1, wherein the wherein the one or more nucleases target SEQ ID NO:1 or SEQ ID NO:2.

3. The cell of claim 1, wherein the exogenous polynucleotide encodes a polypeptide.

4. The cell of claim 1, wherein the exogenous polynucleotide comprises an exogenous promoter.

5. The cell of claim 1, wherein expression of the exogenous polynucleotide is driven by an endogenous promoter.

6. The cell of claim 3, wherein the polypeptide is selected from the group consisting of an antibody, an antigen, an enzyme, a growth factor, a cell surface receptor, a nuclear receptor, a hormone, a lymphokine, a cytokine, a reporter, functional fragments thereof and combinations thereof.

7. The cell of claim 6, wherein the reporter comprises GFP.

8. The cell of claim 1, where the exogenous polynucleotide encodes a RNA, and further wherein the RNA is selected from the group consisting of one or more shRNAs, one or more RNAi molecules, one or more miRNAs, one or more mRNAs, and combinations thereof.

9. The cell of claim 1, wherein the exogenous polynucleotide further comprises a first nucleotide sequence that is homologous to a first sequence in the AAVS1 gene.

10. The cell of claim 9, wherein the exogenous polynucleotide further comprises a second nucleotide sequence that is homologous to a second sequence in the AAVS1 gene.

11. The cell of claim 9, wherein the exogenous polynucleotide comprises a tandem cassette.

12. The cell of claim 1, wherein the exogenous polynucleotide is a plasmid.

13. The cell of claim 1, wherein the polynucleotide is a linear DNA molecule.

14. The cell of claim 1, wherein the at least one nuclease comprises one or more cleavage half-domains are from a Type IIS restriction endonuclease.

15. The cell of claim 14, wherein the Type IIS restriction endonuclease is selected from the group consisting of FokI and StsI.

16. The cell of claim 15, wherein at least one of the cleavage half-domains comprises an alteration in the amino acid sequence of the dimerization interface.

17. The cell of claim 1, wherein the cell is a stem cell.

18. The cell of claim 17, wherein the cell is a hematopoietic stem cell.

19. The cell of claim 1, where the cell is a cell line.

Details for Patent 9,267,154

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 ⤷  Try a Trial 2027-04-26
Merck Sharp & Dohme Corp. INTRON A interferon alfa-2b For Injection 103132 ⤷  Try a Trial 2027-04-26
Merck Sharp & Dohme Corp. INTRON A interferon alfa-2b Injection 103132 ⤷  Try a Trial 2027-04-26
>Applicant >Tradename >Biologic Ingredient >Dosage Form >BLA >Approval Date >Patent No. >Expiredate

Make Better Decisions: Try a trial or see plans & pricing

Drugs may be covered by multiple patents or regulatory protections. All trademarks and applicant names are the property of their respective owners or licensors. Although great care is taken in the proper and correct provision of this service, thinkBiotech LLC does not accept any responsibility for possible consequences of errors or omissions in the provided data. The data presented herein is for information purposes only. There is no warranty that the data contained herein is error free. thinkBiotech performs no independent verification of facts as provided by public sources nor are attempts made to provide legal or investing advice. Any reliance on data provided herein is done solely at the discretion of the user. Users of this service are advised to seek professional advice and independent confirmation before considering acting on any of the provided information. thinkBiotech LLC reserves the right to amend, extend or withdraw any part or all of the offered service without notice.