U.S. Biotech Breakthroughs

Madrigal Pharmaceuticals

Market Cap: US$10.3b

A biopharmaceutical company, focuses on delivering novel therapeutics for metabolic dysfunction-associated steatohepatitis (MASH) in the United States.

Travere Therapeutics

Market Cap: US$2.6b

A biopharmaceutical company, identifies, develops, and delivers therapies to people living with rare kidney and metabolic diseases in the United States.

Syndax Pharmaceuticals

Market Cap: US$2.1b

A commercial-stage biopharmaceutical company, develops therapies for the treatment of cancer.

Insmed

Market Cap: US$31.0b

Develops and commercializes therapies for patients with serious and rare diseases in the United States, Europe, Japan, and internationally.

Marker Therapeutics

Market Cap: US$21.8m

A clinical-stage immuno-oncology company, develops and commercializes T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications in the United States.

Rhythm Pharmaceuticals

Market Cap: US$5.8b

A commercial-stage biopharmaceutical company, focuses on the rare neuroendocrine diseases in the United States and internationally.

Ardelyx

Market Cap: US$1.3b

Ardelyx, Inc. discovers, develops, and commercializes medicines to treat unmet medical needs in the United States and internationally.

Cogent Biosciences

Market Cap: US$5.7b

A clinical-stage biotechnology company, focuses on developing precision therapies for genetically defined diseases.

Aldeyra Therapeutics

Market Cap: US$134.2m

A biotechnology company, discovers and develops therapies designed to treat immune-mediated diseases.

Capricor Therapeutics

Market Cap: US$1.7b

A clinical-stage biotechnology company, engages in the development of transformative cell and exosome-based therapeutics for treating duchenne muscular dystrophy (DMD) and other diseases with unmet medical needs in the United States.

ARS Pharmaceuticals

Market Cap: US$761.6m

A biopharmaceutical company, develops and commercializes treatments for severe allergic reactions in the United States and internationally.

Lineage Cell Therapeutics

Market Cap: US$366.2m

A clinical-stage biotechnology company, developing cell replacement therapies to treat serious medical conditions in the United States and internationally.

Pelthos Therapeutics

Market Cap: US$73.8m

A bio-pharmaceutical company, develops and commercializes therapeutic products to help patients with unmet treatment burdens.

Tenaya Therapeutics

Market Cap: US$163.1m

A clinical-stage biotechnology company, discovers, develops, and delivers therapies for heart disease in the United States.

Achieve Life Sciences

Market Cap: US$215.6m

A late-stage pharmaceutical company, develops and commercializes cytisinicline for nicotine independence in Canada, the United States, and the United Kingdom.

RenovoRx

Market Cap: US$48.7m

A clinical-stage biopharmaceutical company, engages in the developing of targeted combination therapies to enhance therapeutic outcomes for cancer patients undergoing treatment.

Viridian Therapeutics

Market Cap: US$2.8b

Engages in discovering, developing, and commercializing treatments for serious and rare diseases.

Arcutis Biotherapeutics

Market Cap: US$2.8b

A biopharmaceutical company, focuses on developing and commercializing treatments for dermatological diseases.

CG Oncology

Market Cap: US$5.5b

A late-stage clinical biopharmaceutical company, develops and commercializes cretostimogene grenadenorepvec for patients with bladder cancer in the United States.

AC Immune

Market Cap: US$285.7m

A clinical stage biopharmaceutical company, discovers, designs, and develops medicines, and therapeutic and diagnostic products for the prevention and treatment of neurodegenerative diseases associated with protein misfolding.

CollPlant Biotechnologies

Market Cap: US$8.7m

A regenerative and aesthetic medicine company, focuses on three-dimensional (3D) bioprinting of tissues and organs, and medical aesthetics in the United States, Canada, Israel, Europe, and internationally.

Precigen

Market Cap: US$1.1b

A discovery and clinical-stage biopharmaceutical company, develops gene and cell therapies using precision technology to target diseases in areas of immuno-oncology, autoimmune disorders, and infectious diseases.

Vera Therapeutics

Market Cap: US$2.9b

A clinical stage biotechnology company, focuses on the development and commercialization of transformative treatments for patients with immunological diseases.

Invivyd

Market Cap: US$404.1m

Engages in the research, development, commercialization, and sale of antibody therapies for the prevention and treatment of viral infectious diseases in the United States.

Anavex Life Sciences

Market Cap: US$411.5m

Operates as a biopharmaceutical company.

Unicycive Therapeutics

Market Cap: US$145.5m

A clinical-stage biotechnology company, identifies, develops, and commercializes therapies to address unmet medical needs in the United States.

Praxis Precision Medicines

Market Cap: US$8.2b

A clinical-stage biopharmaceutical company, engages in the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance in the United States.

Day One Biopharmaceuticals

Market Cap: US$2.2b

A commercial-stage biopharmaceutical company, engages in the identification, development, and commercialization of medicines for childhood and adult diseases with equal intensity in the United States.

Amicus Therapeutics

Market Cap: US$4.5b

A biotechnology company, focuses on discovering, developing, and delivering novel medicines for rare diseases in the United States and internationally.

Benitec Biopharma

Market Cap: US$383.3m

A clinical-stage biotechnology company, focuses on the development of novel genetic medicines.

Mirum Pharmaceuticals

Market Cap: US$5.4b

A biopharmaceutical company, focuses on the development and commercialization of novel therapies for debilitating rare and orphan diseases.

Fortress Biotech

Market Cap: US$92.5m

A biopharmaceutical company, engages in the development and commercialization of biopharmaceutical products.

Ionis Pharmaceuticals

Market Cap: US$11.7b

A commercial-stage biotechnology company, provides RNA-targeted medicines in the United States.

Arcellx

Market Cap: US$6.7b

Together with its subsidiary, engages in the development of various immunotherapies for patients with cancer and other incurable diseases in the United States.

Savara

Market Cap: US$1.3b

A clinical stage biopharmaceutical company, focuses on rare respiratory diseases in the United States.

Prelude Therapeutics

Market Cap: US$257.5m

A clinical-stage precision oncology company, focuses on the discovery and development of novel precision cancer medicines to underserved patients.