Neurocrine Biosciences

Voyager Therapeutics, Inc. is a clinical-stage gene therapy company dedicated to developing innovative treatments for severe neurological diseases that significantly impact patients' lives. The company's lead candidate, VY-AADC, is currently undergoing an open-label Phase 1b clinical trial for Parkinson's disease. In addition to this, Voyager's preclinical pipeline includes therapies targeting amyotrophic lateral sclerosis (VY-SOD102), Huntington's disease (VY-HTT01), Friedreich's ataxia (VY-FXN01), and various tauopathies and synucleinopathies, such as Alzheimer's disease and Lewy Body Dementia. Voyager employs an adeno-associated virus (AAV) gene therapy approach, which is designed to modify protein production to alleviate disease symptoms, thereby aiming for clinically meaningful outcomes. The company has established strategic collaborations with institutions like the University of Massachusetts and ClearPoint Neuro, as well as partnerships with Brammer Bio and Fujifilm Diosynth Biotechnologies to advance its gene therapy programs. Founded in 2013 and based in Cambridge, Massachusetts, Voyager Therapeutics is committed to addressing unmet medical needs within the central nervous system.

Diurnal Group plc is a specialty pharmaceutical company based in Cardiff, United Kingdom, that focuses on developing hormone therapeutics for chronic endocrine conditions. Founded in 2004, the company offers innovative treatments such as Alkindi, a replacement therapy for pediatric adrenal insufficiency in Europe. Diurnal is also advancing its product pipeline with Chronocort, which has completed Phase III clinical trials for congenital adrenal hyperplasia and adrenal insufficiency. Additionally, the company's early-stage pipeline includes Native Oral Testosterone for hypogonadism, a short interfering RNA therapy for adrenocorticotropin-dependent Cushing's syndrome, and a modified-release preparation of T3 hormone for hypothyroidism. Diurnal's research is centered on circadian-based endocrinology, aimed at providing effective and long-term solutions for patients with rare and chronic endocrine disorders.

Xenon Pharmaceuticals Inc. is a clinical-stage biopharmaceutical company based in Burnaby, Canada, focused on developing therapeutics for neurological disorders. The company's clinical pipeline includes several product candidates aimed at treating epilepsy, such as XEN496 and XEN1101, both Kv7 potassium channel modulators currently in Phase II trials, and XEN901, a selective Nav1.6 sodium channel inhibitor in Phase I trials. Additionally, Xenon is developing XEN007, a central nervous system-acting calcium channel modulator, also in Phase I trials. The company leverages insights from genetic research, particularly from families with severe phenotypes, to identify drug targets, which may extend beyond rare genetic disorders. Furthermore, Xenon has established a collaboration with Neurocrine Biosciences to advance the development of innovative epilepsy treatments.

Northwest Neurologic Inc. (NNL) identifies and discovers neuroendocrine pathways to control obesity, stroke, depression, and other medical problems.