Biotech Stocks: 5 Biggest Companies

Market capitalization: US$105.58 billion

Vertex Pharmaceuticals is a global biotech firm focused on developing and commercializing therapies for treating cystic fibrosis. It has a number of approved treatments for cystic fibrosis, and a pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes.

Vertex announced in March 2024 that the US Food and Drug Administration (FDA) has cleared an Investigational New Drug Application for its drug candidate VX-407, which targets the underlying cause of the most common inherited kidney disease, autosomal dominant polycystic kidney disease, in patients with a subset of PKD1 genetic variants.

Market capitalization: US$102.47 billion

Biotech leader Regeneron Pharmaceuticals develops and commercializes medicines targeting cancer, pain and a wide variety of diseases, including inflammatory, cardiovascular, metabolic, hematologic and rare diseases.

The FDA has approved nine of the treatments in the company’s portfolio, and the company also has a number of product candidates in development. Its Regeneron Genetics Center is investigating approaches to speeding up the discovery and development of medicines for serious illnesses.

The company's announced in March that an extension for its Praluent treatment for heterozygous familial hypercholesterolemia, an inherited genetic disorder that leads to extremely high levels of cholesterol and associated health risks, has been approved by the FDA for pediatric patients aged 8 and older in combination with diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies.

Market capitalization: US$39.97 billion

Best known today for the rapid deployment of effective COVID-19 vaccines, Moderna leads the world in the field of mRNA-based medicine. Alongside a diverse clinical and intellectual property portfolio targeting infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases and autoimmune diseases, the company’s assets include an integrated manufacturing plant that allows for both clinical and commercial production.

In April 2023, the biotechnology and pharmaceutical company partnered with technology giant IBM (NYSE:IBM) to explore the use of quantum computing and generative artificial intelligence in developing mRNA medicines.

Moderna completed the construction of its state-of-the art mRNA manufacturing facility in Laval, Québec, Canada, in February of this year. Beginning in 2025, pending regulatory approvals, the facility is expected to manufacture respiratory mRNA vaccines for Canadians.

“The construction of this facility marks an important milestone in Moderna’s strategic partnership with the federal government to support domestic preparedness for future pandemics for all Canadians,” the press release stated.

Market capitalization: US$23.1 billion

Belgian global immunology company Argenx is developing an extensive pipeline of novel antibody-based medicines for the treatment of patients with severe autoimmune diseases.

The company’s portfolio includes commercial drug product Vyvgart, which is indicated for the treatment of the neuromuscular disease generalized myasthenia gravis in adult patients. The treatment uses IgG antibodies, a protein made by the immune system.

In late March 2024, Argenx announced that Vyvgart had obtained approval in Japan for its use as an intravenous treatment in adults with primary immune thrombocytopenia.

Market capitalization: US$21.81 billion

Biopharmaceutical firm BioNTech is advancing immunotherapies for serious diseases such as cancer. The company’s portfolio of oncology product candidates includes mRNA-based therapies, CAR-T cell therapies and targeted cancer antibodies. In partnership with Pfizer (NYSE:PFE), BioNTech brought to market one of the world’s most important COVID-19 vaccines.

Last year, BioNTech inked a potential US$1.67 billion collaboration deal with China-based biotech company Duality Biologics. Under the agreement, BioNTech received licensing rights outside of China, Hong Kong and Macau to develop, manufacture and license two of Duality Biologics' antibody-drug conjugate candidates targeting solid tumors.

This January, the partner companies announced that the FDA granted Fast Track designation for the use of the candidate BNT325/DB-1305 for the treatment of platinum-resistant ovarian epithelial cancer, fallopian tube cancer or primary peritoneal cancer in patients who have previously received one to three systemic treatment regimens.