Patent ID: 8349308

Claim:
A modified polyacetal of Formula (VI): wherein: each of W 1 , W 2 , W 3 , W 4 , W 5 and W 6 , independently, is a covalent bond or —C(O)—Y— with —C(O) connected to the polyacetal backbone; Y is —[C(R 9 R 10 )] a — or —[C(R 9 R 10 )] a —X 1 —[C(R 9 R 10 )] b —; X 1 is an oxygen atom, a sulfur atom or —NR 11 ; each of R 9 and R 10 independently is hydrogen, C 1-6 alkyl, C 6-10 aryl, 5 to 12-membered heteroaryl or C 3-8 cycloalkyl; R 11 is hydrogen, C 1-6 alkyl, C 6-10 aryl, 5 to 12-membered heteroaryl, C 3-8 cycloalkyl or —C(O)—C 1-3 alkyl; Z 9 is Z 6 -T 1 or Z 8 ; T 1 is —Z 7 —R 6 ; Z 8 is a linear or branched polyamino moiety substituted with one or more —Z 7 —R 6 and optionally substituted with one or more substituents selected from the group consisting of -Q-R 1 , -Q-R 3 , -Q-R 4 , and -Q-R 5 ; each Q independently is a covalent bond or —C(O)—; each of Z 1 , Z 2 , Z 3 , Z 4 , Z 5 , and Z 6 , independently, is a covalent bond, —NR 17 , or —NR 17 R 18 —, in which each of R 17 and R 18 independently is H, C 2-8 alkyl, or —C 2-10 alkyl-N(R x )—, R x being H or an amino acid attached to the nitrogen via the carbonyl group of the amino acid; or R 17 and R 18 , together with the nitrogen atom to which they are attached form a 4 to 7-membered heterocycloalkyl ring containing 0 or 1 additional heteroatom selected from N, O, and S; each Z 7 independently is —C(O)-T 2 -T 3 - or —N(R′)-T 2 -T 3 - with T 3 connected to R 6 , in which R′ is H or C 1-6 alkyl, T 2 is selected from alkylthioaryl, arylthioalkyl, alkylthioalkyl, arylthioaryl, alkyldithioaryl, aryldithioalkyl, alkyldithioalkyl and aryldithioaryl, and T 3 is a covalent bond, —C(O)N(R″)—C 1-8 alkyl, —N(R″)C(O)—C 1-8 alkyl, or C 1-8 alkyl, in which R″ is H or C 1-6 alkyl; each of a and b independently is an integer between 1 and 6 inclusive; each of n, n 1 , n 2 , n 3 , n 4 , n 5 , and n 6 is the molar fraction of the corresponding polyacetal unit ranging between 0 and 1; n+n 1 +n 2 +n 3 +n 4 +n 5 +n 6 =1; provided that neither n nor n 6 is 0; R 1 is a targeting group for a selected tissue, pathogen, cell, or cellular location; R 2 is a charge group optionally substituted with one or more substituents selected from the group consisting of -Q-R 1 , -Q-R 3 , -Q-R 4 , and -Q-R 5 ; R 3 is a charge-modifying group; R 4 is a hydrophobic group; R 5 is a protective group; R 6 is a polynucleotide; the ratio (m 1 ) of the number of R 1 to the total number of polyacetal units of the polyacetal is 0 to 0.25; the ratio (m 3 ) of the number of R 3 to the total number of polyacetal units of the polyacetal is 0 to 100; the ratio (m 4 ) of the number of R 4 to the total number of polyacetal units of the polyacetal is 0 to 30; the ratio (m 5 ) of the number of R 5 to the total number of polyacetal units of the polyacetal is 0 to 0.03; the ratio (m 6 ) of the number of R 6 to the total number of polyacetal units of the polyacetal is 0.0004 to 0.10; and the polyacetal backbone has a molecular weight of about 10 kDa to about 250 kDa.