PEGylation is the process of both covalent and non-covalent attachment or amalgamation of polyethylene glycol polymer chains to molecules and macrostructures, such as a drug, therapeutic protein or vesicle, which is then described as PEGylated. PEGylation affects the resulting derivatives or aggregates interactions, which typically slows down their coalescence and degradation as well as elimination in vivo.
Lipid nanoparticles (LNPs) are nanoparticles composed of lipids. They are a novel pharmaceutical drug delivery system, and a novel pharmaceutical formulation. LNPs as a drug delivery vehicle were first approved in 2018 for the siRNA drug Onpattro. LNPs became more widely known in late 2020, as some COVID-19 vaccines that use RNA vaccine technology coat the fragile mRNA strands with PEGylated lipid nanoparticles as their delivery vehicle.
Arcturus Therapeutics is an American RNA medicines biotechnology company focused on the discovery, development and commercialization of therapeutics for rare diseases and infectious diseases. Arcturus has developed a novel, potent, and safe RNA therapeutics platform called Lunar, a proprietary lipid-enabled delivery system for nucleic acid medicines including small interfering RNA (siRNA), messenger RNA (mRNA), gene editing RNA, DNA, antisense oligonucleotides (ASO), and microRNA.
An mRNAvaccine is a type of vaccine that uses a copy of a molecule called messenger RNA (mRNA) to produce an immune response. The vaccine delivers molecules of antigen-encoding mRNA into immune cells, which use the designed mRNA as a blueprint to build foreign protein that would normally be produced by a pathogen or by a cancer cell. These protein molecules stimulate an adaptive immune response that teaches the body to identify and destroy the corresponding pathogen or cancer cells. The mRNA is delivered by a co-formulation of the RNA encapsulated in lipid nanoparticles that protect the RNA strands and help their absorption into the cells.
A COVID‑19 vaccine is a vaccine intended to provide acquired immunity against severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), the virus that causes coronavirus disease 2019 (COVID‑19).
The Moderna COVID‑19 vaccine, sold under the brand name Spikevax, is a COVID-19 vaccine developed by American company Moderna, the United States National Institute of Allergy and Infectious Diseases (NIAID), and the Biomedical Advanced Research and Development Authority (BARDA). Depending on the jurisdiction, it is authorized for use in people aged six months, twelve years, or eighteen years and older. It provides protection against COVID-19 which is caused by infection by the SARS-CoV-2 virus. It is designed to be administered as two or three 0.5 mL doses given by intramuscular injection at an interval of at least 28 days apart.
Katalin Karikó is a Hungarian-American biochemist who specializes in RNA-mediated mechanisms. Her research has been the development of in vitro-transcribed mRNA for protein therapies. She co-founded and was CEO of RNARx, from 2006 to 2013. Since 2013, she has been associated with BioNTech RNA Pharmaceuticals, first as a vice president and promoted to senior vice president in 2019. She also is an adjunct professor at the University of Pennsylvania.
The Pfizer–BioNTech COVID-19 vaccine, sold under the brand name Comirnaty, is an mRNA-based COVID-19 vaccine developed by the German biotechnology company BioNTech. For its development, BioNTech collaborated with American company Pfizer to carry out clinical trials, logistics, and manufacturing. It is authorized for use in people to provide protection against COVID-19, caused by infection with the SARS-CoV-2 virus. The vaccine is given by intramuscular injection. It is composed of nucleoside-modified mRNA (modRNA) encoding a mutated form of the full-length spike protein of SARS-CoV-2, which is encapsulated in lipid nanoparticles. Initial advice indicated that vaccination required two doses given 21 days apart, but the interval was later extended to up to 42 days in the US, and up to four months in Canada.
A nucleoside-modified messenger RNA (modRNA) is a synthetic messenger RNA (mRNA) in which some nucleosides are replaced by other naturally modified nucleosides or by synthetic nucleoside analogues. modRNA is used to induce the production of a desired protein in certain cells. An important application is the development of mRNA vaccines, of which the first authorized were COVID-19 vaccines.
