Top 20 RNAi Therapeutic Companies Based on 2022 R&D Expenditure

Shots:

RNAi Therapeutic leverages the biological response of degrading (mRNA) before it gets translated into a protein. By using short double-stranded RNA in silencing a targeted disease-causing mutation, researchers are working steadfastly in multiple indications to expand RNAi therapeutic
RNAi therapy comes to the rescue when targeting a specific protein gets difficult using a conventional approach. In 2022, the global market size of RNAi drug delivery was valued at $69.2B. This year Roche has secured the top position with a total R&D Expenditure of $15.20B, followed by Johnson & Johnson and AstraZeneca
PharmaShots presents the Top 20 RNAi therapeutic companies based on their R&D Expenditure in the year 2022

R&D Expenditure: $43.1M

Founded Year: 1994

Total Employees: ~120

Headquarters: London, United Kingdom

Market Cap: $189.2M

Stock Exchange: NASDAQ

Silence Therapeutics, an emerging biotech company in the siRNA therapeutic industry, aims to design and develop siRNAs to meet the growing needs of patients with insufficient therapy options. The company produces siRNA therapies for its internal development pipeline and out-licensed programs by making use of its mRNAi GOLD platform. Its development pipeline primarily focuses on therapy areas, Cardiovascular Diseases, Hematology, and Rare Diseases. The company has ongoing research and development collaborations with AstraZeneca, Mallinckrodt, and Hansoh. Even though the company manages to generate a considerable revenue of $21.18M in 2022 through its collaborative agreements, Silence Therapeutics still waiting for the approval of its products. Zerlasiran (SLN360) is its P-II product that is being evaluated for cardiovascular diseases.

R&D Expenditure: $64.2M

Founded Year: 1867

Total Employees: ~2700

Headquarters: Dublin, Ireland

Market Cap: $77.7M

Stock Exchange: NYSE

With two simple yet generous visions of managing complexity and improving lives, Mallinckrodt works extensively in developing, manufacturing, marketing, and distributing specialty pharmaceutical products and therapies. Its primary focus areas include Autoimmune and Rare Diseases with a specialty in Neurology, Rheumatology, Hepatology, Nephrology, Pulmonology, Ophthalmology, and Oncology. In 2019, Mallinckrodt entered into a collaboration agreement with Silence Therapeutics to develop and commercialize RNAi therapeutics to treat life-threatening unmet diseases. Through the 2019 agreement, Mallinckrodt received the exclusive worldwide license to Silence’s SLN500. Currently, Mallinckrodt is focused on developing RNAi therapies, SLN501, SLN-MNK-2, and SLN-MNK-3 under its development pipeline.

R&D Expenditure: $84.4M

Founded Year: 2007

Total Employees: ~100

Headquarters: Pennsylvania, United States

Market Cap: $477.1M

Stock Exchange: NASDAQ

Driven by the objective to bring a dynamic portfolio of clinical and pre-clinical assets for the patient’s needs, Arbutus is working steadfastly to mark its dominance in RNAi therapies. AB-729 is the lead RNAi asset in the company’s pipeline. It is a subcutaneous RNAi therapeutic designed to reduce HBV viral antigens, including HBsAg. This drug is currently in multiple P-IIa clinical trials and is being evaluated in combination with other antiviral agents for cHBV.

R&D Expenditure: $85.1M

Founded Year: 2018

Total Employees: ~80

Headquarters: California, United States

Market Cap: $43.1M

Stock Exchange: NASDAQ

Aligos is a clinical-stage biopharma company dedicated to developing novel therapeutics to address the unmet medical needs of patients with liver diseases and viral infections, including NASH, Coronavirus, and Chronic Hepatitis B (CBH). The company is currently evaluating its siRNA therapy ALG‑125755 in patients with Chronic Hepatitis B. ALG‑125755 is currently being evaluated in an ongoing P-I clinical trial for patients with Chronic Hepatitis B across New Zealand, and various countries in Eastern Europe. The preclinical study of ALG‑125755 has already depicted a high potency and long-lasting durability in non-clinical CBH models. Aligos is developing ALG‑125755 under its oligonucleotide platform.

