Oligonucleotides are short DNA or RNA molecules, oligomers, that have a wide range of applications in genetic testing, research, and forensics. Commonly made in the laboratory by solid-phase chemical synthesis, these small fragments of nucleic acids can be manufactured as single-stranded molecules with any user-specified sequence, and so are vital for artificial gene synthesis, polymerase chain reaction (PCR), DNA sequencing, molecular cloning and as molecular probes. In nature, oligonucleotides are usually found as small RNA molecules that function in the regulation of gene expression, or are degradation intermediates derived from the breakdown of larger nucleic acid molecules.
Gene silencing is the regulation of gene expression in a cell to prevent the expression of a certain gene. Gene silencing can occur during either transcription or translation and is often used in research. In particular, methods used to silence genes are being increasingly used to produce therapeutics to combat cancer and other diseases, such as infectious diseases and neurodegenerative disorders.
The Takeda Pharmaceutical Company Limited is a Japanese multinational pharmaceutical company. It is the third largest pharmaceutical company in Asia, behind Sinopharm and Shanghai Pharmaceuticals, and one of the top 20 largest pharmaceutical companies in the world by revenue. The company has over 49,578 employees worldwide and achieved US$19.299 billion in revenue during the 2018 fiscal year. The company is focused on oncology, rare diseases, neuroscience, gastroenterology, plasma-derived therapies and vaccines. Its headquarters is located in Chuo-ku, Osaka, and it has an office in Nihonbashi, Chuo, Tokyo. In January 2012, Fortune Magazine ranked the Takeda Oncology Company as one of the 100 best companies to work for in the United States. As of 2015, Christophe Weber was appointed as the CEO and president of Takeda.
Small interfering RNA (siRNA), sometimes known as short interfering RNA or silencing RNA, is a class of double-stranded RNA at first non-coding RNA molecules, typically 20–24 base pairs in length, similar to miRNA, and operating within the RNA interference (RNAi) pathway. It interferes with the expression of specific genes with complementary nucleotide sequences by degrading mRNA after transcription, preventing translation. It was discovered in 1998, by Andrew Fire at Carnegie Institution for Science in Washington DC and Craig Mello at University of Massachusetts in Worcester.
Regeneron Pharmaceuticals, Inc. is an American biotechnology company headquartered in Westchester County, New York. The company was founded in 1988. Originally focused on neurotrophic factors and their regenerative capabilities, giving rise to its name, the company then branched out into the study of both cytokine and tyrosine kinase receptors, which gave rise to their first product, which is a VEGF-trap.
Craig Cameron Mello is an American biologist and professor of molecular medicine at the University of Massachusetts Medical School in Worcester, Massachusetts. He was awarded the 2006 Nobel Prize for Physiology or Medicine, along with Andrew Z. Fire, for the discovery of RNA interference. This research was conducted at the Carnegie Institution of Washington and published in 1998. Mello has been a Howard Hughes Medical Institute investigator since 2000.
RNA interference (RNAi) is a biological process in which RNA molecules are involved in sequence-specific suppression of gene expression by double-stranded RNA, through translational or transcriptional repression. Historically, RNAi was known by other names, including co-suppression, post-transcriptional gene silencing (PTGS), and quelling. The detailed study of each of these seemingly different processes elucidated that the identity of these phenomena were all actually RNAi. Andrew Fire and Craig C. Mello shared the 2006 Nobel Prize in Physiology or Medicine for their work on RNAi in the nematode worm Caenorhabditis elegans, which they published in 1998. Since the discovery of RNAi and its regulatory potentials, it has become evident that RNAi has immense potential in suppression of desired genes. RNAi is now known as precise, efficient, stable and better than antisense therapy for gene suppression. Antisense RNA produced intracellularly by an expression vector may be developed and find utility as novel therapeutic agents.
Galena Biopharma was a publicly traded pharmaceutical company based in San Ramon, California. The company was founded in Worcester, Massachusetts. In 2011, it moved to Oregon, and in 2015 moved to San Ramon, California. Mark Schwartz was the company's president and chief executive officer. As of December 29, 2017, the company was acquired by Sellas Life Sciences Group Ltd. through a reverse merger transaction. Galena Biopharma was renamed to Sellas Life Sciences Group, Inc.
