|
Pelacarsen
Pelacarsen is an antisense therapy that is designed to reduce Lipoprotein(a) concentrations in people with high levels of the lipoprotein and who are at risk of cardiovascular disease. It was developed by Ionis Pharmaceuticals and Novartis Novartis AG is a Swiss multinational corporation, multinational pharmaceutical company, pharmaceutical corporation based in Basel, Switzerland. Novartis is one of the largest pharmaceutical companies in the world and was the eighth largest by re .... References Antisense RNA Drugs developed by Novartis {{pharma-stub ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Lipoprotein(a)
Lipoprotein(a) is a low-density lipoprotein variant containing a protein called apolipoprotein(a). Genetic and epidemiological studies have identified lipoprotein(a) as a risk factor for atherosclerosis and related diseases, such as coronary heart disease and stroke. Lipoprotein(a) was discovered in 1963 by Kåre Berg. The human gene encoding apolipoprotein(a) was successfully cloned in 1987. Structure Lipoprotein(a) p(a)consists of an LDL-like particle and the specific apolipoprotein(a), which is bound covalently to the apoB contained in the outer shell of the particle. Lp(a) plasma concentrations are highly heritable and mainly controlled by the ''LPA'' gene located on chromosome 6q25.3–q26. Apo(a) proteins vary in size due to a size polymorphism VNTR which is caused by a variable number of kringle domain">kringle IV repeats in the ''LPA'' gene. This size variation at the gene level is expressed on the protein level as well, resulting in apo(a) proteins with 10 to ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Antisense Therapy
Antisense therapy is a form of treatment that uses antisense oligonucleotides (ASOs) to target messenger RNA (mRNA). ASOs are capable of altering mRNA expression through a variety of mechanisms, including ribonuclease H mediated decay of the pre-mRNA, direct steric blockage, and exon content modulation through Splicing (genetics), splicing site binding on pre-mRNA. Several ASOs have been approved in the United States, the European Union, and elsewhere. Nomenclature The common stem for antisense oligonucleotides drugs is -rsen. The substem -virsen designates antiviral antisense oligonucleotides. Antisense Oligonucleotide Development Developments in ASO modification are separated into three generations. Generation one is called backbone-modified and focuses on the phosphodiester group of the nucleotide. This impacts inter-nucleotide binding. These modifications led to better distribution, reduced urinary excretion, and prolonged residence time of the ASOs in the cell. Some exam ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Cardiovascular Disease
Cardiovascular disease (CVD) is any disease involving the heart or blood vessels. CVDs constitute a class of diseases that includes: coronary artery diseases (e.g. angina, heart attack), heart failure, hypertensive heart disease, rheumatic heart disease, cardiomyopathy, arrhythmia, congenital heart disease, valvular heart disease, carditis, aortic aneurysms, peripheral artery disease, thromboembolic disease, and venous thrombosis. The underlying mechanisms vary depending on the disease. It is estimated that dietary risk factors are associated with 53% of CVD deaths. Coronary artery disease, stroke, and peripheral artery disease involve atherosclerosis. This may be caused by high blood pressure, smoking, diabetes mellitus, lack of exercise, obesity, high blood cholesterol, poor diet, excessive alcohol consumption, and poor sleep, among other things. High blood pressure is estimated to account for approximately 13% of CVD deaths, while tobacco accounts for 9%, di ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Ionis Pharmaceuticals
Ionis Pharmaceuticals, Inc. is a biotechnology company based in Carlsbad, California, that specializes in discovering and developing RNA-targeted therapeutics. The company has three commercially approved medicines: Spinraza ( Nusinersen), Tegsedi ( Inotersen), and Waylivra ( Volanesorsen), and has four drugs in pivotal studies: tominersen for Huntington's disease (together with Roche), tofersen for SOD1- ALS, AKCEA-APO(a)-LRx for cardiovascular disease, and AKCEA-TTR-LRx for all forms of TTR amyloidosis. The company was named Isis Pharmaceuticals until December 2015. History The company was founded in 1989 as Isis Pharmaceuticals by Stanley T. Crooke, a former head of research of GlaxoSmithKline, with the goal of commercializing antisense therapy. In 1992, the company received its first approval by the Food and Drug Administration for an investigational new drug application in 1992 for a genital warts drug candidate. The FDA approval marked the first time for the company t ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Novartis
Novartis AG is a Swiss multinational corporation, multinational pharmaceutical company, pharmaceutical corporation based in Basel, Switzerland. Novartis is one of the largest pharmaceutical companies in the world and was the eighth largest by revenue in 2024. Novartis manufactures the drugs clozapine (Clozaril), diclofenac (Voltaren; sold to GlaxoSmithKline in 2015 deal), carbamazepine (Tegretol), valsartan (Diovan), imatinib mesylate (Gleevec/Glivec), cyclosporine (Neoral/Sandimmune), letrozole (Femara), methylphenidate (Ritalin; produced by Sandoz since 2023), terbinafine (Lamisil), deferasirox (Exjade), and others. Novartis was formed in 1996 by the merger of Ciba-Geigy and Sandoz. It was considered the largest corporate merger in history during that time. The pharmaceutical and agrochemical divisions of both companies formed Novartis as an independent entity. The name Novartis was based on the Latin terms, ''novae artes'' (new skills). After the merger, other Ciba-Geigy and ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Antisense RNA
Antisense RNA (asRNA), also referred to as antisense transcript, natural antisense transcript (NAT) or antisense oligonucleotide, is a single stranded RNA that is complementary to a protein coding messenger RNA (mRNA) with which it hybridizes, and thereby blocks its Translation (biology), translation into protein. The asRNAs (which occur naturally) have been found in both prokaryotes and eukaryotes, and can be classified into short (200 nucleotides) non-coding RNAs (ncRNAs). The primary function of asRNA is regulating gene expression. asRNAs may also be produced synthetically and have found wide spread use as research tools for gene knockdown. They may also have therapeutic applications. Discovery and history in drug development Some of the earliest asRNAs were discovered while investigating functional proteins. An example was MicF RNA, micF asRNA. While characterizing the outer membrane Porin (protein), porin in Escherichia coli, ''E.coli'', some of the promoter clones observ ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
