Top 20 RNAi Therapeutic Companies of 2024

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Top 20 RNAi Therapeutic Companies of 2024


  • RNAi therapeutics are innovative medications that enable rapid identification of specific inhibitors of disease targets across a wide range of molecule classes. Several biopharma companies are investing in RNAi therapeutics extravagantly to maintain a healthy foothold in innovative medicines 
  • In 2023, the global RNAi drug delivery market size was valued at $84.1B and is anticipated to register a market size of $410.3B by 2032 with a CAGR of 18.17 percent. Like the previous year, Roche retains the topmost position with an R&D expenditure of $15.73B followed by Novartis and AstraZeneca with $11.37B and $10.93B, respectively 
  • PharmaShots brings an insightful report on the Top 20 RNAi Therapeutics Companies based on 2023 R&D expenditure 


20. Arcturus Therapeutics 

R&D Expenditure: $192.13M 

Founded Year: 2013 

Total Employees: 180 

Headquarters: San Diego, California 

Market Cap: $0.72B 

Stock Exchange: NASDAQ 

  • Arcturus Therapeutics develops mRNA medicines and vaccines globally for rare and infectious diseases by enabling technologies like LUNAR lipid-mediated delivery, and STARR mRNA technology   
  • With an ongoing collaboration with CSL Seqirus to develop and commercialize self-amplifying mRNA vaccines, Arcturus entered into a joint venture in Japan with ARCALIS to develop mRNA vaccines and therapeutics. In 2023, Arcturus generated $166.8M through this collaboration. ARCT-2301 and ARCT-2303, are among the 7 pipeline candidates of Arcturus 
  • In 2023, Arcturus Therapeutics received the ODD from the US FDA for ARCT-032, for the treatment of cystic fibrosis    


19. Arrowhead Pharmaceuticals 

R&D Expenditure: $353.18M 

Founded Year: 1989 

Total Employees: 525 

Headquarters: California, United States 

Market Cap: $2.89B 

Stock Exchange: NASDAQ 

  • A global biopharma company, Arrowhead Pharmaceuticals develops, manufactures, and commercializes RNA products. The company focuses on cardiometabolic and pulmonary disorders  
  • Arrowhead works in collaboration with Takeda to develop Fazirsiran. ARO-DM1, ARO-HBV, ARO-PNPLA3, and ARO-APOC3 are the company's four pipeline RNAi candidates 
  • In Nov 2023, Arrowhead presented P-II results for Plozasiran and Zodasiran at AHA 2023. In 2023, Arrowhead & GSK entered into an exclusive agreement with Janssen Pharmaceuticals to transfer exclusive global rights to further develop and commercialize JNJ-3989 to GSK  


18. Vir Biotechnology 

R&D Expenditure: $589.67M 

Founded Year: 2016 

Total Employees: 587 

Headquarters: California, United States 

Market Cap: $1.09B 

Stock Exchange: NASDAQ 

  • A commercial-stage biotech company, Vir Biotechnology develops treatments to cure and prevent major infectious diseases by utilizing platforms like the Proprietary Antibody Platform and T Cell-Based Viral Vector Platform  
  • Sotrovimab, sold under the brand name Xevudy is the only marketed product of the company, developed in collaboration with GSK. Vir Biotechnology works in collaboration with many companies to develop various products across multiple indications. The company collaborated with Alnylam to evaluate tobevibart + elebsiran in patients with chronic hepatitis delta (CHD) virus   
  • In 2023, Vir Biotechnology presented initial results from its ongoing P-II CHD and B study through the oral presentation at AASLD’s The Liver Meeting 2023  


17. Jazz Pharmaceuticals 

R&D Expenditure: $849.65M 

Founded Year: 2005 

Total Employees: 2800 

Headquarters:  Ireland 

Market Cap: $6.75B 

Stock Exchange: NASDAQ 

  • Jazz Pharmaceuticals is a global biopharma company that discovers, develops, and commercializes products in oncology and neuroscience   
  • In 2023, Defibrotide, the company’s only approved RNA product generated a total of $0.18B in revenue 
  • In July 2023, Jazz Pharmaceuticals received a positive opinion from the CHMP (EMA committee) for marketing authorization of JZP458 for Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma 


