US6022874A — Piperazine based cytofectins

Claim 1

Independent Claim 406 words

A piperazine based cytofectin of the following structure: ##STR8## wherein R 1 and R 4 are independently H, linear C 1 -C 23 groups, or branched C 1 -C 23 groups, said linear or branched C 1 -C 23 groups being alkyl, acyl, alkene, or heteroalkyl groups, said alkyl, acyl, alkene or heteroalkyl groups being unsubstituted or substituted, having 0 to 6 sites of unsaturation, containing 0 to 5 heteroatoms, or containing cyclic or aryl groups, said cyclic or aryl groups containing 0-5 heteroatoms; R 2 is a linear C 1 -C 23 group, or a branched C 1 -C 23 group, said linear or branched C 1 -C 23 groups being alkyl, acyl, alkene or heteroalkyl groups, said alkyl, acyl, alkene or heteroalkyl groups being unsubstituted or substituted, having 0 to 6 sites of unsaturation, containing 0 to 5 heteroatoms, or containing cyclic or aryl groups, said cyclic or aryl groups containing 0-5 heteroatoms; R 3 is H, an amino acid, peptide, polypeptide, protein, nucleic acid, nucleotide, nucleoside, polynucleotide, polynucleoside, monosaccharide, disaccharide, polysaccharide, bioactive agent, pharmaceutical agent, a linear C 1 -C 23 group, or a branched C 1 -C 23 group, said linear or branched C 1 -C 23 groups being alkyl, acyl, alkene, or heteroalkyl groups, said alkyl, acyl, alkene or heteroalkyl groups being unsubstituted or substituted, having 0 to 6 sites of unsaturation, containing 0 to 5 heteroatoms, or containing cyclic or aryl groups, said cyclic or aryl groups containing 0-5 heteroatoms, or a group having the following structure: ##STR9## wherein R 7 through R 12 are independently absent, H, an amino acid, nucleotide, polynucleotide, monosaccharide, disaccharide, polysaccharide, bioactive agent, pharmaceutical agent, linear C 1 -C 23 groups, or branched C 1 -C 23 groups, said linear or branched C 1 -C 23 groups being alkyl, acyl, alkene or heteroalkyl groups, said alkyl, acyl, alkene or heteroalkyl groups being unsubstituted or substituted, having 0 to 6 sites of unsaturation, containing 0 to 5 heteroatoms, or containing cyclic or aryl groups, said cyclic or aryl groups containing 0 to 5 heteroatoms; G is absent, O, N, NH, S, SH, Se, C, CH, or CR 1 ; T is O, N, S, Se, or C; A is O, N, S, Se, or C; Y 1 and Y 2 are independently O, N, NH, S, or Se; X 1 is O or NH; m and p are independently 1-6; W is a pharmaceutically acceptable anion.

Claim 2

Dependent Claim 406 words

A piperazine based cytofectin of the following structure: ##STR8## wherein R 1 and R 4 are independently H, linear C 1 -C 23 groups, or branched C 1 -C 23 groups, said linear or branched C 1 -C 23 groups being alkyl, acyl, alkene, or heteroalkyl groups, said alkyl, acyl, alkene or heteroalkyl groups being unsubstituted or substituted, having 0 to 6 sites of unsaturation, containing 0 to 5 heteroatoms, or containing cyclic or aryl groups, said cyclic or aryl groups containing 0-5 heteroatoms; R 2 is a linear C 1 -C 23 group, or a branched C 1 -C 23 group, said linear or branched C 1 -C 23 groups being alkyl, acyl, alkene or heteroalkyl groups, said alkyl, acyl, alkene or heteroalkyl groups being unsubstituted or substituted, having 0 to 6 sites of unsaturation, containing 0 to 5 heteroatoms, or containing cyclic or aryl groups, said cyclic or aryl groups containing 0-5 heteroatoms; R 3 is H, an amino acid, peptide, polypeptide, protein, nucleic acid, nucleotide, nucleoside, polynucleotide, polynucleoside, monosaccharide, disaccharide, polysaccharide, bioactive agent, pharmaceutical agent, a linear C 1 -C 23 group, or a branched C 1 -C 23 group, said linear or branched C 1 -C 23 groups being alkyl, acyl, alkene, or heteroalkyl groups, said alkyl, acyl, alkene or heteroalkyl groups being unsubstituted or substituted, having 0 to 6 sites of unsaturation, containing 0 to 5 heteroatoms, or containing cyclic or aryl groups, said cyclic or aryl groups containing 0-5 heteroatoms, or a group having the following structure: ##STR9## wherein R 7 through R 12 are independently absent, H, an amino acid, nucleotide, polynucleotide, monosaccharide, disaccharide, polysaccharide, bioactive agent, pharmaceutical agent, linear C 1 -C 23 groups, or branched C 1 -C 23 groups, said linear or branched C 1 -C 23 groups being alkyl, acyl, alkene or heteroalkyl groups, said alkyl, acyl, alkene or heteroalkyl groups being unsubstituted or substituted, having 0 to 6 sites of unsaturation, containing 0 to 5 heteroatoms, or containing cyclic or aryl groups, said cyclic or aryl groups containing 0 to 5 heteroatoms; G is absent, O, N, NH, S, SH, Se, C, CH, or CR 1 ; T is O, N, S, Se, or C; A is O, N, S, Se, or C; Y 1 and Y 2 are independently O, N, NH, S, or Se; X 1 is O or NH; m and p are independently 1-6; W is a pharmaceutically acceptable anion.