R&D Expenditure: $150.4M

Founded Year: 2012

Total Employees: ~190

Headquarters: California, United States

Market Cap: $1.2B

Stock Exchange: NASDAQ

Avidity is a biopharma company that aims to significantly improve patient’s lives by redefining the delivery of RNA therapeutics. It is committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs). Its AOC platform is inclined to target the underlying causes of previously untreated diseases by combining the specificity of monoclonal antibodies with the precision of RNA therapeutics. The company develops AOC-1001, AOC-1020, and AOC-1044 as siRNA therapies under its pipeline. AOC-1001 is currently being assessed in a P-I/II clinical trial (MARINA) in patients with DM1 with results validating the potential of the company’s AOC platform to address previously unreachable targets and diseases.

R&D Expenditure: $297.3M

Founded Year: 1989

Total Employees: ~500

Headquarters: California, United States

Market Cap: $3.2B

Stock Exchange: NASDAQ

With around twelve development programs ranging from pre-clinical discovery to late-stage clinical development, Arrowhead is working dedicatedly to developing therapies based on siRNAs focusing on a wide range of therapy areas. The company uses a broad portfolio of RNA and efficient modes of delivery to trigger the RNA interference mechanism to induce rapid, deep, and durable suppression of the target genes. ARO-AAT and ARO-APOC3 are among the highest-phase products developing under Arrowhead’s pipeline for the treatment of liver diseases. As a company primarily focusing on developing RNAi therapeutics, its 2022 revenue stood at $243.23M, majorly contributed by its collaborations with GSK, Johnson & Johnson, and Amgen.

R&D Expenditure: $437.9M

Founded Year: 2008

Total Employees: ~2110

Headquarters: Dublin, Ireland

Market Cap: $1.7M

Stock Exchange: NASDAQ

Horizon Therapeutics is a global biotech company working to deliver, develop, and commercialize therapeutic solutions that address the needs of patients impacted by rare, autoimmune, and severe inflammatory diseases. In Jun 2021, Horizon Therapeutics entered into a global collaboration and license agreement with Arrowhead Pharmaceuticals for ARO-XDH as a treatment for patients with uncontrolled gout. Later in Dec 2022, Horizon initiated a P-I clinical trial evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of HZN-457.

R&D Expenditure: $474.6M

Founded Year: 2016

Total Employees: ~580

Headquarters: California, United States

Market Cap: $3.4B

Stock Exchange: NASDAQ

A commercial-stage immunology company, Vir Biotechnology is primarily focused on developing therapies by combining immunologic insights and cutting-edge technologies to treat and prevent serious infectious diseases. Through its internal development, partnerships, and acquisitions, the company has put together four technology platforms that primarily focus on antibodies, T cells, innate immunity, and siRNAs. Currently, the company is developing VIR-2218 under its HBV therapy programs in multiple collaborations with Anlylam, Brii Biosciences, and Gilead. VIR-2218 is an investigational candidate which is subcutaneously administered HBV-targeting siRNA.

R&D Expenditure: $883M

Founded Year: 2002

Total Employees: ~2000

Headquarters: Massachusetts, United States

Market Cap: $25.4B

Stock Exchange: NASDAQ

Alnylam is a renowned name in developing RNAi therapies. The company has pioneered the science behind RNAi therapies by developing and receiving approval for Onpattro (patisiran) in 2018, Givlaari (givosiran) in 2019, Oxlumo (lumasiran) in 2020, and Amvuttra (vutrisiran) in 2022. In 2022, the company generated total revenue of $1.04B. Onpattro contributed a sale of $557.61M, Givlaari contributed $173.14M, $ 93.79M from Amvuttra, and $69.78M from Oxlumo. The company has entered into collaboration agreements with Novartis and Regeneron to enhance the outreach of RNAi therapeutics.

