220431-66-3

Upstream product

• 200133-20-6 di-tert-butyl 3,3'-((2-(((benzyloxy)carbonyl)amino)-2-((3-(tert-butoxy)-3-oxopropoxy)methyl)propane-1,3-diyl)bis(oxy))dipropionate 
• 175724-30-8 tris{[2-(tert-butoxycarbonyl)ethoxy]methyl}methylamine 
• 132491-89-5 tris(cyanoethoxymethyl)aminomethane 
• 132491-90-8 3-[2-Amino-3-(2-ethoxycarbonyl-ethoxy)-2-(2-ethoxycarbonyl-ethoxymethyl)-propoxy]-propionic acid ethyl ester 
• 501-53-1 benzyl chloroformate 
• 64-17-5 ethanol 
• 200133-16-0 benzyl N-tris[(2-carboxyethyloxy)methyl]methylcarbamate 

Downstream Products

• 132491-90-8 3-[2-Amino-3-(2-ethoxycarbonyl-ethoxy)-2-(2-ethoxycarbonyl-ethoxymethyl)-propoxy]-propionic acid ethyl ester 
• 200133-16-0 benzyl N-tris[(2-carboxyethyloxy)methyl]methylcarbamate 
• 849114-67-6 benzyl N-tris{[2-(2,3,4,5,6-pentachlorophenoxycarbonyl)ethoxy]methyl}methyl carbamate 
• 849114-68-7 benzyl N-tris{[2-[((N-hydroxy-N-methylcarbamoyl)methyl)aminocarbonyl]ethoxy]methyl}methylcarbamate 
• 490040-40-9 C₆₃H₉₈N₁₀O₂₀ 
• 1162069-31-9 3-[2-(benzyloxycarbonylamino)-3-(2-carboxyethoxy)-2-(2-carboxyethoxymethyl)-propoxy]propanoic acid 
• 1162069-33-1 C₃₀H₅₃N₇O₈ 

Relevant academic research and scientific papers

Self-association of protected Newkome-type second-generation dendrimers at nanomolar level concentrations in aqueous solution

Protected Newkome-type second-generation dendrimers (based on Lin's amine) were synthesized with a pyrene moiety attached to the core. The photophysical property in aqueous solution of the protected dendrimers shows self-association behavior in water. Pyr

Receptor-Specific Delivery of Peptide Nucleic Acids Conjugated to Three Sequentially Linked N-Acetyl Galactosamine Moieties into Hepatocytes

Peptide nucleic acids (PNAs) are DNA analogs that bind with high affinity to DNA and RNA in a sequence-specific manner but have poor cell permeability, limiting use as therapeutic agents. The work described here is motivated by recent reports of efficient gene silencing specifically in hepatocytes by small interfering RNAs conjugated to triantennary N-acetyl galactosamine (GalNAc), the ligand recognized by the asialoglycoprotein receptor (ASGPR). PNAs conjugated to either triantennary GalNAc at the N-terminus (the branched architecture) or monomeric GalNAc moieties anchored at Cγ of three consecutive PNA monomers of N-(2-aminoethyl)glycine (aeg) scaffolds (the sequential architecture) were synthesized on the solid phase. These formed duplexes with complementary DNA and RNA as shown by UV and circular dichroism spectroscopy. The fluorescently labeled analogs of GalNAc-conjugated PNAs were internalized by HepG2 cells that express the ASGPR but were not taken up by HEK-293 cells that lack this receptor. The sequential conjugate was internalized about 13-fold more efficiently than the branched conjugate into HepG2 cells, as demonstrated by confocal microscopy. The results presented here highlight the potential significance of the architecture of GalNAc conjugation for efficient uptake by target liver cells and indicate that GalNAc-conjugated PNAs have possible therapeutic applications.

TARGETED THERAPEUTIC NUCLEOSIDES AND THEIR USE

Provided herein are compounds comprising one or more therapeutic nucleosides and one or more targeting groups. In certain embodiments, the compounds further comprise one or more oligonucleotides. In certain embodiments, a targeting group comprises one or more N-Acetylgalactosamine.

COMPOSITIONS AND METHODS FOR ENHANCED INTESTINAL ABSORPTION OF CONJUGATED OLIGOMERIC COMPOUNDS

Provided herein are compositions and methods for non-parenteral delivery of conjugated oligomeric compounds. In certain embodiments, compositions and methods are provided for oral delivery of conjugated oligomeric compounds. In certain embodiments, the ol

COMPOSITIONS AND METHODS FOR MODULATING COMPLEMENT FACTOR B EXPRESSION

The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with dysregulation of the complement alternative pathway by administering a Complement Factor B (CFB) specific inhibitor to a subject.

COMPOSITIONS AND METHODS FOR MODULATING GROWTH HORMONE RECEPTOR EXPRESSION

The present embodiments provide methods, compounds, and compositions for treating, preventing, ameliorating a disease associated with excess growth hormone using antisense compounds oligonucleotides targeted to growth hormone receptor (GHR).

COMPOSITIONS AND METHODS FOR MODULATING ANGIOPOIETIN-LIKE 3 EXPRESSION

Provided herein are methods, compounds, and compositions for reducing expression of an ANGPTL3 mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for reducing lipids and/or glucose in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate any one or more of cardiovascular disease and/or metabolic disease, or a symptom thereof, in an individual in need thereof.

CONJUGATED ANTISENSE COMPOUNDS AND THEIR USE

Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.

COMPOSITIONS AND METHODS FOR MODULATION OF TARGET NUCLEIC ACIDS

The present disclosure pertains generally to chemically-modified oligonucleotides for use in research, diagnostics, and/or therapeutics. In certain embodiments, the present disclosure describes compounds and methods for the modulation of a target nucleic

Dendrimers as molecular translocators

Transport molecules include a dendrimer and a biologically active molecule. The dendrimer of such transport molecules includes at least one guanidine group, at least one protonated guanidine group, at least one protected guanidine group, at least one amid