Pharmaceutical Companies Developing RNA Therapies

   Alnylam Pharmaceuticals (Nasdaq: ALNY)

Alnylam Pharmaceuticals, Inc., a biopharmaceutical company, focuses on discovering, developing, and commercializing RNA interference (RNAi) therapeutics. The company's pipeline of investigational RNAi therapeutics focuses on genetic medicines, cardio-metabolic diseases, hepatic infectious diseases, and central nervous system/ocular diseases. 


Arbutus Biopharma (Nasdaq: ABUS)

Arbutus Biopharma Corporation is a publicly-traded (Nasdaq: ABUS) biopharmaceutical company primarily dedicated to discovering, developing, and commercializing a cure for people with chronic hepatitis B virus (HBV) infection. The Company is advancing multiple drug product candidates that may be combined into a potentially curative regimen for chronic HBV infection. Arbutus has also initiated drug discovery and development efforts for treating coronaviruses (including COVID-19). 

Arrowhead Pharmaceuticals (Nasdaq: ARWR)

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.

Benitec Biopharma Ltd. (Nasdaq: BNTC)

Benitec is a biotechnology company developing a proprietary therapeutic technology platform that combines RNA interference with gene therapy for the goal of providing sustained, long-lasting silencing of disease-causing genes from a single administration. Benitec utilizes a technology, called DNA-directed RNA interference, or ddRNAi, to develop product candidates in chronic and life-threatening human diseases.

Bio-Path Holdings Inc.(Nasdaq: BPHT)

Bio-Path Holdings, Inc. operates as a clinical and preclinical stage oncology-focused RNAi nano particle drug development company in the United States. The company develops products based on DNAbilize, drug delivery and antisense technology that uses P-ethoxy, which is a deoxyribonucleic acid (DNA) backbone modification intended to protect the DNA from destruction.

Calando Pharmaceuticals

Calando Pharmaceuticals Inc., a majority-owned subsidiary of Arrowhead Research Corporation (NASDAQ:ARWR - News), has developed proprietary technologies to create targeted siRNA-based therapeutics and small-molecule nanoparticle drug conjugates. Calando's innovative CyclosertTM and RONDELTM nanoparticle systems have been designed to solve the long-standing obstacle of effective delivery and targeting for oligonucleotide and small molecule therapeutics. Calando has developed two clinical-stage drug candidates for the treatment of cancer.

Dicerna Pharmaceuticals (Nasdaq: DRNA)

Dicerna Pharmaceutical utilizes RNA interference technology that silence or turn off the genes that cause disease. Using our GalXC technology platform, we develop potent and patient-friendly RNAi therapies – in-house and with our partners – for both rare diseases and common diseases with a genetic component.

Glaxo Smith Kline (Nasdaq: GSK)

GlaxoSmithKline plc engages in the creation, discovery, development, manufacture, and marketing of pharmaceutical products, vaccines, over-the-counter medicines, and health-related consumer products in the United Kingdom, the United States, and internationally. It operates through four segments: Pharmaceuticals, Pharmaceuticals R&D, Vaccines, and Consumer Healthcare.

Ionis Pharmaceuticals (Nasdaq: IONS)

Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. Ionis is a leader in discovering and developing RNA-targeted therapeutics. The efficient, broadly applicable drug discovery platform, called antisense technology, can treat illnesses where no other therapeutic approaches have proven effective or ever existed. 

Olix Pharmaceuticals

OliX Pharmaceuticals is a clinical-stage pharmaceutical company developing therapeutics against a variety of disorders by down-regulating the expression of disease-causing genes, based on its own proprietary RNAi technology. The Company’s core RNAi platform, asymmetric siRNA (asiRNA), is a unique gene silencing technology based on RNA interference (RNAi), which is considered as the most efficient gene silencing technology. Based on asiRNA technology, OliX has developed cell-penetrating asiRNA (cp-asiRNA), a therapeutic RNAi platform to effectively target locally administrable diseases, such as hypertrophic scar, dry and wet age-related macular degeneration (AMD), subretinal fibrosis, idiopathic pulmonary fibrosis (IPF), and neuropathic pain. 

Quark Pharmaceuticals

Quark Pharmaceuticals, Inc. is a late-stage pharmaceutical company and a leader in the discovery and development of novel small interfering RNA (siRNA) therapeutics. The company aims to improve the lives of millions of patients by harnessing the natural mechanism of siRNAs. By inhibiting key disease-related genes, the approach can address serious unmet medical needs.

Sanofi Genzyme (Nasdaq: SNY)

Sanofi Genzyme is the specialty care global business unit of Sanofi, focused on rare diseases, rare blood disorders, multiple sclerosis, oncology, and immunology. The company is dedicated to discovering and advancing new therapies, providing hope to patients and their families around the world. The company inspires to be the industry leader in specialty care. 

Phio Pharmaceuticals Corp (Nasdaq: PHIO)

Phio Pharmaceuticals Corp.  is a biotechnology company developing the next generation of immuno-oncology therapeutics based on its self-delivering RNAi (INTASYL™) therapeutic platform. The Company's efforts are focused on silencing tumor-induced suppression of the immune system through its proprietary INTASYL platform with utility in immune cells and the tumor micro-environment. 

Regulus Therapeutics (Nasdaq: RGLS) 

Regulus Therapeutics Inc. is a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs. Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a pipeline complemented by a rich intellectual property estate in the microRNA field. 

Rxi Pharmaceuticals (Nasdaq: RXII)

RXi Pharmaceuticals Corporation is a biotechnology company focused on discovering, developing, and commercializing innovative therapies based on its proprietary, self-delivering RNAi (sd-rxRNA®) platform. Therapeutics that use RNA interference, or "RNAi," have great promise because of their ability to down-regulate the expression of specific genes that may be over-expressed in disease conditions. 

Sarepta Therapeutics (Nasdaq:  SRPT)

Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics, gene therapy, and other genetic therapeutic modalities approach for the treatment of rare diseases. The Company has built an impressive pipeline with investigational drug for Duchenne muscular dystrophy (DMD), limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. 

Silence Therapeutics (Nasdaq: SLNCF)

Silence Therapeutics is developing a new generation of medicines by harnessing the body’s natural mechanism of RNA interference, or RNAi, to inhibit the expression of specific target genes thought to play a role in the pathology of diseases with significant unmet medical need. Silence’s proprietary technology can be used to engineer short interfering ribonucleic acids (siRNAs) that bind specifically to and silence, through the RNAi pathway, almost any gene in the human genome to which siRNA can be delivered.

Silenseed Ltd.

Silenseed Ltd is a biopharmaceutical company that develops proprietary RNA interference (RNAi)-based cancer drugs and delivery systems designed to penetrate and treat malignant solid tumors.

Sirnaomics Inc.

Sirnaomics is a clinical-stage biopharmaceutical company leveraging an outstanding level of knowledge and experience in RNA interference (RNAi) technology to forge a path to high-value creation through the discovery and development of therapeutics for human disorders with unmet medical needs. The GalXC approach seeks to optimize the activity of the RNAi pathway so that it operates in the most specific and potent fashion. 

Stoke Therapeutics (Nasdaq: STOK)
Stoke is pioneering a new way to treat the underlying cause of severe genetic diseases by precisely upregulating protein expression. The company develops antisense oligonucleotide medicines that increase gene expression to treat genetic epilepsies and other severe monogenic diseases, including genetic conditions affecting the central nervous system, eye, liver, and kidney.


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