R&D Expenditure: $3.5B

Founded Year: 1923

Total Employees: ~55,180

Headquarters: Bagsvaerd, Denmark

Market Cap: $300.4B

Stock Exchange: NYSE

Novo Nordisk is a global healthcare biopharma company focusing on its mission to drive change and fight against diabetes and other serious chronic diseases such as obesity, rare blood, and rare endocrine diseases. In Nov 2021, Novo Nordisk reported the acquisition of Dicerna Pharmaceuticals, giving Novo the rights to Dicerna’s RNAi platform and products, including Nedosiran, Belcesiran, DCR-AUD, LXRa, and MARC1. Dicerna is currently functions as a subsidiary of Novo for developing RNAi therapies. Under its pipeline, the company is currently developing Nedosiran for the treatment of Primary Hyperoxaluria, Belcesiran for AATD, DCR-AUD for Alcohol use disorder, and both LXRa and MARC1 for NASH.

R&D Expenditure: $3.6B

Founded Year: 1988

Total Employees: ~11,850

Headquarters: New York, United States

Market Cap: $87.1B

Stock Exchange: NASDAQ

With therapy areas extending to eye, inflammatory, cancer, cardiovascular, metabolic, inflammatory, and rare diseases, Regeneron is a biotech company that discovers, develops, and commercializes therapeutic entities to address the unmet needs of patients. In 2018, Regeneron entered into a collaboration agreement with Alnylam to discover RNAi therapies for NASH and to research, co-develop, and commercialize products emerging from this discovery, including ALN-HSD. The company, along with Alnylam is currently investigating the safety and tolerability of ALN-HSD in a P-I clinical trial among patients with NASH. In 2022, Alnylam opted out from further development activities related to ALN-HSD, leaving Regeneron with the exclusive rights to develop and commercialize ALN-HSD while still owing royalties to Alnylam.

R&D Expenditure: $4.3B

Founded Year: 1781

Total Employees: ~50,000

Headquarters: Tokyo, Japan

Market Cap: $53.5B

Stock Exchange: TYO

Takeda is an R&D-driven global pharma company that primarily focuses on delivering targeted therapies for Oncology, Metabolic Disorders, Gastroenterology (GI), Neurology, and Immunology. The company strives by the motto of bringing “Better Health and a Brighter Future.” In 2020, Takeda entered into a collaboration and license agreement with Arrowhead Pharmaceuticals to develop ARO-AAT (Fazirsiran), an RNAi therapy to treat AATLD. Takeda and Arrowhead are jointly evaluating ARO-AAT in a P-II clinical trial under its Gastroenterology program.

R&D Expenditure: $4.4B

Founded Year: 1980

Total Employees: ~25,200

Headquarters: California, United States

Market Cap: $130.8B

Stock Exchange: NASDAQ

By leveraging its expertise in finding solutions for improving health outcomes Amgen dedicatedly discovers, develops, manufactures, and delivers innovative human therapeutics. Olpasiran is the company’s lead asset in siRNA therapies. Olpasiran is a siRNA designed to lower the body’s production of apolipoprotein(a) or Lp(a) and is currently being evaluated in a P-II clinical trial (OCEAN(a)-DOSE). In Nov 2022, Amgen presented the results from the P-II clinical trial (OCEAN(a)-DOSE), depicting a reduction in patients’ Lp(a) levels by 95%.

R&D Expenditure: $6.6B

Founded Year: 2000

Total Employees: ~69,400

Headquarters: London, United Kingdom

Market Cap: $75.3B

Stock Exchange: LON

GSK is a global biopharma company driven by the idea to unite science, technology, and talent to get ahead of diseases. The company strives to discover and develop therapeutic products under its business segments, including General Medicines, Specialty Medicines, and Vaccines. Its primary treatment areas are Infectious Diseases, HIV, Immunology, Respiratory, and Oncology. In Nov 2021, GSK entered into a license agreement with Arrowhead to develop and commercialize Arrowhead’s RNAi therapy, ARO-HSD. GSK and Arrowhead are currently evaluating ARO-HSD in a P-II clinical trial for NASH.

R&D Expenditure: $7.19B

Founded Year: 2004

Total Employees: ~91,573,440

Headquarters: Paris, France

Market Cap: $143.9B

Stock Exchange: NASDAQ

A renowned innovative global healthcare company, Sanofi aims to develop products for various indications, including Oncology, Cardiovascular, Neurology, Immunology, and Inflammatory. As per its 2018 collaboration and license agreement with Alnylam, Sanofi is now evaluating the safety and efficacy of Fitusiran (SAR439774) in a P-III clinical trial. Fitusiran (SAR439774) is an investigation siRNA developed to treat patients with haemophilia A or B, with or without inhibitors. In 2022, the company presented the results from the P-III clinical trial (ATLAS-PPX) evaluating Fitusiran (SAR439774).

