Ionizable Lipid Products

ARO-AAT is a second-generation RNAi drug. ARO-AAT consistes of a cholesterol-conjugated RNAi trigger (chol-RNAi) to selectively degrade AAT mRNA by RNAi and a melittin-derived peptide conjugated to N-acetylgalactosamine (NAG) formulated as the excipient EX1 to promote endosomal escape of the chol-RNAi in hepatocytes.

Tofersen (BIIB067) is an antisense oligonucleotide that mediates RNase H-dependent degradation of superoxide dismutase 1 (SOD1) mRNA to reduce the synthesis of SOD1 protein. Tofersen can be used for the research of amyotrophic lateral sclerosis (ALS).

Tivanisiran (SYL1001) is a siRNA used for the study of dry eye disease. Tivanisiran was designed to silence transient receptor potential vanilloid 1 (TRPV1).

Miravirsen (SPC-3649), a β-d-oxy-locked nucleic acid-modified phosphorothioate antisense oligonucleotide, inhibit the biogenesis of miR-122. Miravirsen (SPC-3649) is used in the study for HCV infections.

Nusinersen is an antisense oligonucleotide drug that modifies pre–messenger RNA splicing of the SMN2 gene and thus promotes increased production of full-length SMN protein.

DSG-PEG2K-triGalNAc is a functionalized PEG lipid that enhances targeted drug delivery by binding to the Asialoglycoprotein Receptor (ASGPR) which are predominantly found on the surface of liver cells. By functionalizing DSG-PEG2K with triGalNac, it allows this PEG lipid to be used in the formation of liposomes or LNPs that target the liver for drug delivery.

N7-Methyl-guanosine-5'-triphosphate-5'-adenosine (m7GpppA) is a dinucleotide cap analog that can be used for in vitro RNA transcription.

m7GpppAmpG Na salt is a trinucleotide 5′ cap analog with the capping efficiencies for the obtained RNAs of 90%.

m7GpppAmpG ammonium is a trinucleotide 5′ cap analog with the capping efficiencies for the obtained RNAs of 90%.

3'OMe-m7GpppAmpG ammonium solution (100mM) is a trinucleotide Cap analogue. 3'OMe-m7GpppAmpG ammonium shows a significant translational efficiency. 3'OMe-m7GpppAmpG ammonium can be used as a potential molecular biology tool in the field of mRNA vaccines and mRNA transfection, such as protein production, gene therapy and anti-cancer immunization.

SM-6.1 is a small molecule delivery vehicle developed by Arrowhead, targeting αvβ6, which selectively delivers therapeutic siRNA to lung epithelial cells and mediates durable gene silencing post-inhalation.

SM-6.1 is a small molecule delivery vehicle developed by Arrowhead, targeting αvβ6, which selectively delivers therapeutic siRNA to lung epithelial cells and mediates durable gene silencing post-inhalation.

6-[1-(Hexyloxy)ethoxy]hexanal is the tail fragement for MK16.MK16 is a novel blood-brain barrier (BBB)-crossing lipid nanoparticle (BLNP) platform developed for efficient mRNA delivery to the central nervous system (CNS).

Tail of CICL-1 lipid

Tail fragment of LP-01

10-Nitrooleic acid (CXA-10), a nitro fatty acid, has potential effects in disease states in which oxidative stress, inflammation, fibrosis, and/or direct tissue toxicity play significant roles.