Patent application EP16305367A (EP), filed 2016-03-30
Publication number: EP3225257A1
Title: AMPHIPHILIC MONOMERS BASED NANOVECTORS AND THEIR USE FOR SIRNA DELIVERY
Abstract: Amphiphilic monomer of formula (I)   Li-CC-CC-L 2 -X-Z(I)  wherein L 1  is CH 3 (CH 2 ) m  , with m being an integer from 1 to 24, L 2  is -CH 2 (CH 2 ) n -CONH-, with n being an integer from 1 to 24, X is -(CH 2 ) t -O-(CH 2 CH 2 O) p (CH 2 ) t' -NH-, with t and t' being independently from each other 2 or 3 and p being an integer from 2 to 500 and Z is H or a positively charged group selected from natural or non natural amino acid residues, carboxylated polyamines residues, and peptides comprising from 2 to 5 amino acids, at least one of said amino-acids being an histidine residue, their physiologically acceptable salts and their use in transfection and delivery methods.
IPC codes: A61K  48/    00            A I
A61K   9/    51            A I
C12N  15/    88            A I
Claims: number: 1
text: Amphiphilic monomer of formula (I)
number: 2
text: Amphiphilic monomer of formula (I) according to claim 1 wherein Z is selected from:
number: 3
text: A polymerized micelle comprising a polymer obtainable by the photopolymerization of one monomer of formula (I) or of copolymers of monomers of formula (I) with the proviso that when there is only one monomer, then Z is not H.
number: 4
text: A polymerized fiber comprising a polymer obtainable by the photopolymerization of one monomer of formula (I) or of copolymers of monomers of formula (I).
number: 5
text: A transfection complex comprising a polymerized micelle according to claim 3 or a polymerized fiber according to claim 4 and at least one isolated nucleic acid.
number: 6
text: A transfection complex according to claim 5 wherein the isolated nucleic acid is a small interfering RNA.
number: 7
text: A pharmaceutical composition comprising a transfection complex according to any of claims 5 or 6 and a pharmaceutically acceptable excipient.
number: 8
text: An in vitro transfection or delivery method comprising the step of introducing a transfection complex according to any of claims 5 or 6 into a host cell.
number: 9
text: An in vitro transfection method according to claim 8 wherein said host cell is an eukaryotic cell.
number: 10
text: An in vitro transfection method according to claim 9 wherein said eukaryotic cell is a mammalian cell selected.
number: 11
text: An in vitro transfection method according to claim 10 wherein said mammalian cell is a human cell.
number: 12
text: Process for obtaining polymerized micelles according to claim 3 or polymerized fibers according to claim 4, characterized in that it comprises the following steps:
Office action: search_report, dated 2017-10-04
Rejection reasons: novelty_epc_54
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ref_id: - JENNY K.W. LAM ET AL: "Effective endogenous gene silencing mediated by pH responsive peptides proceeds via multiple pathways", JOURNAL OF CONTROLLED RELEASE, vol. 158, no. 2, 1 March 2012 (2012-03-01), pages 293 - 303, XP055132138, ISSN: 0168-3659, DOI: 10.1016/j.jconrel.2011.11.024
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ref_id: - SAMYN PIETER ET AL, "Colorimetric sensing properties of catechol-functional polymerized vesicles in aqueous solution and at solid surfaces", COLLOIDS AND SURFACES A: PHYSICACHEMICAL AND ENGINEERING ASPECTS, (20130913), vol. 441, doi:10.1016/J.COLSURFA.2013.09.003, ISSN 0927-7757, pages 242 - 254, XP028793750 [X] 1-3 * figure 1 *
ref_type: npl
severity: novelty_destroying
ref_id: - PATRICK NEUBERG ET AL, "Photopolymerized micelles of diacetylene amphiphile: physical characterization and cell delivery properties", CHEMICAL COMMUNICATIONS, (20150616), vol. 51, no. 58, doi:10.1039/C5CC03820K, ISSN 1359-7345, pages 11595 - 11598, XP055299310 [I] 1,3,12 * the whole document * * figure 1 *
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severity: background
ref_id: [ ]     - Patrick Neuberg ET AL, "Electronic Supplementary Information for Photopolymerized micelles of diacetylene amphiphile: physical characterization and cell delivery properties", (20150616), pages 1 - 10, URL: http://www.rsc.org/suppdata/c5/cc/c5cc03820k/c5cc03820k1.pdf, (20160831), XP055298856
ref_type: npl
severity: background
ref_id: - PATRICK MIDOUX ET AL, "Chemical vectors for gene delivery: a current review on polymers, peptides and lipids containing histidine or imidazole as nucleic acids carriers", BRITISH JOURNAL OF PHARMACOLOGY, vol. 157, doi:10.1111/J.1476-5381.2009.00288.X, ISSN 0007-1188, (20090101), pages 166 - 178, (20090428), XP007914499 [A] 1-12 * abstract * * table 2 *
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severity: background
ref_id: - JENNY K.W. LAM ET AL, "Effective endogenous gene silencing mediated by pH responsive peptides proceeds via multiple pathways", JOURNAL OF CONTROLLED RELEASE, (20120301), vol. 158, no. 2, doi:10.1016/j.jconrel.2011.11.024, ISSN 0168-3659, pages 293 - 303, XP055132138 [A] 1-12 * the whole document *
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severity: background
Final disposition: pending (2017-10-04)