R&D Expenditure: $7.19B

Founded Year: 1876

Total Employees: ~21,000

Headquarters: Indianapolis, United States

Market Cap: $365.1B

Stock Exchange: NYSE

Eli Lilly is an American pharma company that primarily focuses on discovering, developing, manufacturing, and marketing products under multiple therapy areas, including Diabetes, Oncology, Immunology, and Neuroscience. In 2018, Eli Lilly entered a global license and research collaboration with Dicerna to discover, develop, and commercialize new potential RNAi therapies. This partnership leverages Dicerna’s GalXC RNAi technology platform by allowing both companies to jointly discover therapies for cardio-metabolic diseases, neurodegeneration, and pain. Eli Lilly is developing RNAi therapies, LY3819469 and LY3561774, under its development pipeline. In 2022 the company initiated the P-II clinical trial for LY3819469 and LY3561774 RNAi therapies.

R&D Expenditure: $9.2B

Founded Year: 1996

Total Employees: ~1,02,000

Headquarters: Basel, Switzerland

Market Cap: $227.6B

Stock Exchange: SWX

With multiple therapy areas focussing on Oncology, Hepatology, Immunology, Dermatology, Musculoskeletal Diseases, Neurosciences, and Ophthalmology, Novartis is a global biopharma company that aims to integrate innovative science and digital technologies into transformative treatment options. The company has marked its spot among the companies developing RNAi therapeutics by entering into a collaboration agreement with Alnylam Therapeutics. Novartis obtained the global rights to develop, manufacture, and commercialize Leqvio from Alnylam Pharmaceuticals. Leqvio is a siRNA therapy designed to reduce LDL cholesterol, a critical risk for ASCVD. In 2022, Leqvio solely contributed $112M to the company’s overall revenue.

R&D Expenditure: $9.8B

Founded Year: 1923

Total Employees: ~83,500

Headquarters: Cambridge, United Kingdom

Market Cap: $236.7B

Stock Exchange: LON

AstraZeneca combines research and the latest development model to yield quality pharmaceutical products. The company has also entered the list of siRNAs targeting companies through its siRNA targeting molecule, ALXN2030. AstraZeneca is currently evaluating the potential of ALXN2030 in multiple clinical development programs for siRNA targeting complement C3 nephrology. Currently, AstraZeneca is conducting a P-I clinical trial for the evaluation of ALXN2030 in healthy individuals with data anticipated in 2024.

R&D Expenditure: $14.6B

Founded Year: 1886

Total Employees: ~1,52,700

Headquarters: New Jersey, United States

Market Cap: $425.3B

Stock Exchange: NYSE

A multi-disciplinary company with focus areas extending to Cardiovascular, Oncology, Immunology, and Endocrinology, Johnson & Johnson (J&J) is a prominent name in developing medical devices, pharmaceuticals, and consumer packaged products. In 2018, J&J’s subsidiary Janssen Pharmaceuticals entered into a collaboration agreement with Arrowhead to develop and commercialize ARO-HBV, its RNAi therapy asset, and received the options for up to three additional RNAi therapeutics for new targets. Currently, J&J is evaluating ARO-HBV as JNJ-3989 under its development pipeline in a P-II clinical trial for Hepatitis B.

R&D Expenditure: $15.2B

Founded Year: 1896

Total Employees: ~1,03,600

Headquarters: Basel, Switzerland

Market Cap: $258.4B

Stock Exchange: SWX

Roche is a global biopharma company dedicated to developing and commercializing products for several therapy areas, including Oncology, Immunology, Ophthalmology, Cardiovascular, and Respiratory. Roche added RNAi therapies to its development pipeline, through its collaboration with Dicerna. The collaboration gave Roche the right to develop novel RNAi therapies to treat chronic HBV infection using Dicerna’s proprietary GalXC RNAi platform technology. Roche is currently evaluating an RNAi asset, RG6346 (Xalnesiran), in a P-II clinical trial for chronic HBV along with Dicerna.

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