Alnylam Pharmaceuticals, Inc., a biopharmaceutical company, focuses on discovering, developing, and commercializing novel therapeutics based on ribonucleic acid interference. Its marketed products include ONPATTRO (patisiran) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; AMVUTTRA for the treatment of hATTR amyloidosis with polyneuropathy in adults; GIVLAARI for the treatment of adults with acute hepatic porphyria; and OXLUMO for the treatment of primary hyperoxaluria type 1. In addition, the company develops patisiran for the treatment of transthyretin amyloidosis, or ATTR amyloidosis, with cardiomyopathy; cemdisiran to treat complement-mediated diseases; Belcesiran for the treatment of alpha-1 liver disease; Elebsiran to treat chronic HBV infection; Zilebesiran to treat hypertension; ALN-APP to treat Alzheimer's disease and cerebral amyloid angiopathy; and ALN-HSD to treat NASH. Further, it offers Fitusiran for the treatment of hemophilia, Inclisiran to treat hypercholesterolemia, lumasiran for the treatment of advanced PH1, and vutrisiran for the treatment of ATTR amyloidosis, which is in phase 3 clinical trial. Alnylam Pharmaceuticals, Inc. has strategic collaborations with Regeneron Pharmaceuticals, Inc. to discover, develop, and commercialize RNAi therapeutics for a range of diseases by addressing therapeutic targets expressed in the eye and CNS; and Roche to develop pharmaceutical products containing zilebesiran. It also has license and collaboration agreements with Novartis AG; Vir Biotechnology, Inc.; Dicerna Pharmaceuticals, Inc.; Ionis Pharmaceuticals, Inc.; and PeptiDream, Inc. The company was founded in 2002 and is headquartered in Cambridge, Massachusetts.

Apogee Therapeutics, Inc., through its subsidiary, operates as a biotechnology company that develops biologics for the treatment of atopic dermatitis (AD), asthma, chronic obstructive pulmonary disease (COPD), and related inflammatory and immunology indications. The company primarily develops APG777, a subcutaneous (SQ) extended half-life monoclonal antibody (mAb) for AD; and APG808, an SQ extended half-life mAb for COPD. Its earlier-stage programs comprise APG990, an SQ extended half-life mAb for the treatment of AD; and APG222, an extended half-life SQ antibodies for AD. Apogee Therapeutics, Inc. was incorporated in 2022 and is headquartered in Waltham, Massachusetts.

Beam Therapeutics Inc., a biotechnology company, engages in the development of precision genetic medicines for patients suffering from serious diseases in the United States. It develops BEAM-101 for the treatment of sickle cell disease or beta-thalassemia; and BEAM-302, a liver-targeting LNP formulation to treat severe alpha-1 antitrypsin deficiency; BEAM-201, an anti-CD7 CAR-T product candidate, which is in Phase 1/2 clinical trials for the treatment of refractory T-cell acute lymphoblastic leukemia/T cell lymphoblastic lymphoma; and BEAM-301, a liver-targeting LNP formulation for the treatment of glycogen storage disease 1a. The company has research collaboration agreement with Pfizer Inc., focus on in vivo base editing programs for targets rare genetic diseases of the liver, muscle, and central nervous system; Apellis Pharmaceuticals to conduct preclinical research on target specific genes within the complement system in various organs, including the eye, liver, and brain; Verve Therapeutics, Inc., for cardiovascular disease treatments; Sana Biotechnology, Inc., to research, develop, and commercialize rights to CRISPR Cas12b for cell therapy programs; and Orbital Therapeutics design RNA for the prevention, treatment or diagnosis of human disease. Beam Therapeutics Inc. was incorporated in 2017 and is based in Cambridge, Massachusetts.