16. Sarepta 

R&D Expenditure: $877.3M 

Founded Year: 1980 

Total Employees: 1314 

Headquarters: Massachusetts, USA 

Market Cap: $11.66B 

Stock Exchange: NASDAQ 

  • A global biopharma company, Sarepta develops precision genetic medicines for rare diseases. The company focuses on Duchenne muscular dystrophy (DMD), Limb-girdle muscular dystrophies (LGMD), Charcot-Marie-Tooth (CMT), and CNS-related disorders. Sarepta develops medicines through three platforms viz. gene therapy, RNA technologies, and gene editing  
  • SRP-5051 (vesleteplirsen), is Sarepta's only pipeline candidate, being evaluated for the treatment of Duchenne Muscular Dystrophy, and several products lined up being developed through different technologies for various indications  
  • In 2023, Quest and Sarepta Therapeutics expanded their collaboration to further develop AAV Companion Diagnostics (CDx) for Sarepta's gene therapies  


15. Alnylam Pharmaceuticals 

R&D Expenditure: $1B 

Founded Year: 2002 

Total Employees: 2100 

Headquarters: Massachusetts, United States 

Market Cap: $18.11B 

Stock Exchange: NASDAQ 

  • Anlylam is an American biopharmaceutical company that discovers, develops, and commercializes RNAi therapeutics. The company operates in a single reported segment 
  • Currently, Alnylam has five approved products Onpattro, Amvuttra, Givlaari, Oxlumo, and Leqvio (collaboration with Novartis). In 2023, Alnylam generated $1.82B in revenue through both collaboration and RNA products. Alnylam is currently working in collaboration with Roche, Regeneron Pharmaceuticals, and Novartis. The company has 15 RNA candidates under development  
  • In 2023, Alnylam reported data from the KARDIA-1, a P-II dose-ranging study of Zilebesiran (RNAi Therapeutic) for the treatment of hypertension in patients at high cardiovascular risk


14. Ionis 

R&D Expenditure: $1.13B 

Founded Year: 1989 

Total Employees: 927 

Headquarters: California, United States 

Market Cap: $6.19B 

Stock Exchange: NASDAQ 

  • Ionis is a US-based biopharma company that focuses on cardiovascular, metabolic, severe, and rare diseases, including neurological disorders and cancer  
  • Spinraza (Nusinersen), Tegsedi (Inotersen), and Waylivra (Volanesorsen) are three approved products. Ionis RNA candidates include Zilganersen, ION541, and Pelacarsen. The company currently works in collaboration with Biogen for ION541 and Novartis AG for Pelacarsen RNAi medicines   
  • In 2023, Ionis entered into a license agreement with Otsuka under which Otsuka gets all the exclusive rights in Europe to commercialize donidalorsen for the treatment of hereditary angioedema (HAE)  


13. BioNTech 

R&D Expenditure: $1.96B   

Founded Year: 2008 

Total Employees: 6133  

Headquarters: Germany 

Market Cap: $20.94B 

Stock Exchange: NASDAQ 

  • BioNTech is a German biotech company that discovers, develops, and manufactures medicines for oncology and infectious diseases based on mRNA  
  • BNT111, BNT113, BNT116, and BNT122 are the four RNA candidates of BioNTech that are under clinical development. BNT122 is being developed in collaboration with Genentech using the iNeST platform   
  • In Nov 2023, Biotheus Collaborated with BioNTech to develop and commercialize PM8002 for the treatment of Multiple Solid Tumor Indications  


12. Biogen 

R&D Expenditure: $2.46B 

Founded Year: 1978 

Total Employees: 7570 

Headquarters: Massachusetts, United States 

Market Cap: $29.36B 

Stock Exchange: NASDAQ 

  • Biogen is an American multinational biotech company serving worldwide with a primary focus on neurological diseases  
  • In collaboration with Ionis Pharmaceuticals, Biogen is developing three RNA candidates, viz. BIIB105, BIIB094, and BIIB101 for various indications   
  • In 2023, Zurzuvae (zuranolone), developed by Biogen and Sage Therapeutics, received US FDA approval for women with postpartum depression  


11. Regeneron Pharmaceuticals 

R&D Expenditure: $4.43B 

Founded Year: 1988 

Total Employees: 1980 

Headquarters: Westchester County, New York 

Market Cap: $99.50B 

Stock Exchange: NASDAQ 

  • A global biotech company, Regeneron focuses on eye, inflammatory, cancer, cardiovascular, metabolic, inflammatory, and rare diseases 
  • Veopoz (pozelimab) is the company’s only approved product for the treatment of CHAPLE in adults, adolescents, and pediatrics aged ≥1. In collaboration with Alnylam Pharmaceuticals, Regeneron is developing ALN-PNP and ALN-APP and ALN-HSD 
  • In Dec 2023, Regeneron reported positive results of Odronextamab in the P-II (ELM-2) study for the treatment of follicular lymphoma  


