Sirna Therapeutics, Inc. was a San Francisco, California based biotechnology company that explored the use of RNA interference in human disease therapy. Sirna's development pipeline includes several small interfering RNA (siRNA) drugs, thought to stably silence the expression of specific disease-related genes. Sirna's clinical trial of an siRNA-based treatment for age-related macular degeneration is the first such trial for an siRNA drug. [1] [2]
Sirna Therapeutics was acquired by Merck & Co. in 2006, the cash transaction deal was worth $1.1 billion. [3]
Small interfering ribonucleic acid
Sirna maintained its laboratory on the northeastern edge of Boulder, Colorado. [4] Originally the corporate headquarters were also located in Boulder. Sirna, at one point, launched a search for a new headquarters complex. It originally considered multiple cities, including Cambridge, Massachusetts and San Diego, California.
Sirna selected San Francisco due to its proximity to various scientific institutions, its workforce, and the payroll tax exemption for biotechnology companies. Sirna's move was a part of a trend of biotechnology companies moving to San Francisco, bolstered due to a campaign to attract biotechnology companies to San Francisco and tax breaks instituted by Mayor of San Francisco Gavin Newsom. Business leaders of San Francisco who hoped that the city would have a major biotechnology presence had a positive reception to the Sirna decision. [5]
The mayor's office, the Chamber of Commerce, and the San Francisco Center for Economic Development contributed to bringing Sirna to San Francisco. Howard Robin, the president and chief executive officer of Sirna, said that the first wave of moves would be the ten highest ranking employees; he did not say how many employees would relocate to San Francisco. The company planned to maintain a facility in Boulder. [5] Ultimately Sirna occupied Suite 6504 at China Basin Landing, while the laboratory facilities remained in Boulder, with administrative, manufacturing, and research facilities. [6]
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.
Small interfering RNA (siRNA), sometimes known as short interfering RNA or silencing RNA, is a class of double-stranded RNA non-coding RNA molecules, typically 20-27 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.
Gilead Sciences, Inc., is an American biopharmaceutical company headquartered in Foster City, California that researches, develops and commercializes drugs. The company focuses primarily on antiviral drugs used in the treatment of HIV, hepatitis B, hepatitis C, and influenza, including Harvoni and Sovaldi.
Sarepta Therapeutics, Inc. is a medical research and drug development company with corporate offices and research facilities in Cambridge, Massachusetts, United States. Incorporated in 1980 as AntiVirals, shortly before going public the company changed its name from AntiVirals to AVI BioPharma soon with stock symbol AVII and in July 2012 changed name from AVI BioPharma to Sarepta Therapeutics and SRPT respectively. As of the end of 2019, the company has two approved drugs.
MilliporeSigma is a German chemical, life science and biotechnology company, before 2014 known as Sigma-Aldrich, owned by Merck KGaA.
Celgene Corporation is a pharmaceutical company that makes cancer and immunology drugs. Its major product is Revlimid (lenalidomide), which is used in the treatment of multiple myeloma, and also in certain anemias. The company is incorporated in Delaware, headquartered in Summit, New Jersey, and a subsidiary of Bristol Myers Squibb (BMS).
Hubert Jacob Paul Schoemaker was a Dutch biotechnologist. He was a co-founder and the president of one of America's first biotechnology companies, Centocor, which was founded in 1979 for the commercialising of monoclonal antibodies. In 1999 he founded Neuronyx, Inc., for the manufacture of stem cells and the development of stem-cell therapies.
RNA silencing or RNA interference refers to a family of gene silencing effects by which gene expression is negatively regulated by non-coding RNAs such as microRNAs. RNA silencing may also be defined as sequence-specific regulation of gene expression triggered by double-stranded RNA (dsRNA). RNA silencing mechanisms are highly conserved in most eukaryotes. The most common and well-studied example is RNA interference (RNAi), in which endogenously expressed microRNA (miRNA) or exogenously derived small interfering RNA (siRNA) induces the degradation of complementary messenger RNA. Other classes of small RNA have been identified, including piwi-interacting RNA (piRNA) and its subspecies repeat associated small interfering RNA (rasiRNA).
Quark Pharmaceuticals is a pharmaceutical company that develops RNA interference-based treatments for chronic and acute diseases.
Nuvelo Inc. was a biopharmaceutical company engaged in the discovery, development and commercialization of drugs for acute cardiovascular disease, cancer and other debilitating medical conditions. On January 27, 2009, the company was acquired by ARCA Biopharma, Inc. in a reverse takeover transaction.
RNA interference (RNAi) is a biological process in which RNA molecules inhibit gene expression or translation, by neutralizing targeted mRNA molecules. 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 RNA interference 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. However, antisense RNA produced intracellularly by an expression vector may be developed and find utility as novel therapeutic agents.
Merck & Co., Inc., d.b.a. Merck Sharp & Dohme (MSD) outside the United States and Canada, is an American multinational pharmaceutical company and one of the largest pharmaceutical companies in the world. Merck is incorporated in New Jersey.
Regulus Therapeutics Inc. or Regulus is a clinical stage biopharmaceutical company focused on the development of first-in-class drugs that target microRNAs to treat a broad range of diseases. Regulus was established in September 2007 by Alnylam Pharmaceuticals and Isis Pharmaceuticals.
Silence Therapeutics (AIM:SLN) is a biotechnology company that develops gene therapeutic technology based on RNA interference (RNAi). They are involved in developmental research of targeted RNAi therapeutics for the treatment of serious diseases. Formed in 1994 as a publicly traded drug research and development company, Silence Therapeutics has offices in London and Berlin, with its corporate headquarters in Hammersmith, London.
AbbVie is an American publicly traded biopharmaceutical company founded in 2013. It originated as a spin-off of Abbott Laboratories.
Alnylam Pharmaceuticals Inc. is a 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.
Nektar Therapeutics (Nektar) is an American biopharmaceutical company. The company was founded in 1990 and is based in San Francisco, California. The company develops new drug candidates by applying its proprietary PEGylation and advanced polymer conjugate technologies to modify chemical structure of substances. It is a technology supplier to a number of pharmaceutical companies including Affymax, Amgen, Merck, Pfizer and UCB Pharma, etc. The company developed the world's first inhalable non-injectable insulin, Exubera, which was awarded as the bronze award by Wall Street Journal for its technological breakthrough.
Arcturus Therapeutics is an American biotech company focused on the discovery, development and commercialization of therapeutics for rare diseases with focus on RNA. Arcturus has developed a novel, potent and safe RNA therapeutics platform called LUNAR, a proprietary lipid-enabled delivery system for RNA medicines including small interfering RNA, messenger RNA, gene editing, DNA, antisense, and microRNA oligotherapeutics. The company's pipeline includes RNA therapeutics towards rare diseases such as ornithine transcarbamylase deficiency (OTCD), and respiratory diseases such as cystic fibrosis. Vaccine medicines include a vaccine candidate for COVID-19.
Ionis Pharmaceuticals is a biotechnology company based in Carlsbad, California that specializes in discovering and developing RNA-targeted therapeutics. The company has 3 commercially approved medicines: Spinraza (Nusinersen), Tegsedi (Inotersen), and WAYLIVRA and has 4 drugs in pivotal studies: tominersen for Huntington’s disease, tofersen for SOD1-ALS, AKCEA-APO(a)-LRx for cardiovascular disease, and AKCEA-TTR-LRx for all forms of TTR amyloidosis.