Title:
FORMULATION FOR ADMINISTRATION OF RNA
Document Type and Number:
WIPO Patent Application WO/2019/137999
Kind Code:
A1
Abstract:
The present invention relates to compositions comprising polyplex formulations for delivery of RNA to a target organ or a target cell after parenteral administration, in particular after intramuscular administration. More precisely, the present invention relates to formulations for administration of RNA such as self-replicating RNA, in particular by intramuscular injection. In more detail, the formulations comprise polyplex particles from single stranded RNA and a polyalkyleneimine.
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Inventors:
SAHIN UGUR (DE)
HAAS HEINRICH (DE)
VOGEL ANNETTE (DE)
ERBAR STEPHANIE (DE)
WALZER KERSTIN (DE)
SCHLEGEL ANNE (DE)
HÖRNER SEBASTIAN (DE)
MORENO HERRERO JORGE (DE)
KLAMP THORSTEN (DE)
KREITER SEBASTIAN (DE)
DIKEN MUSTAFA (DE)
HAAS HEINRICH (DE)
VOGEL ANNETTE (DE)
ERBAR STEPHANIE (DE)
WALZER KERSTIN (DE)
SCHLEGEL ANNE (DE)
HÖRNER SEBASTIAN (DE)
MORENO HERRERO JORGE (DE)
KLAMP THORSTEN (DE)
KREITER SEBASTIAN (DE)
DIKEN MUSTAFA (DE)
Application Number:
PCT/EP2019/050551
Publication Date:
July 18, 2019
Filing Date:
January 10, 2019
Export Citation:
Assignee:
BIONTECH RNA PHARMACEUTICALS GMBH (DE)
TRON TRANSLATIONALE ONKOLOGIE AN DER UNIV DER JOHANNES GUTENBERG UNIV MAINZ GGMBH (DE)
TRON TRANSLATIONALE ONKOLOGIE AN DER UNIV DER JOHANNES GUTENBERG UNIV MAINZ GGMBH (DE)
International Classes:
A61K31/7105; A61K9/00; A61K39/12; C12N15/87
Domestic Patent References:
| WO2008119827A1 | 2008-10-09 | |||
| WO2000053780A2 | 2000-09-14 | |||
| WO2014071963A1 | 2014-05-15 |
Foreign References:
| EP3034539A1 | 2016-06-22 | |||
| US67131208A | 2008-07-31 |
Other References:
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S. SAJEESH ET AL: "Long dsRNA-Mediated RNA Interference and Immunostimulation: A Targeted Delivery Approach Using Polyethyleneimine Based Nano-Carriers", MOLECULAR PHARMACEUTICS, vol. 11, no. 3, 3 March 2014 (2014-03-03), US, pages 872 - 884, XP055330364, ISSN: 1543-8384, DOI: 10.1021/mp400541z
JESSICA PAHLE ET AL: "Vectors and strategies for nonviral cancer gene therapy", EXPERT OPINION ON BIOLOGICAL THERAPY, vol. 16, no. 4, 13 January 2016 (2016-01-13), ASHLEY, LONDON; GB, pages 443 - 461, XP055330366, ISSN: 1471-2598, DOI: 10.1517/14712598.2016.1134480
TUBULEKAS ET AL., GENE, vol. 190, 1997, pages 191 - 195
"Helvetica Chimica Acta", 1995, article "A multilingual glossary of biotechnological terms: (IUPAC Recommendations"
"Molecular Cloning: A Laboratory Manual", 1989, COLD SPRING HARBOR LABORATORY PRESS
KOPPEL, D., J. CHEM. PHYS., vol. 57, 1972, pages 4814 - 4820
JOSE ET AL., FUTURE MICROBIOL., vol. 4, 2009, pages 837 - 856
GOULD ET AL., ANTIVIRAL RES., vol. 87, 2010, pages 111 - 124
STRAUSS & STRAUSS, MICROBIOL. REV., vol. 58, 1994, pages 491 - 562
RUPP ET AL., J. GEN. VIROLOGY, vol. 96, 2015, pages 2483 - 2500
SHIRAKO & STRAUSS, J. VIROL., vol. 68, 1994, pages 1874 - 1885
KIM ET AL., VIROLOGY, vol. 323, 2004, pages 153 - 163
VASILJEVA ET AL., J. BIOL. CHEM., vol. 278, 2003, pages 41636 - 41645
PETTERSSON ET AL., EUR. J. BIOCHEM., vol. 105, 1980, pages 435 - 443
ROZANOV ET AL., J. GEN. VIROLOGY, vol. 73, 1992, pages 2129 - 2134
RUBACH ET AL., VIROLOGY, vol. 384, 2009, pages 201 - 208
WHITE ET AL., J. VIROL., vol. 72, 1998, pages 4320 - 4326
FROLOV ET AL., RNA, vol. 7, 2001, pages 1638 - 1651
FROLOV, RNA, vol. 7, 2001, pages 1638 - 1651
MCKINNEY ET AL., AM. J. TROP. MED. HYG., vol. 12, 1963, pages 597 - 603
HARDY; RICE, J. VIROL., vol. 79, 2005, pages 4630 - 4639
SMITH; WATERMAN, ADS APP. MATH., vol. 2, 1981, pages 482
NEEDLEMAN; WUNSCH, J. MOL. BIOL., vol. 48, 1970, pages 443
PEARSON; LIPMAN, PROC. NATL ACAD. SCI. USA, vol. 85, 1988, pages 2444
"Molecular Cloning: A Laboratory Manual", 1989, COLD SPRING HARBOR LABORATORY PRESS
"Current Protocols in Molecular Biology", JOHN WILEY & SONS, INC.
