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.

Amicus Therapeutics, Inc., a biotechnology company, focuses on discovering, developing, and delivering medicines for rare diseases. Its commercial product and product candidates include Galafold, an oral precision medicine for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant; and Pombiliti + Opfolda, for the treatment of late onset. It has collaboration and license agreements with the University of Pennsylvania to research and develop parvovirus gene therapy products; and GlaxoSmithKline. Amicus Therapeutics, Inc. was incorporated in 2002 and is headquartered in Princeton, New Jersey.

Ligand Pharmaceuticals Incorporated, a biopharmaceutical company, engages in the development and licensing of biopharmaceutical assets worldwide. Its commercial programs include Kyprolis and Evomela, which are used to treat multiple myeloma; Rylaze, a recombinant erwinia asparaginase for the treatment of acute lymphoblastic leukemia or lymphoblastic lymphoma in adult and pediatric patients; Filspari, a dual endothelin and angiotensin II receptor antagonist in development for rare kidney diseases and non-immunosuppressive treatment indicated for immunoglobulin A nephropathy; Teriparatide injection product for osteoporosis; Vaxneuvance for the prevention of invasive disease caused by streptococcus pneumoniae serotypes; and Pneumosil, a pneumococcal conjugate vaccine to help fight against pneumococcal pneumonia among children. The company also offers TZIELD, a CD3-directed antibody indicated to delay the onset of Stage 3 type 1 diabetes (T1D) in adults and children aged 8 years and older with Stage 2 T1D; Nexterone, a captisol-enabled formulation of amiodarone; Zulresso, a captisol-enabled formulation of brexanolone for the treatment of postpartum depression; and Veklury, an antiviral treatment for moderate or severe COVID-19. In addition, it provides Noxafil-IV, a captisol-enabled formulation of posaconazole for IV use; Duavee for the treatment of post-menopausal symptoms in women; Exemptia for autoimmune diseases; Vivitra for breast cancer; and Bryxta and Zybev for various indications. The company has alliances, licenses, and other business relationships with Amgen, Merck, Pfizer, Jazz, Takeda, Gilead Sciences, and Baxter International. Further, it sells Captisol materials. Ligand Pharmaceuticals Incorporated was incorporated in 1987 and is based in Jupiter, Florida.

BioCryst Pharmaceuticals, Inc., a biotechnology company, develops oral small-molecule and protein therapeutics to treat rare diseases. The company markets peramivir injection, an intravenous neuraminidase inhibitor for the treatment of acute uncomplicated influenza under the RAPIVAB, RAPIACTA, and PERAMIFLU names; and ORLADEYO, an oral serine protease inhibitor to treat hereditary angioedema. It is also developing BCX10013, an oral factor D inhibitor for complement-mediated diseases. The company has collaborations and in-license relationships with the Torii Pharmaceutical Co., Ltd.; Seqirus UK Limited; Shionogi & Co., Ltd.; Green Cross Corporation; National Institute of Allergy and Infectious Diseases; Biomedical Advanced Research and Development Authority; the U.S. Department of Health and Human Services; and The University of Alabama at Birmingham, as well as Albert Einstein College of Medicine of Yeshiva University and Industrial Research, Ltd. BioCryst Pharmaceuticals, Inc. was founded in 1986 and is headquartered in Durham, North Carolina.

MediciNova, Inc., a biopharmaceutical company, focuses on developing novel and small molecule therapeutics for the treatment of serious diseases with unmet medical needs in the United States. It is developing MN-166 (ibudilast), an oral anti-inflammatory and neuroprotective agent for treating neurological and other disorders, such as primary and secondary progressive multiple sclerosis, amyotrophic lateral sclerosis, chemotherapy-induced peripheral neuropathy, degenerative cervical myelopathy, glioblastoma, and substance dependence and addiction, as well as prevention of acute respiratory distress syndrome, and long COVID. The company's product pipeline also includes MN-221 (bedoradrine), a selective beta-2-adrenergic receptor agonist for the treatment of acute exacerbations of asthma; MN-001 (tipelukast), an orally bioavailable small molecule compound to treat fibrotic and other diseases, including nonalcoholic fatty liver disease and idiopathic pulmonary fibrosis; and MN-029 (denibulin), a tubulin binding agent for treating solid tumor cancers. It has license agreements with Kyorin Pharmaceutical Co., Ltd; Angiogene Pharmaceuticals, Ltd.; and Meiji Seika Kaisha, Ltd. The company was incorporated in 2000 and is headquartered in La Jolla, California.

Pulmatrix, Inc., a clinical stage biotechnology company, focused on development of novel inhaled therapeutic products to prevent and treat respiratory and other diseases with unmet medical needs in the United States. The company offers iSPERSE, an engineered dry powder delivery platform, which enables delivery of small or large molecule drugs to the lungs by inhalation for local or systemic applications. It engages in developing PUR1800, a narrow spectrum kinase inhibitor completed Phase 1b clinical trials for the treatment of acute exacerbations in chronic obstructive pulmonary disease; PUR1900 for the treatment of allergic bronchopulmonary aspergillosis in patients with asthma and cystic fibrosis; and PUR3100, an iSPERSE formulation of dihydroergotamine which is in Phase 1 for the treatment of acute migraine. It has a license agreement with RespiVert Ltd. for access to a portfolio of kinase inhibitor drug candidates. The company was founded in 2003 and is headquartered in Bedford, Massachusetts.

Landos Biopharma, Inc., a clinical-stage biopharmaceutical company, discovers and develops oral therapeutics for patients with autoimmune diseases. Its lead product candidate is NX-13, an oral gut-selective Nucleotide Oligomerization Domain (NLRX1), a mitochondria-associated receptor associated with the modulation of inflammatory cytokines that is in Phase 2 clinical trial to treat ulcerative colitis (UC) and completed Phase 1 to treat Crohn's disease (CD). The company's preclinical candidates in development include LABP-73, an oral and small molecule NLRX1 pathway agonist for the treatment of asthma and eosinophilic disorders; LABP-66 is an oral and small molecule NLRX1 pathway agonist for the treatment of multiple sclerosis and neurodegenerative disorders; and LABP-69, an oral PLXDC2 agonist for the treatment of rheumatoid arthritis, UC, and CD. The company has a license and collaboration agreement with LianBio Respiratory Limited to develop, manufacture, and commercialize omilancor and NX-13. Landos Biopharma, Inc. was incorporated in 2017 and is headquartered in Blacksburg, Virginia.

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.