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.

ACADIA Pharmaceuticals Inc., a biopharmaceutical company, focuses on the development and commercialization innovative medicines that address unmet medical needs in central nervous system (CNS) disorders and rare diseases in the United States. The company offers NUPLAZID (pimavanserin) for the treatment of hallucinations and delusions associated with Parkinson's disease psychosis; and DAYBUE, a novel synthetic analog of the amino-terminal tripeptide of insulin-like growth factor 1 for treatment of Rett Syndrome. It also develops Pimavanserin that is in Phase III ADVANCE-2 study to treat the negative symptoms of schizophrenia; ACP-101 whixh is in Phase III for the treatment of hyperphagia in Prader-Willi syndrome; ACP-204 which is in Phase II for the treatment of Alzheimer's disease psychosis; ACP-2591 that is in Phase I for Rett syndrome and Fragile X syndrome; preclinical antisense oligonucleotide programs; and other programs for neuropsychiatric symptoms. It has a license agreement with Neuren Pharmaceuticals Limited to develop and commercialize trofinetide for Rett syndrome and other indications; and a license and collaboration agreement with Stoke Therapeutics, Inc. to discover, develop and commercialize novel RNA-based medicines for the potential treatment of severe and rare genetic neurodevelopmental diseases of the CNS. The company was formerly known as Receptor Technologies, Inc. and changed its name ACADIA Pharmaceuticals Inc. in 1997. ACADIA Pharmaceuticals Inc. was incorporated in 1993 and is headquartered in San Diego, California.

Acelyrin, Inc., a clinical biopharma company, focuses on identifying, acquiring, and accelerating the development and commercialization of transformative medicines. The company's lead product candidate is izokibep, a small protein therapeutic designed to inhibit IL-17A with high potency, which is in Phase 3 clinical trials for use in the treatment of Hidradenitis Suppurativa, Psoriatic Arthritis, and uveitis, as well as in Phase 2 clinical trials for use in the treatment of Axial Spondyloarthritis. It is also developing lonigutamab, a humanized immunoglobulin G1 (IgG1) monoclonal antibody which is in Phase 1 clinical trials for use in the treatment of thyroid eye disease. In addition, the company's develops SLRN-517, a fully human IgG1 monoclonal antibody targeting c-KIT, which is in preclinical stage for use in the treatment of chronic urticaria. Acelyrin, Inc. was incorporated in 2020 and is headquartered in Agoura Hills, California.

AC Immune SA, a clinical stage biopharmaceutical company, discovers, designs, and develops medicines and diagnostic products for the prevention and treatment of neurodegenerative diseases associated with protein misfolding. The company's SupraAntigen and Morphomer platforms are designed to generate biologics and small molecules, which selectively interact with misfolded proteins that are common in a range of neurodegenerative diseases. It is developing Crenezumab, a humanized, conformation-specific monoclonal antibody used to slow Alzheimer's disease (AD) progression; and Semorinemab, an investigational monoclonal anti-Tau antibody that targets the N-terminal portion of the Tau protein and is designed to bind to Tau and slow its spread between neurons for the treatment of AD. The company is also developing ACI-24.060 for the treatment of down syndrome-related AD; ACI-7104.056, to treat Parkinson's disease; and ACI-35.030, designs to stimulate a patient's immune system to produce antibodies against pathological phosphorylated Tau. In addition, it is developing diagnostic programs, consisting of PI-2620, a Tau diagnostic for AD, as well as non-AD Tauopathies; and ACI-12589, a-sync PET tracer that supports the differential diagnosis of multiple system atrophy from other neurodegenerative disease. Further, the company is researching and developing Morphomer Tau aggregation inhibitors to evaluate candidates in AD and NeuroOrphan Tauopathies. Additionally, it has discovery and preclinical stage molecules targeting range of neurodegenerative diseases, which include diagnostics targeting TDP-43, alpha-synuclein, and NLRP3. The company has license agreements and collaborations with Genentech, Inc.; Janssen Pharmaceuticals, Inc.; Life Molecular Imaging SA; and Eli Lilly and Company. AC Immune SA was incorporated in 2003 and is headquartered in Lausanne, Switzerland.

