Arrowhead Pharmaceuticals, Inc. develops medicines for the treatment of intractable diseases in the United States. The company's products in pipeline includes ARO-AAT, a RNA interference (RNAi) therapeutic candidate that is in Phase II clinical trial for the treatment of liver diseases associated with alpha-1 antitrypsin deficiency; ARO-APOC3, which is in phase 2b and one phase 3 clinical trial to treat hypertriglyceridemia; ARO-ANG3 that is in Phase 2b clinical trial to reduce production of angiopoietin-like protein 3; ARO-HSD, which is in Phase 1/2a clinical trial to treat liver diseases; ARO-ENaC, which is in a Phase 1/2a clinical trial to reduce production of the epithelial sodium channel alpha subunit in the airways of the lung; ARO-C3 for the treatment of complement-mediated disease that is in Phase 1/2a clinical trial; ARO-Lung2 for the treatment of chronic obstructive pulmonary disorder; ARO-DUX4 for the treatment of facioscapulohumeral muscular dystrophy; ARO-XDH to treat uncontrolled gout; ARO-COV for the treatment of COVID-19 and other pulmonary-borne pathogens; and ARO-HIF2, which is in phase 1b clinical trial to treat clear cell renal cell carcinoma.

CytomX Therapeutics, Inc. operates as an oncology-focused biopharmaceutical company in the United States. The company develops antibody therapeutics based on its Probody technology platform for the treatment of cancer.

Assembly Biosciences, Inc., a clinical-stage biotechnology company, discovers and develops therapeutic candidates for the treatment of hepatitis B virus (HBV) infection in the United States.

Apellis Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of therapeutic compounds through the inhibition of the complement system for autoimmune and inflammatory diseases.

Iteos Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in the discovery and development of immuno-oncology therapeutics for patients. The company's product pipeline includes inupadenant, a small molecule antagonist of the adenosine A2AR antagonists that is in Phase 2 clinical trials; and EOS-448, an antagonist of TIGIT or T-cell immunoreceptor with Ig and ITIM domains, which is in Phase 1/2 clinical trial, as well as used to engage the Fc gamma receptor, or Fc?R to activate dendritic cells and macrophages and to promote antibody-dependent cellular cytotoxicity, or ADCC activity.

TScan Therapeutics, Inc., a preclinical-stage biopharmaceutical company, develops T cell receptor-engineered T cell therapies for the treatment of patients with cancer.

ProQR Therapeutics N.V., a biopharmaceutical company, engages in the discovery and development of RNA-based therapeutics for the treatment of genetic disorders.

Alnylam Pharmaceuticals, Inc., a biopharmaceutical company, focuses on discovering, developing, and commercializing novel therapeutics based on ribonucleic acid interference. The company's pipeline of investigational RNAi therapeutics focuses on genetic medicines, cardio-metabolic diseases, hepatic infectious diseases, and central nervous system (CNS)/ocular diseases.

Blueprint Medicines Corporation, a precision therapy company, develops medicines for genomically defined cancers and blood disorders in the United States and internationally.

Terns Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops small-molecule single-agent and combination therapy candidates for the treatment of non-alcoholic steatohepatitis (NASH) and obesity.

Amylyx Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, engages in developing various therapeutics for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases.

Acumen Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, discovers and develops therapies for the treatment of Alzheimer's disease. The company focuses on advancing a targeted immunotherapy drug candidate ACU193, a humanized monoclonal antibody that is in Phase I clinical-stage to target soluble amyloid-beta oligomers.

Inozyme Pharma, Inc., a rare disease biopharmaceutical company, engages in developing therapeutics for treating abnormal mineralization impacting the vasculature, soft tissue, and skeleton diseases.

X4 Pharmaceuticals, Inc., a late-stage clinical biopharmaceutical company, focuses on the research, development, and commercialization of novel therapeutics for the treatment of rare diseases.

Day One Biopharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops and commercializes targeted therapies for patients with genetically defined cancers. Its lead product candidate is DAY101, an oral brain-penetrant type II pan-rapidly accelerated fibrosarcoma kinase inhibitor that is in Phase II clinical trial for pediatric patients with relapsed/progressive low-grade glioma.

Mereo BioPharma Group plc, a biopharmaceutical company, develops and commercializes therapeutics for the treatment of oncology and rare diseases in the United Kingdom and internationally.