US2023174985A1PendingUtilityA1
Modified peptide nucleic acid compositions
Est. expiryMar 30, 2040(~13.7 yrs left)· nominal 20-yr term from priority
A61K 9/08A61K 9/127G01N 33/6848A61K 9/0085A61P 25/28A61K 9/0019A61K 9/10A61K 9/4841C07K 14/003G01N 30/7233A61K 9/2004C12N 2310/3181C12N 2320/34C07K 5/0821C12N 2310/11A61K 47/645A61K 9/0053A61K 9/06A61K 51/0491A61K 38/00A61K 47/02G01N 33/6896A61K 47/12A61K 47/542C12N 15/113A61K 9/107A61K 9/0095
62
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Claims
Abstract
The present disclosure relates to compounds useful for the detection or modulation of target nucleic acids, including DNA and RNA. The present disclosure further relates to methods for treatment of trinucleotide repeat disorders, which can include administration of oligonucleotide analogues that can bind pathogenic nucleotide repeats in DNA or RNA.
Claims
exact text as granted — not AI-modified1 - 121 . (canceled)
122 . A compound having the formula (I):
wherein:
each B is independently a nucleobase;
each R 1 is independently a side chain of a natural amino acid, a guanidino(C 1 -C 4 )alkyl, or hydrogen;
each R 2 is independently a side chain of a natural amino acid, a guanidino(C 1 -C 4 )alkyl, or hydrogen;
R 5 is a sequence comprising at least one alpha amino acid residue, beta amino acid residue, gamma amino acid residue, or a combination thereof; hydrogen;
or a water solubilizing group;
n is an integer from 3-30; and
G is OH, NH 2 , or
wherein:
R 3 is hydrogen or an amino(C 1 -C 4 )alkyl;
R 4 is a sequence comprising at least one alpha amino acid residue, beta amino acid residue, gamma amino acid residue, or a combination thereof;
or hydrogen; and
m is 0 or 1;
wherein:
the compound comprises at least one guanine-cytosine-thymine sequence; and at least one R 1 is guanidino(C 1 -C 4 )alkyl, or at least one R 2 is guanidino(C 1 -C 4 )alkyl;
or a pharmaceutically-acceptable salt thereof
123 . The compound of claim 122 , wherein each B is independently guanine, thymine, or cytosine.
124 - 129 . (canceled)
130 . The compound of claim 122 , wherein G is
131 . The compound of claim 130 , wherein R 3 is 4-aminobut-1-yl.
132 . (canceled)
133 . The compound of claim 130 , wherein m is 0.
134 . (canceled)
135 . The compound of claim 130 , wherein R 4 is hydrogen.
136 . The compound of claim 130 , wherein R 4 is a sequence comprising at least one alpha amino acid residue.
137 . The compound of claim 122 , wherein R 4 is
wherein
p is 1, 2, 3, 4, 5, 6, 7, 8, 9, or 10; and
each R 6 is independently hydrogen or an amino(C 1 -C 4 )alkyl.
138 . The compound of claim 137 , wherein R 4 is
139 - 140 . (canceled)
141 . The compound of claim 122 , wherein R 5 is hydrogen.
142 . The compound of claim 122 , wherein R 5 is
wherein
each R 7 is independently a side chain of a natural amino acid; and
q is 0 or 1.
143 . The compound of claim 142 , wherein R 5 is
144 . The compound of claim 142 , wherein R 5 is
145 - 147 . (canceled)
148 . The compound of claim 122 , wherein, when G is OH or NH 2 , at least one of R 1 and R 2 is a side chain of a natural amino acid or a guanidino(C 1 -C 4 )alkyl, and R 5 is not hydrogen.
149 . The compound of claim 122 , which has the formula:
150 - 159 . (canceled)
160 . The compound of claim 149 , which has the formula:
wherein:
each B 1a , B 2a , and B 3a is independently cytosine, guanine, or thymine; and
n 1 is 1, 2, 3, 4, 5, 6, 7, 8, 9, or 10.
161 - 162 . (canceled)
163 . The compound of claim 160 , which has the formula:
164 . The compound of claim 163 , wherein B 1a is cytosine, B 2a is thymine and B 3a is guanine.
165 . The compound of claim 164 , wherein n 1 is 2.
166 . (canceled)
167 . The compound of claim 149 , which has the formula:
168 . (canceled)
169 . The compound of claim 167 , which has the formula:
wherein:
each B 1b , B 2b and B 3b is independently cytosine, guanine or thymine; and
n 2 is 1, 2, 3, 4, 5, 6, 7, 8, 9, or 10.
170 - 171 . (canceled)
172 . The compound of claim 169 , which has the formula:
173 - 175 . (canceled)
176 . The compound of claim 149 , which has the formula:
wherein
p is 1, 2, 3, 4, 5, 6, 7, 8, 9, or 10; and
each R 6 is independently hydrogen or an amino(C 1 -C 4 )alkyl.
177 - 195 . (canceled)
196 . The compound of claim 176 , which has the formula
wherein R 7 is a side chain of a natural amino acid.
197 - 213 . (canceled)
214 . The compound of claim 149 , which has the formula:
wherein R 7 is a side chain of a natural amino acid.
215 - 216 . (canceled)
217 . The compound of claim 214 , which has the structure
wherein:
X is;
Y is
each B 1d , B 2d , B 3d , B 4d , B 5d , B 6d , B 7d , B 8d , B 9d , and B 10d is independently cytosine, guanine or thymine;
n 4 is 1, 2, 3, 4, 5, 6, 7, 8, 9, or 10; and
n 5 is 1, 2, 3, 4, 5, 6, 7, 8, 9, or 10.
218 - 235 . (canceled)
236 . The compound of claim 217 , which has the structure:
wherein:
X 1 is
and
y 1 is
237 . The compound of claim 236 , which has the structure
X 2 is
and
y 2 is
238 . The compound of claim 214 , which has the structure
wherein:
L is
M is
each B 1e , B 2e , B 3e , B 4e , B 5e , B 6e , B 7e , B 8e , B 9e and B 10e is independently cytosine, guanine or thymine;
n 6 is 1, 2, 3, 4, 5, 6, 7, 8, 9, or 10; and
n 7 is 1, 2, 3, 4, 5, 6, 7, 8, 9, or 10.
239 - 258 . (canceled)
259 . The compound of claim 238 , which has the structure
wherein:
L 1 is
and
M 1 is
260 . (canceled)Cited by (0)
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