DrugPatentWatch Database Preview➤ Get the DrugPatentWatch Daily Briefing
« Back to Dashboard
Summary for Patent: 10,023,867
|Title:||Oligonucleotides for modulating gene expression and uses thereof|
|Abstract:||The present invention regards oligonucleotides for modulating the expression of a gene, in particular for modulating a gene responsible for a pathology of genetic, tumoral or viral origin. Moreover, the present invention relates to the use of said oligonucleotides, possibly chemically modified, for the treatment and/or the diagnosis of said diseases.|
|Inventor(s):||Tonelli; Roberto (Granarolo Emilia, IT), Venturelli; Leonardo (Calderara di Reno, IT), Tortori; Andrea (Citerna, IT), Montemurro; Luca (Bologna, IT)|
|Assignee:||BIOGENERA S.P.A. (Porretta Terme (BO), IT)|
|Patent Claims:||1. A single strand anti-gene oligonucleotide complementary to the anti-sense DNA strand of a target gene, comprising 6-30 nucleotides, said oligonucleotides comprising
a sequence comprising at least three groups of at least two consecutive guanines, wherein said oligonucleotide is at least one molecule of natural nucleic acid or a synthetic nucleic acid or a combination of a natural nucleic acid and said synthetic
nucleic acid, wherein said at least one molecule of natural nucleic acid comprises at least one nucleotide modified at the level of the phosphodiester bond as a phosphorotioate bond.
2. The oligonucleotide according to claim 1, wherein the groups of at least two consecutive guanines are continuous with each other or spaced apart by at least one nucleotide, said nucleotide not being a guanine.
3. The oligonucleotide according to claim 1, wherein the groups of at least two consecutive guanines are at least four, five or six in number.
4. The oligonucleotide according to claim 1, wherein said oligonucleotide is conjugated with a carrier sequence at the 3' and/or 5' end of said oligonucleotide, said carrier being selected from the group consisting of: SEQ ID NO: 47-56.
5. The oligonucleotide according to claim 1, wherein said at least one molecule of natural nucleic or synthetic acid comprises at least one DNA, RNA LNA, methylphosphonate-LNA, BNA, RNG, DNG, GNA, UNA, ENA, ANA, F-ANA, PNA, G-PNA or morpholino nucleotide; or comprises a 2'0-Methyl, 2'0-Methoxyethyl or 2'-fluoro RNA nucleotide.
6. The oligonucleotide according to claim 5, wherein said PNA has a modified backbone wherein the alpha carbon has the side chain of arginine or lysine as a substituent.
7. The oligonucleotide according to claim 1, wherein said oligonucleotide is directed against at least one gene responsible for a disease of genetic or viral origin or against at least one tumor gene, wherein said gene is selected from the group consisting of at least one gene belonging to the MYC family.
8. The oligonucleotide according to claim 7, wherein said at least one gene is selected from the group consisting of MYC, MYCN or MYCL1, BIRC5, BCL2, PLK4, ALK, PKM2, CASP8, and RASSF1.
9. The oligonucleotide according to claim 1, wherein said oligonucleotide is selected among: SEQ ID NO: 2, 4, 5 6, 46 and SEQ ID NO: 68-84.
10. The oligonucleotide according to claim 9, wherein SEQ ID NO: 2, 4, 5, 6, 68 and 69 are directed against the MYCN gene.
11. The oligonucleotide according to claim 9, wherein SEQ ID NO: 70-74 are directed against the gene MYC, wherein SEQ ID NO: 75 and 76 are directed against the gene BIRC5; wherein the SEQ ID NO:77-79 are directed against the gene ALK; wherein SEQ ID NO: 80-82 are directed against the gene BCL2; wherein SEQ ID NO:83, 84 are directed against the gene PLK4.
12. A composition comprising at least one oligonucleotide according to claim 1 and at least one pharmacologically acceptable excipient.
13. A composition comprising at least one oligonucleotide according to claim 1 in combination with a compound with pharmacological action, selected from the group consisting of: NGF, somatostatin, retinoic acid, actinomycin D, asparaginase, bleomycin, busulphan capecitabine, carboplatin, cyclophosphamide, cyclosporine, cisplatin, cytarabine, clorambucil, dacarbazine, daunorubicin, docetaxel, doxorubicin hydrochloride, epirubicin hydrochloride, etoposide, fludarabine phosphate, fluorouracil, gemcitabine, idarubicin hydrochloride, hydroxyurea, ifophosphamide, irinotecan hydrochloride, melphalan, mercaptopurine, methotrexate, mitomycin, mitoxantrone, oxaliplatin, paclitaxel, procarbazine, raltitrexed, streptozocin, tegafururacil, temozolomide, thioguanine, thiotepe, topotecan, vinblastine, vincristine sulphate, vindesine and vinorelbine.
14. The composition according to claim 13, wherein said combination is: SEQ ID: NO 1 and carboplatin, or etoposide or cisplatin or vincristine; or SEQ ID: NO 5 and carboplatin, or etoposide or cisplatin or vincristine.
|Applicant||Tradename||Biologic Ingredient||Dosage Form||BLA||Number||Approval Date||Patent No.||Assignee||Estimated Patent Expiration||Status||Orphan||Source|
|Merck||ELSPAR||asparaginase||VIAL||101063||001||1978-01-10||⤷ Free Forever Trial||BIOGENERA S.P.A. (Porretta Terme (BO), IT)||2032-02-24||RX||search|
|>Applicant||>Tradename||>Biologic Ingredient||>Dosage Form||>BLA||>Number||>Approval Date||>Patent No.||>Assignee||>Estimated Patent Expiration||>Status||>Orphan||>Source|
Make Better Decisions: Try a trial or see plans & pricing
Serving leading biopharmaceutical companies globally:
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.