Nonviral vectors for gene therapy

Patent metadata

Field	Value
Publication number	US-2025084414-A1
Application number	US-202318845379-A
Country	US
Kind code	A1
Filing date	Mar 6, 2023
Priority date	Mar 9, 2022
Publication date	Mar 13, 2025
Grant date	—

Abstract

The present invention relates to fluorinated amphiphilic dendrimer structures (FJDs) capable of self-assembling into supramolecular systems of different size and shape. A further object is a supramolecular complex comprising at least one of the dendrimer structures according to the present invention and the use thereof in gene therapy.

First claim

1 . Fluorinated dendrimeric structure of Formula (I)

Wherein
n is an integer between 1 and 5;
R is selected from

R 1 is selected from

X is selected independently from:
OH; (6)
COOH (7)
NH 2 (8)
NH 3 + Y − (9)
NR 2 2 (10)
NR 2 3 + Y − (11)

Wherein
R 2 is selected independently from —CH 3 , —CH 2 CH 3 , —CH 2 CH 2 CH 3 , —CH(CH 3 ) 2 , —CH 2 OH, —CH 2 CH 2 OH;
Y − is selected from:

2 . The structure according to claim 1 , wherein n is 2 or 3.

3 . The structure according to claim 1 , wherein R is

4 . The structure according to claim 1 , wherein R 1 is

5 . The structure according to claim 1 , wherein X=NH 3+ Y − and Y is selected from 6 .

6 . The structure according to claim 1 , wherein n=3, R is (2), R1 is (5), X=NH 3 + Y − , Y is (12) and it is FDG 2 N.

7 . A supramolecular complex comprising at least one of the dendrimeric structures according to claim 1 and one or more nucleic acids.

8 . The complex according to claim 7 , wherein the ratio between the nitrogen atoms of said dendrimeric structure and the phosphorus atoms of said one or more nucleic acids (N/P) is comprised between 5 and 40.

9 . The complex according to claim 7 , wherein N/P=30.

10 . A supramolecular complex according to claim 7 , for use in gene therapy.

Assignees

• Milano Politecnico
• Fondazione Irccs St Neurologico Carlo Besta

Inventors

• Baldelli Francesca
• Metrangolo Pierangelo
• Cavallo Gabriella
• Bono Nina
• Candiani Gabriele
• Rosati Marta
• Pinter Lauria Giuseppe
• Marcuzzo Stefania

Classifications

• C12N2320/32

  Special delivery means, e.g. tissue-specific · CPC title

• C12N2310/351

  Conjugate · CPC title

• C12N2310/141

  MicroRNAs, miRNAs · CPC title

• C08G83/002

  Dendritic macromolecules · CPC title

• A61K48/005

  characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered · CPC title