Intellia Therapeutics, Inc., a genome editing company, focuses on the development of curative therapeutics. The company's in vivo programs include NTLA-2001, which is in Phase 1 clinical trial for the treatment of transthyretin amyloidosis; NTLA-2002 for the treatment of hereditary angioedema; and NTLA-3001 for alpha-1 antitrypsin deficiency associated lung disease. It also focusses on programs comprising hemophilia A and hemophilia B; and research of proprietary programs focused on developing engineered cell therapies to treat various cancers and autoimmune diseases. In addition, the company offers tools comprising of Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) system. It has license and collaboration agreement with Regeneron Pharmaceuticals, Inc. to co-develop potential products for the treatment of hemophilia A and hemophilia B; AvenCell Therapeutics, Inc. to develop allogeneic universal CAR-T cell therapies, and co-develop and co-commercialize allogeneic universal CAR-T cell products for an immuno-oncology indication; SparingVision SAS to develop novel genomic medicines utilizing CRISPR/Cas9 technology for the treatment of ocular diseases; Kyverna Therapeutics, Inc. for the development of an allogeneic CD19 CAR-T cell therapy for the treatment of a variety of B cell-mediated autoimmune diseases; and ONK Therapeutics, Ltd. for the development of engineered NK cell therapies to cure patients with cancer. Intellia Therapeutics, Inc. was incorporated in 2014 and is headquartered in Cambridge, Massachusetts.

Akari Therapeutics, Plc, a clinical-stage biopharmaceutical company, focuses on developing advanced therapies for autoimmune and inflammatory diseases. Its lead product candidate is nomacopan, a second-generation complement inhibitor that prevents inflammatory and prothrombotic activities. The company also develops PASylated-nomacopan, which is in preclinical development for the treatment of geographic atrophy secondary to dry age-related macular degeneration; and hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA) combined with nomacopan, which is in phase 3 clinical development for the treatment of pediatric HSCT-TMA. The company is headquartered in Boston, Massachusetts.

Q32 Bio Inc., a clinical-stage biotechnology company, develops biologic therapeutics to restore healthy immune balance in patients with autoimmune and inflammatory diseases driven by pathological immune dysfunction in the United States. Its lead product candidate is ADX-097, a humanized anti-C3d monoclonal antibody fusion protein to restore complement regulation, which has completed Phase I clinical trial for the treatment of renal and other complement-mediated diseases of high unmet need, including lupus nephritis, immunoglobulin A nephropathy, complement component 3 glomerulopathy, and anti-neutrophil cytoplasmic antibody-associated vasculitis. The company is also developing Bempikibart (ADX-914), a fully human anti–interleukin-7 receptor alpha antagonist monoclonal antibody designed to re-regulate adaptive immune function by blocking signaling mediated by interleukin-7 and thymic stromal lymphopoietin that is Phase II clinical trial for the treatment of atopic dermatitis and alopecia areata. Q32 Bio Inc. was formerly known as AdMIRx Inc. and changed its name to Q32 Bio Inc. in April 2020. The company was founded in 2017 and is based in Waltham, Massachusetts.

Alpine Immune Sciences, Inc. operates as a clinical-stage biopharmaceutical company. It focuses on the discovery and development of immunotherapies through protein engineering technologies for treating autoimmune and inflammatory diseases. The company's product pipeline includes Povetacicept, a dual B cell cytokine antagonist, which is in Phase 2 clinical trial for the treatment of multiple autoimmune and inflammatory diseases; and Acazicolcept, a dual inhibitor of the CD28 and ICOS T cell costimulatory pathways that is in preclinical trial for the treatment of systemic lupus erythematosus. It is also developing RUBY-3 that has completed Phase 1 clinical trial for the treatment of glomerulonephritis; and RUBY-4, which has completed Phase 1 clinical trial for the treatment of cytopenias. The company was incorporated in 2007 and is headquartered in Seattle, Washington.

Morphic Holding, Inc., a biopharmaceutical company, discovers and develops oral small-molecule integrin therapeutics for the treatment of autoimmune, cardiovascular, and metabolic diseases, as well as fibrosis and cancer. Its lead product candidate MORF-057, an a4ß7-specific integrin inhibitor affecting inflammation is in Phase 2 clinical trials for the treatment of inflammatory bowel disease. The company's products in pipeline include Next Gen a4ß7 Inhibitors for additional potential GI indications, such as EGIDs, pouchitis, etc.; avß8 for the treatment of Myelofibrosis and solid tumor; and fibronectin integrin for pulmonary hypertensive. In addition, it has a license agreement with Janssen to discover and develop novel integrin therapeutics; collaboration agreement with Schrödinger for integrin targets; and license agreement with Children's Medical Center Corporation to develop and commercialize products worldwide for any therapeutic or diagnostic use in humans and veterinary applications. Morphic Holding, Inc. was incorporated in 2014 and is headquartered in Waltham, Massachusetts.