Alector, Inc., a clinical stage biopharmaceutical company, develops therapies for the treatment of neurodegeneration diseases. Its products include AL001, an immune activity in the brain with genetic links to multiple neurodegenerative disorders, which is in Phase III clinical trial for the treatment of frontotemporal dementia, Alzheimer's, Parkinson's, and amyotrophic lateral sclerosis diseases; and AL101 that is in Phase I clinical trial for the treatment of neurodegenerative diseases, including Alzheimer's and Parkinson's diseases. The company also offers AL002, a product candidate that is in Phase II clinical trial for the treatment of Alzheimer's disease. Alector, Inc. has a collaboration agreement with Adimab, LLC for the research and development of antibodies; and a strategic collaboration agreement with GlaxoSmithKline plc for the development and commercialization of monoclonal antibodies, such as AL001 and AL101 to treat neurodegenerative diseases. The company was founded in 2013 and is headquartered in South San Francisco, California.

Vigil Neuroscience, Inc., a clinical-stage biotechnology company, focuses on developing treatments for rare and common neurodegenerative diseases by restoring the vigilance of microglia, the sentinel immune cells of the brain. Its lead candidate is VGL101(Iluzanebart), a human monoclonal antibody agonist targeting human triggering receptor expressed on myeloid cells 2 and is in a Phase 2 trial in patients with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), a rare and fatal neurodegenerative disease. The company is also developing VG-3927, an orally-available small molecule TREM2 agonist to treat common neurodegenerative diseases associated with microglial dysfunction, with an initial focus on Alzheimer's disease in genetically defined subpopulations, which is being evaluated in a Phase 1 clinical trial. It has license agreement with Amgen Inc. to commercially develop, manufacture, use, distribute, and sell therapeutic products containing compounds that bind to TREM2. The company was incorporated in 2020 and is headquartered in Watertown, Massachusetts.

Vaccinex, Inc., a clinical-stage biotechnology company, engages in the discovery and development of targeted biotherapeutics to treat cancer, neurodegenerative diseases, and autoimmune disorders. The company's lead drug candidate, pepinemab, a humanized monoclonal antibody that binds and blocks the activity of SEMA4D. It is developing Pepinemab, which is in phase 2 study for treatment of Alzheimer's disease and has completed phase 2 study for treatment of Huntington's disease; Pepinemab in combination with Pembrolizumab in phase 2 study for head and neck cancer, Avelumab in phase 2 study for pancreatic cancer, and completed phase 2 study with Avelumab for non-small cell lung cancer; and Pepinemab in combination with Nivolumab completed phase 2 study for melanoma, and trastuzumab and DC vaccine in phase 2 study for breast cancer. The company has also developed ActivMAb, an antibody drug discovery platform based on a novel method for complex targets, such as multi-pass membrane receptors or large and diverse libraries of full-length human monoclonal antibodies on the surface of pox viruses. It has collaborations with Merck Sharp & Dohme; Ares Trading S.A.; The Children's Hospital of Philadelphia; Emory University; Huntington Study Group; H. Lee Moffitt Cancer Center and Research Institute, Inc; Catalent Pharma Solutions, LLC; Surface Oncology, Inc.; and Pharmaceutical and Biotech Co. The company was incorporated in 2001 and is headquartered in Rochester, New York.

Alzamend Neuro, Inc., a clinical-stage biopharmaceutical company, focuses on developing various products for the treatment of neurodegenerative and psychiatric disorders. Its pipeline includes AL001, which delivers a therapeutic combination of lithium, proline, and salicylate for the treatment of Alzheimer's, bi-polar disorder, post-traumatic stress disorder, major depressive disorder, other neurodegenerative diseases, and psychiatric disorders; and ALZN002 stage, which uses a method using a mutant-peptide sensitized cell as a cell-based therapeutic vaccine to restore the ability of a patient's immunological system to combat Alzheimer's disease. The company has partnership agreement with Massachusetts General Hospital for the Phase II clinical trial of AL001. Alzamend Neuro, Inc. was incorporated in 2016 and is headquartered in Atlanta, Georgia.