Top 20 RNAi Therapeutic Companies of 2024

Shots:

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:

Rank	Company	Total 2022 R&D Expenditure	Total 2023 R&D Expenditure	Percentage Change
1	Roche	$15.20B	$15.73B	3.48%
2	Novartis	$9.17B	$11.37B	23.99%
3	AstraZeneca	$9.76B	$10.93B	11.98%
4	Eli Lilly	$7.19B	$9.31B	29.48%
5	GSK	$6.64B	$7.89B	18.82%
6	Sanofi	$7.19B	$7.42B	3.19%
7	Moderna	$3.29B	$4.84B	47.11%
8	Novo Nordisk	$3.46B	$4.80B	38.72%
9	Amgen	$4.43B	$4.78B	7.90%
10	Takeda	$4.3B	$4.77B	10.93%
11	Regeneron Pharmaceuticals	$3.59B	$4.43B	23.39%
12	Biogen	$2.23B	$2.46B	10.31%
13	BioNtech	$1.64B	$1.96B	19.51%
14	Ionis	$0.95B	$1.13B	18.94%
15	Alnylam Pharmaceuticals	$883.01M	$1B	13.74%
16	Sarepta	$877.09M	$877.3M	0.02%
17	Jazz Pharmaceuticals	$590.45M	$849.65M	43.89%
18	Vir Biotechnology	$474.64M	$589.67M	24.23%
19	Arrowhead Pharmaceuticals	$297.31M	$353.18M	18.79%
20	Arcturus Therapeutics	$147.75M	$192.13M	30.03%