ALC-0315 is a synthetic lipid. A colorless oily material, it has attracted attention as a component of the SARS-CoV-2 vaccine, BNT162b2, from BioNTech and Pfizer. Specifically, it is one of four components that form lipid nanoparticles (LNPs), which encapsulate and protect the otherwise fragile mRNA that is the active ingredient in these drugs. These nanoparticles promote the uptake of therapeutically effective nucleic acids such as oligonucleotides or mRNA both in vitro and in vivo.
Distearoylphosphatidylcholine is a phosphatidylcholine, a kind of phospholipid. It is a natural constituent of cell membranes, eg. soybean phosphatidylcholines are mostly different 18-carbon phosphatidylcholines, and their hydrogenation results in 85% DSPC. It can be used to prepare lipid nanoparticles which are used in mRNA vaccines, In particular, it forms part of the drug delivery system for the Moderna and Pfizer COVID-19 vaccines.
ALC-0159 is a PEG/lipid conjugate, specifically, it is the N,N-dimyristylamide of 2-hydroxyacetic acid, O-pegylated to a PEG chain mass of about 2 kilodaltons. It is a non-ionic surfactant by its nature. It has been deployed in the Pfizer-BioNTech SARS-CoV-2 mRNA vaccine that contains the active ingredient tozinameran.
SM-102 is a synthetic amino lipid which is used in combination with other lipids to form lipid nanoparticles. These are used for the delivery of mRNA-based vaccines, and in particular SM-102 forms part of the drug delivery system for the Moderna COVID-19 vaccine.
The CureVac COVID-19 vaccine was a COVID-19 vaccine candidate developed by CureVac N.V. and the Coalition for Epidemic Preparedness Innovations (CEPI). The vaccine showed inadequate results in its Phase III trials with only 47% efficacy. In October 2021 CureVac abandoned further development and production plans for CVnCoV and refocused efforts on a cooperation with GlaxoSmithKline.
AWcorna, originally termed ARCoV and also known as the Walvax COVID-19 vaccine, is an mRNA COVID-19 vaccine developed by Walvax Biotechnology, Suzhou Abogen Biosciences, and the PLA Academy of Military Science. In contrast to other mRNA COVID vaccines, such as those by Pfizer-BioNtech and Moderna, this vaccine primarily targets the Sars-CoV-2 receptor-binding domain of the spike protein, rather than the entire spike protein. It is approved for Phase III trials in China, Mexico, Indonesia, and Nepal.
mRNA-1283 is a COVID-19 vaccine candidate developed by Moderna.
Pieter Rutter Cullis is a Canadian physicist and biochemist known for his contributions to the field of lipid nanoparticles (LNP). Lipid nanoparticles are essential to current mRNA vaccines as a delivery system. Prof. Cullis is best known for the development of ionizable cationic lipids. These lipids are able to complex with negatively charged nucleic acids at low pH (≈4.0) where they are positively charged because they have a pKa if approximately 6.4. They reduce or eliminate toxicity associated with cationic lipids at physiological pH of 7.4 because they adopt a net neutral charge. Finally, they enable endosomal escape because they again become positively charged in acidified endosomes and promote formation of non-bilayer structures by interaction with negatively charged lipids. These properties are critical to the function of the mRNA vaccines and are rapidly enabling gene therapy in clinical settings.
Intracellular delivery is the process of introducing external materials into living cells. Materials that are delivered into cells include nucleic acids, proteins, peptides, impermeable small molecules, synthetic nanomaterials, organelles, and micron-scale tracers, devices and objects. Such molecules and materials can be used to investigate cellular behavior, engineer cell operations or correct a pathological function.
Acuitas Therapeutics Inc. is a Canadian biotechnology company based in Vancouver, British Columbia. The company was established in February 2009 to specialize in the development of delivery systems for nucleic acid therapeutics based on lipid nanoparticle (LNP) technology, a key component of the mRNA vaccines deployed for COVID-19.