10. Takeda 

R&D Expenditure: $4.77B 

Founded Year: 1781 

Total Employees: ~50,000 

Headquarters: Tokyo, Japan 

Market Cap: $41.57B 

Stock Exchange: TYO 

  • Takeda is a multinational pharmaceutical company focusing on five key business areas, including Gastrointestinal, Rare diseases, PDT Immunology, Oncology, and Neuroscience  

  • Fazirsiran (P-III) is the only RNAi product that is being developed in collaboration with Arrowhead for the treatment of liver disease associated with alpha-1 antitrypsin deficiency (AATD-LD)  

  • In 2023, Arrowhead and Takeda Report P-II Study (SEQUOIA) of Fazirsiran for AATD-LD  


09. Amgen 

R&D Expenditure: $4.78B 

Founded Year: 1980 

Total Employees: ~26700 

Headquarters: California, United States 

Market Cap: $146.31B 

Stock Exchange: NASDAQ 

  • Amgen is a global biotech company that discovers, develops, manufactures, and delivers innovative therapies. The company focuses on cardiovascular diseases, oncology, bone health, neuroscience, nephrology, and inflammation  
  • Amgen’s Olpasiran (only RNAi candidate) is currently being evaluated in P-III for the treatment of ASCVD  
  • At ASH 2023, Amgen Reported Results for ABP 959 in Clinical Trial for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)   


08. Novo Nordisk 

R&D Expenditure: $4.80B 

Founded Year: 1923 

Total Employees: 64,319 

Headquarters: Denmark 

Market Cap: $428.84B 

Stock Exchange: NYSE 

  • Novo Nordisk is a Danish multinational biopharma company that discovers, develops, manufactures, and commercializes pharmaceuticals for a variety of indications  
  • MARC1 and LXRα are the company’s siRNA candidates, currently being evaluated in P-I 
  • In Aug 2023, Novo Nordisk bought all of Embark Biotech's key assets, including its metabolic program. Novo Nordisk has also committed to a three-year R&D partnership and option agreement with Embark Laboratories to identify and develop medicines for the treatment of obesity and related co-morbidities  


07. Moderna 

R&D Expenditure: $4.84B 

Founded Year: 2010 

Total Employees: 5600 

Headquarters: Massachusetts, United States 

Market Cap: $41.68B 

Stock Exchange: NASDAQ 

  • Moderna is an American multination biopharma company that develops messenger RNA (mRNA) medicines using its mRNA platform  
  • The company generated a total of $6.85B in revenue from there RNA products and is currently working in collaboration with Merck, Vertex, and CytomX to discover, develop, and commercialize potential mRNA medicines. Spikevax is Moderna’s only approved product. mRNA-1283, mRNA-1010, mRNA-1020, and mRNA-1030 are among the company’s 44 pipeline candidates 
  • In Jan'23, Moderna signed a collaboration agreement with CytomX to develop mRNA-based conditionally activated therapies  


06. Sanofi 

R&D Expenditure: $7.42B 

Founded Year: 1956 

Total Employees: 11,660 

Headquarters: Paris, France 

Market Cap: $117.02B 

Stock Exchange: NASDAQ 

  • Sanofi is a global French pharmaceutical and healthcare company that researches, develops, manufactures, and markets pharmaceutical products in immunology and inflammation, neurology, oncology, rare blood disorders, rare diseases, and vaccines  
  • Fitusiran (SAR439774) is the only siRNA therapy in P-III for hemophilia A or B, with plans to file with the US FDA in 2024  
  • In Jul 2023, Sanofi and AstraZeneca’s Beyfortus (nirsevimab-alip)  received approval from the US FDA for the prevention of respiratory syncytial virus (RSV)  


05. GSK 

R&D Expenditure: $7.89B 

Founded Year: 2000 

Total Employees: 70,212 

Headquarters: London, United Kingdom 

Market Cap: $84.79B 

Stock Exchange: LON 

  • GSK is a British multinational biopharma company that researches and develops treatments for infectious diseases, HIV, immunology, respiratory, and cancer  
  • In 2023, Arrowhead & GSK entered an exclusive agreement with Janssen Pharmaceuticals to transfer exclusive global rights to further develop and commercialize JNJ-3989 to GSK. The company also has six candidates lined up in its pipeline namely GSK4532990, GSK4382276, GSK4396687 and GSK5637608 etc 
  • In 2023, GSK entered into an exclusive arrangement with Zhifei to co-promote GSK's shingles vaccine, Shingrix, across China for an initial three-year  