Claim 3

Dependent Claim 13 words

The piperazine based cytofectins of claim 1 wherein m and p are 2.

Claim 4

Dependent Claim 15 words

The piperazine based cytofectins of claim 2 wherein Y 1 and Y 2 are O.

Claim 5

Dependent Claim 36 words

The piperazine based cytofectins of claim 3 wherein R 1 and R 4 are selected from the group consisting of C 10 H 21 , C 12 H 25 , and C 14 H 29 .

Claim 6

Dependent Claim 45 words

The piperazine based cytofectins of claim 4 wherein R 2 is selected from the group consisting of absent, CH 2 , (CH 2 ) 2 , (CH 2 ) 3 , (CH 2 ) 4 ,(CH 2 ) 5 , (CH 2 ) 6 .

Claim 7

Dependent Claim 378 words

A piperazine based cytofectin of the following structure: ##STR10## wherein R 1 , R 2 , R 4 , and R 5 are independently H, linear C 1 -C 23 groups, or branched C 1 -C 23 groups, said linear or branched C 1 -C 23 groups being alkyl, acyl, alkene or heteroalkyl groups, said alkyl, acyl, alkene, or heteroalkyl groups being unsubstituted or substituted, having 0 to 6 sites of unsaturation, containing 0 to 5 heteroatoms, or containing cyclic or aryl groups, said cyclic or aryl groups containing 0-5 heteroatoms; R 3 and R 6 are each independently H, an amino acid, peptide, polypeptide, protein, nucleic acid, nucleotide, nucleoside, polynucleotide, polynucleoside, monosaccharide, disaccharide, polysaccharide, bioactive agent, pharmaceutical agent, a linear C 1 -C 23 group, or a branched C 1 -C 23 group, said linear or branched C 1 -C 23 groups being alkyl, acyl, alkene or heteroalkyl groups, said alkyl, acyl, alkene or heteroalkyl groups being unsubstituted or substituted, having 0 to 6 sites of unsaturation, containing 0 to 5 heteroatoms, or containing cyclic or aryl groups, said cyclic or aryl groups containing 0-5 heteroatoms, or a group having the following structure: ##STR11## wherein R 7 through R 12 are independently absent, H, an amino acid, nucleotide, polynucleotide, monosaccharide, disaccharide, polysaccharide, bioactive agent, pharmaceutical agent, linear C 1 -C 23 groups, or branched C 1 -C 23 groups, said linear or branched C 1 -C 23 groups being alkyl, acyl, alkene, or heteroalkyl groups, said alkyl, acyl, alkene, or heteroalkyl groups being substituted or unsubstituted, having 0 to 6 sites of unsaturation, containing from 0 to 5 heteroatoms, or containing cyclic or aryl groups, said cyclic or aryl groups containing 0 to 5 heteroatoms; G is absent, O, N, NH, S, SH, Se, C, CH, or CR 1 ; T is O, N, S, Se, or C; A is O, N, S, Se, or C; Y 1 and Y 2 are independently O, N, NH, S, or Se; X 1 and X 2 are independently selected from the group consisting of absent, O, and NH; m and p are independently 1-6; W is a pharmaceutically acceptable anion; and wherein either X 1 or X 2 is present or both X 1 and X 2 are present.