Herpes simplex research includes all medical research that attempts to prevent, treat, or cure herpes, as well as fundamental research about the nature of herpes. Examples of particular herpes research include drug development, vaccines and genome editing. HSV-1 and HSV-2 are commonly thought of as oral and genital herpes respectively, but other members in the herpes family include chickenpox (varicella/zoster), cytomegalovirus, and Epstein-Barr virus. There are many more virus members that infect animals other than humans, some of which cause disease in companion animals or have economic impacts in the agriculture industry.
AbbVie Inc. is an American pharmaceutical company headquartered in North Chicago, Illinois. It is ranked sixth on the list of largest biomedical companies by revenue. In 2023, the company's seat in Forbes Global 2000 was 74. The company's primary product is Humira (adalimumab), administered via injection. It is approved to treat autoimmune diseases including rheumatoid arthritis, Crohn's disease, plaque psoriasis, and ulcerative colitis.
Alnylam Pharmaceuticals, Inc. is an American biopharmaceutical company focused on the discovery, development and commercialization of RNA interference (RNAi) therapeutics for genetically defined diseases. The company was founded in 2002 and is headquartered in Cambridge, Massachusetts. In 2016, Forbes included the company on its "100 Most Innovative Growth Companies" list.
Agios Pharmaceuticals Inc. is a publicly trading American pharmaceutical company pioneering therapies for genetically defined diseases, with a near-term focus on developing therapies for hemolytic anemias. The company was founded in 2008 by Lewis Cantley, Tak Mak and Craig Thompson. Agios is a Delaware corporation headquartered in Cambridge, Massachusetts. The company tendered an initial public offering in July 2013.
A deuterated drug is a small molecule medicinal product in which one or more of the hydrogen atoms in the drug molecule have been replaced by its heavier stable isotope deuterium. Because of the kinetic isotope effect, deuterium-containing drugs may have significantly lower rates of metabolism, and hence a longer half-life.
Molecular Partners AG is a clinical-stage biopharmaceutical company based in Zürich, Switzerland. The company is developing a new class of potent, specific and versatile small-protein therapies called DARPins, with potential clinical applications in a range of disease areas including oncology, immuno-oncology, ophthalmology, and infectious diseases. Molecular Partners currently has two DARPin molecules in clinical development, and a broad pipeline of molecules in preclinical development.
Arrowhead Pharmaceuticals is a publicly traded biopharmaceutical company based in Pasadena, California. Arrowhead’s products in development act through RNA interference (RNAi) mechanisms of action. The company focuses on treatments for hepatitis B, liver disease associated with alpha 1-antitrypsin deficiency and cardiovascular disease. The company has sixteen products in its pipeline, in various stages of development.
Inclisiran, sold under the brand name Leqvio, is a medication used for the treatment of high low-density lipoprotein (LDL) cholesterol and for the treatment of people with atherosclerotic cardiovascular disease (ASCVD), ASCVD risk-equivalents, and heterozygous familial hypercholesterolemia (HeFH). It is a small interfering RNA (siRNA) that acts as an inhibitor of a proprotein convertase, specifically, inhibiting translation of the protein PCSK9.
Phillip D. Zamore is an American molecular biologist and biochemist who co-developed the first in vitro system for studying the mechanism of RNA interference (RNAi). He is the Gretchen Stone Cook Professor of Biomedical Sciences at University of Massachusetts Chan Medical School, Worcester, Massachusetts. Zamore is chair of the RNA Therapeutics Institute (RTI), established in 2009, and has been a Howard Hughes Medical Institute Investigator since 2008.
RNA therapeutics are a new class of medications based on ribonucleic acid (RNA). Research has been working on clinical use since the 1990s, with significant success in cancer therapy in the early 2010s. In 2020 and 2021, mRNA vaccines have been developed globally for use in combating the coronavirus disease. The Pfizer–BioNTech COVID-19 vaccine was the first mRNA vaccine approved by a medicines regulator, followed by the Moderna COVID-19 vaccine, and others.
Marcin Kortylewski is a Polish American cancer researcher and immunologist. He is currently professor of immuno-oncology at the Beckman Research Institute of the City of Hope National Medical Center in Duarte, California. His research has shown that the STAT3 protein plays a role in protecting cancers from immune responses and contributes to resistance to therapies. Later he developed a two-pronged strategy for cancer immunotherapy using simultaneous STAT3 inhibition and TLR9 immune stimulation. Kortylewski invented platform strategy for delivery of oligonucleotides, such as siRNA, miRNA, decoy DNA, antisense molecules and others to selected immune cells.