04. Eli Lilly 

R&D Expenditure: $9.31B 

Founded Year: 1876 

Total Employees: 43,000 

Headquarters: Indianapolis, United States 

Market Cap: $696.15B 

Stock Exchange: NYSE 

  • An American pharmaceutical company, Eli Lilly focuses on bone and joint disease, cancer, cardiovascular disease, diabetes, endocrine disease, immunology, neurodegeneration, neuroscience, and pain management 
  • ProQR and Eli Lilly have expanded their license and partnership arrangement to discover, develop, and commercialize novel genetic therapies using ProQR’s proprietary Axiomer RNA editing platform. Eli Lilly RNA candidates include five drugs Solbinsiran, Lepodisiran, APOC3 SiRNA, PNPLA3 siRNA, SCAP siRNA  
  • In Dec 2023, Eli Lilly completed the acquisition of POINT Biopharma  


03. AstraZeneca 

R&D Expenditure: $10.93B 

Founded Year: 1999 

Total Employees: 89900 

Headquarters: Cambridgeshire, United Kingdom 

Market Cap: $229.88B 

Stock Exchange: LON 

  • AstraZeneca discovers and develops medicines in oncology, cardiovascular, renal, metabolic, respiratory, and immunology, vaccines and immune therapies, and rare diseases 
  • AstraZeneca is working in collaboration with VaxEquity to discover, develop, and commercialize the proprietary saRNA therapeutics platform. ALXN2030 and COVID mRNA VLP vaccine are the two RNAi candidates of AstraZeneca  
  • In Dec 2023, AstraZeneca entered into a definitive agreement to acquire Icosavax  


02. Novartis  

R&D Expenditure: $11.37B 

Founded Year: 1996 

Total Employees: 19,607 

Headquarters: Basel, Switzerland 

Market Cap: $216.52B 

Stock Exchange: SWX 

  • An innovative medicines company, Novartis discovers and develops products in cardiovascular, renal, metabolic (CRM), immunology, neuroscience, and oncology 
  • In 2023, Novartis generated a total of $0.36B through its only RNA-approved product Leqvio, developed in collaboration with Alnylam Pharmaceuticals 
  • In Dec 2023, Novartis’ Fabhalta (iptacopan), received US FDA Approval for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)  


01. Roche 

R&D Expenditure: $15.73B 

Founded Year: 1896 

Total Employees: 103,605 

Headquarters: Basel, Switzerland 

Market Cap: $228.85B 

Stock Exchange: SWX 

  • Roche is a global multinational healthcare company that discovers, develops, and commercializes therapeutic and diagnostic products. The company operates under two business segments, i.e. Pharmaceuticals and Diagnostics   
  • The company’s RNAi asset RG6346 (Xalnesiran) is currently being evaluated in P-II for the treatment of chronic hepatitis B (HBV). Moreover, Roche is investigating two more RNA products RG6299 and RG6180 
  • In Jul’23, Alnylam partnered with Roche to co-develop and co-commercialize Zilebesiran, an investigational RNAi drug for the treatment of Hypertension in Patients with High Cardiovascular risk  


The table below showcases the change in R&D expenses in comparison to the previous year:



Total 2022 R&D Expenditure 

Total 2023 R&D Expenditure 

Percentage Change  













Eli Lilly 
















Novo Nordisk 














Regeneron Pharmaceuticals 




















Alnylam Pharmaceuticals 










Jazz Pharmaceuticals 





Vir Biotechnology 





Arrowhead Pharmaceuticals 





Arcturus Therapeutics 





  • Annual reports 

  • SEC Filings 

  • Press releases 

  • Company websites 

Market Cap Source: Google Finance (Apr 2024) 

Currency Conversion: X-Rates (Apr 2024) 


  • The revenue for Roche Increased in CHF but Decreased in USD (Due to Currency Rate Drop) 

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


Prince Giri

An avid reader and a dedicated learner, Prince works as a Content Writer at PharmaShots. Prince possesses an exceptional quality of breaking down the barriers of words by simplifying the terms in digestible chunks to make content readable and comprehensible. Prince likes to read books and loves to spend his free time learning and upskilling himself.

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