RUPP ET AL., J. GEN. VIROL., vol. 96, pages 2483 - 500
"Remington's Pharmaceutical Sciences", 1985, MACK PUBLISHING CO.
THOMAS ET AL.: "Polyethylenimine-based polyplex delivery of self-replicating RNA vaccines", NANOMEDICINE: NANOTECHNOLOGY, BIOLOGY AND MEDICINE, 2015
S. SAJEESH ET AL: "Long dsRNA-Mediated RNA Interference and Immunostimulation: A Targeted Delivery Approach Using Polyethyleneimine Based Nano-Carriers", MOLECULAR PHARMACEUTICS, vol. 11, no. 3, 3 March 2014 (2014-03-03), US, pages 872 - 884, XP055330364, ISSN: 1543-8384, DOI: 10.1021/mp400541z
JESSICA PAHLE ET AL: "Vectors and strategies for nonviral cancer gene therapy", EXPERT OPINION ON BIOLOGICAL THERAPY, vol. 16, no. 4, 13 January 2016 (2016-01-13), ASHLEY, LONDON; GB, pages 443 - 461, XP055330366, ISSN: 1471-2598, DOI: 10.1517/14712598.2016.1134480
TUBULEKAS ET AL., GENE, vol. 190, 1997, pages 191 - 195
"Helvetica Chimica Acta", 1995, article "A multilingual glossary of biotechnological terms: (IUPAC Recommendations"
"Molecular Cloning: A Laboratory Manual", 1989, COLD SPRING HARBOR LABORATORY PRESS
KOPPEL, D., J. CHEM. PHYS., vol. 57, 1972, pages 4814 - 4820
JOSE ET AL., FUTURE MICROBIOL., vol. 4, 2009, pages 837 - 856
GOULD ET AL., ANTIVIRAL RES., vol. 87, 2010, pages 111 - 124
STRAUSS & STRAUSS, MICROBIOL. REV., vol. 58, 1994, pages 491 - 562
RUPP ET AL., J. GEN. VIROLOGY, vol. 96, 2015, pages 2483 - 2500
SHIRAKO & STRAUSS, J. VIROL., vol. 68, 1994, pages 1874 - 1885
KIM ET AL., VIROLOGY, vol. 323, 2004, pages 153 - 163
VASILJEVA ET AL., J. BIOL. CHEM., vol. 278, 2003, pages 41636 - 41645
PETTERSSON ET AL., EUR. J. BIOCHEM., vol. 105, 1980, pages 435 - 443
ROZANOV ET AL., J. GEN. VIROLOGY, vol. 73, 1992, pages 2129 - 2134
RUBACH ET AL., VIROLOGY, vol. 384, 2009, pages 201 - 208
WHITE ET AL., J. VIROL., vol. 72, 1998, pages 4320 - 4326
FROLOV ET AL., RNA, vol. 7, 2001, pages 1638 - 1651
FROLOV, RNA, vol. 7, 2001, pages 1638 - 1651
MCKINNEY ET AL., AM. J. TROP. MED. HYG., vol. 12, 1963, pages 597 - 603
HARDY; RICE, J. VIROL., vol. 79, 2005, pages 4630 - 4639
SMITH; WATERMAN, ADS APP. MATH., vol. 2, 1981, pages 482
NEEDLEMAN; WUNSCH, J. MOL. BIOL., vol. 48, 1970, pages 443
PEARSON; LIPMAN, PROC. NATL ACAD. SCI. USA, vol. 85, 1988, pages 2444
"Molecular Cloning: A Laboratory Manual", 1989, COLD SPRING HARBOR LABORATORY PRESS
"Current Protocols in Molecular Biology", JOHN WILEY & SONS, INC.
RUPP ET AL., J. GEN. VIROL., vol. 96, pages 2483 - 500
"Remington's Pharmaceutical Sciences", 1985, MACK PUBLISHING CO.
THOMAS ET AL.: "Polyethylenimine-based polyplex delivery of self-replicating RNA vaccines", NANOMEDICINE: NANOTECHNOLOGY, BIOLOGY AND MEDICINE, 2015
Attorney, Agent or Firm:
SCHNAPPAUF, Georg (DE)
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