U.S. Biotech Breakthroughs

Alnylam Pharmaceuticals

Market Cap: US$38.1b

Alnylam Pharmaceuticals, Inc. discovers, develops, and commercializes therapeutics based on ribonucleic acid interference.

Ardelyx

Market Cap: US$954.6m

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

Blueprint Medicines

Market Cap: US$6.6b

A precision therapy company, develops medicines for genomically defined cancers and blood disorders in the United States and internationally.

Legend Biotech

Market Cap: US$5.2b

Through its subsidiaries, operates as a biopharmaceutical company that discovers, develops, manufactures, and commercializes novel cell therapies for oncology and other indications in the United States, China, and Europe.

Madrigal Pharmaceuticals

Market Cap: US$6.2b

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

Aldeyra Therapeutics

Market Cap: US$134.8m

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

Capricor Therapeutics

Market Cap: US$508.3m

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.

Tenaya Therapeutics

Market Cap: US$71.9m

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

SpringWorks Therapeutics

Market Cap: US$3.5b

A commercial-stage biopharmaceutical company, engages in the development and commercialization of medicines for underserved patient populations suffering from rare diseases and cancer.

Soleno Therapeutics

Market Cap: US$3.7b

A clinical-stage biopharmaceutical company, focuses on the development and commercialization of novel therapeutics for the treatment of rare diseases.

Vanda Pharmaceuticals

Market Cap: US$254.6m

A biopharmaceutical company, focuses on the development and commercialization of therapies to address high unmet medical needs worldwide.

RenovoRx

Market Cap: US$44.6m

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

Abeona Therapeutics

Market Cap: US$313.0m

A clinical-stage biopharmaceutical company, develops gene and cell therapies for life-threatening diseases.

Checkpoint Therapeutics

Market Cap: US$349.7m

A commercial -stage immunotherapy and targeted oncology company, focuses on the acquisition, development, and commercialization of novel treatments for patients with solid tumor cancers in the United States and internationally.

Silence Therapeutics

Market Cap: US$242.8m

A biotechnology company, engages in the discovery and development of novel molecules incorporating short interfering ribonucleic acid (siRNA) to inhibit the expression of specific target genes in hematology, cardiovascular, and rare diseases.

OS Therapies

Market Cap: US$31.4m

A clinical stage biopharmaceutical company, focuses on the identification, development, and commercialization of treatments for osteosarcoma and other solid tumors in the United States.

ARS Pharmaceuticals

Market Cap: US$1.4b

A biopharmaceutical company, develops and commercializes treatments for severe allergic reactions.

aTyr Pharma

Market Cap: US$333.8m

A clinical stage biotechnology company, engages in the discovery and development of product candidates that translate tRNA synthetase biology into new therapies for fibrosis and inflammation in the United States.

Arcutis Biotherapeutics

Market Cap: US$1.6b

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

CollPlant Biotechnologies

Market Cap: US$23.4m

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.

XOMA Royalty

Market Cap: US$300.6m

Operates as a biotech royalty aggregator in the United States and the Asia Pacific.

TuHURA Biosciences

Market Cap: US$144.6m

A clinical stage immuno-oncology company, that develops novel technologies for the treatment of cancer immunotherapies.

Invivyd

Market Cap: US$91.2m

A biopharmaceutical company, focuses on the discovery, development, and commercialization of antibody-based solutions for infectious diseases in the United States.

BeiGene

Market Cap: US$25.6b

An oncology company, engages in discovering and developing various treatments for cancer patients in the United States, China, Europe, and internationally.

Rhythm Pharmaceuticals

Market Cap: US$4.0b

A commercial-stage biopharmaceutical company, focuses on the rare neuroendocrine diseases.

Unicycive Therapeutics

Market Cap: US$68.2m

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

Quoin Pharmaceuticals

Market Cap: US$5.0m

A late stage specialty pharmaceutical company, focuses on the development and commercialization of therapeutic products for rare and orphan diseases in the United States.

BioRestorative Therapies

Market Cap: US$11.6m

Develops therapeutic products and medical therapies using cell and tissue protocols primarily involving adult stem cells.

SELLAS Life Sciences Group

Market Cap: US$154.4m

A late-stage clinical biopharmaceutical company, focuses on the development of novel therapeutics for various cancer indications in the United States.

Vertex Pharmaceuticals

Market Cap: US$111.8b

A biotechnology company, engages in developing and commercializing therapies for treating cystic fibrosis (CF).

Anavex Life Sciences

Market Cap: US$664.2m

A clinical stage biopharmaceutical company, engages in the development of therapeutics for the treatment of central nervous system diseases in the United States.

Amicus Therapeutics

Market Cap: US$2.0b

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

Savara

Market Cap: US$613.1m

A clinical stage biopharmaceutical company, focuses on rare respiratory diseases.

Day One Biopharmaceuticals

Market Cap: US$637.6m

A commercial-stage company, focused on advancing class medicines for childhood and adult diseases with equal intensity in the United States.

Mereo BioPharma Group

Market Cap: US$356.2m

A biopharmaceutical company, develops and commercializes therapeutics for the treatment of oncology and rare diseases in the United Kingdom, the United States, and internationally.

Sierra Oncology

Market Cap: US$1.3b

Sierra Oncology, Inc., a late-stage biopharmaceutical company, engages in researching, developing, and commercializing therapies for the treatment of patients with hematology and oncology needs.