Claim 8

Dependent Claim 378 words

A piperazine based cytofectin of the following structure: ##STR10## wherein R 1 , R 2 , R 4 , and R 5 are independently H, linear C 1 -C 23 groups, or branched C 1 -C 23 groups, said linear or branched C 1 -C 23 groups being alkyl, acyl, alkene or heteroalkyl groups, said alkyl, acyl, alkene, or heteroalkyl groups being unsubstituted or substituted, having 0 to 6 sites of unsaturation, containing 0 to 5 heteroatoms, or containing cyclic or aryl groups, said cyclic or aryl groups containing 0-5 heteroatoms; R 3 and R 6 are each independently H, an amino acid, peptide, polypeptide, protein, nucleic acid, nucleotide, nucleoside, polynucleotide, polynucleoside, monosaccharide, disaccharide, polysaccharide, bioactive agent, pharmaceutical agent, a linear C 1 -C 23 group, or a branched C 1 -C 23 group, said linear or branched C 1 -C 23 groups being alkyl, acyl, alkene or heteroalkyl groups, said alkyl, acyl, alkene or heteroalkyl groups being unsubstituted or substituted, having 0 to 6 sites of unsaturation, containing 0 to 5 heteroatoms, or containing cyclic or aryl groups, said cyclic or aryl groups containing 0-5 heteroatoms, or a group having the following structure: ##STR11## wherein R 7 through R 12 are independently absent, H, an amino acid, nucleotide, polynucleotide, monosaccharide, disaccharide, polysaccharide, bioactive agent, pharmaceutical agent, linear C 1 -C 23 groups, or branched C 1 -C 23 groups, said linear or branched C 1 -C 23 groups being alkyl, acyl, alkene, or heteroalkyl groups, said alkyl, acyl, alkene, or heteroalkyl groups being substituted or unsubstituted, having 0 to 6 sites of unsaturation, containing from 0 to 5 heteroatoms, or containing cyclic or aryl groups, said cyclic or aryl groups containing 0 to 5 heteroatoms; G is absent, O, N, NH, S, SH, Se, C, CH, or CR 1 ; T is O, N, S, Se, or C; A is O, N, S, Se, or C; Y 1 and Y 2 are independently O, N, NH, S, or Se; X 1 and X 2 are independently selected from the group consisting of absent, O, and NH; m and p are independently 1-6; W is a pharmaceutically acceptable anion; and wherein either X 1 or X 2 is present or both X 1 and X 2 are present.

Claim 9

Dependent Claim 16 words

The piperazine based cytofectins of claim 6, wherein both X 1 and X 2 are present.

Claim 10

Dependent Claim 19 words

The piperazine based cytofectins of claim 6, wherein either X 1 or X 2 but not both is present.

Claim 11

Dependent Claim 13 words

The piperazine based cytofectins of claim 6 wherein m and p are 2.

Claim 12

Dependent Claim 15 words

The piperazine based cytofectins of claim 9 wherein Y 1 and Y 2 are O.

Claim 13

Dependent Claim 48 words

The piperazine based cytofectins of claim 10, wherein R 5 and R 2 are selected from the group consisting of absent, CH 2 , (CH 2 ) 2 , (CH 2 ) 3 , (CH 2 ) 4 ,(CH 2 ) 5 and (CH 2 ) 6 .

Claim 14

Dependent Claim 36 words

The piperazine based cytofectins of claim 11 wherein R 1 and R 4 are selected from the group consisting of C 10 H 21 , C 12 H 25 , and C 14 H 29 .

Claim 15

Dependent Claim 55 words

A method of deleivering a molecule into a cell comprising the steps of: (a) contacting the moleucle with an effective amount of a cytofectin according to claim 1 to form a complex with the cytofectin; and (b) contacting the cell with the complex formed in step (a) whereby said molecule is delivered into said cell.

Claim 16

Dependent Claim 12 words

The method of claim 13, wherein said molecule is an anionic molecule.

Claim 17

Dependent Claim 19 words

The method of claim 14, wherein said anionic molecule is selected from the group consisting of mRNA and DNA.

Claim 18

Dependent Claim 21 words

The method of claim 13, wherein said complex includes one or more lipids in addition to said cytofectins of claim 1.

Claim 19

Dependent Claim 20 words

The method of claim 16, wherein said lipids are selected from the group consisting of neutral lipids, phospholipids and cholesterol.

Claim 20

Dependent Claim 55 words

A method of deleivering a molecule into a cell comprising the steps of: (a) contacting the moleucle with an effective amount of a cytofectin according to claim 6 to form a complex with the cytofectin; and (b) contacting the cell with the complex formed in step (a) whereby said molecule is delivered into said cell.

Claim 21

Dependent Claim 12 words

The method of claim 18, wherein said molecule is an anionic molecule.

Claim 22

Dependent Claim 19 words

The method of claim 19, wherein said anionic molecule is selected from the group consisting of mRNA and DNA.

Claim 23

Dependent Claim 21 words

The method of claim 18, wherein said complex includes one or more lipids in addition to said cytofectins of claim 1.

Claim 24

Dependent Claim 20 words

The method of claim 21, wherein said lipids are selected from the group consisting of neutral lipids, phospholipids and cholesterol.

Annuity Stage	Window Opens	Payment Deadline	Surcharge Period Until	Estimated Fee	Status
3.5 Years (Year 4)	2003-02-07	2003-08-09	2004-02-08	$2000.00	Window Closed / Lapsed
7.5 Years (Year 8)	2007-02-07	2007-08-09	2008-02-08	$3760.00	Window Closed / Lapsed
11.5 Years (Year 12)	2011-02-07	2011-08-09	2012-02-08	$7700.00	Window Closed / Lapsed

Piperazine based cytofectins

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