Neurocrine Biosciences

Voyager Therapeutics, Inc. is a clinical-stage gene therapy company dedicated to developing innovative treatments for severe neurological diseases. The company’s lead clinical candidate, VY-AADC, is currently undergoing an open-label Phase 1b trial for Parkinson’s disease. Its diverse preclinical pipeline includes candidates for various conditions, such as VY-SOD102 for amyotrophic lateral sclerosis, VY-HTT01 for Huntington’s disease, VY-FXN01 for Friedreich’s ataxia, and programs targeting tauopathies and synucleinopathies, which encompass Alzheimer’s disease and related disorders. Voyager Therapeutics employs a gene therapy platform known as TRACER, which enables the engineering and optimization of adeno-associated virus (AAV) therapies aimed at producing lasting therapeutic effects from a single treatment. The company collaborates with several institutions, including the University of Massachusetts and ClearPoint Neuro, as well as industry partners like Brammer Bio and Fujifilm Diosynth Biotechnologies, to enhance its gene therapy initiatives. Founded in 2013 and headquartered in Cambridge, Massachusetts, Voyager Therapeutics is committed to addressing unmet medical needs in the central nervous system.

Diurnal Group plc is a specialty pharmaceutical company based in Cardiff, United Kingdom, that specializes in developing hormone therapeutics for the treatment of chronic endocrine conditions. Founded in 2004, the company aims to provide innovative solutions for conditions such as congenital adrenal hyperplasia, adrenal insufficiency, hypogonadism, and hypothyroidism. Diurnal's notable products include Alkindi, a replacement therapy for pediatric adrenal insufficiency, and Chronocort, which has completed Phase III clinical trials for adrenal insufficiency and congenital adrenal hyperplasia. The company is also advancing its early-stage pipeline with products such as Native Oral Testosterone for hypogonadism, a siRNA therapy for adrenocorticotropin-dependent Cushing's syndrome, and a modified-release preparation of T3 hormone for hypothyroidism. Diurnal's research emphasizes circadian-based endocrinology, focusing on physiological treatment regimes to address the needs of patients with rare and chronic endocrine disorders.

Xenon Pharmaceuticals Inc. is a clinical-stage biopharmaceutical company based in Burnaby, Canada, focused on developing innovative therapeutics for neurological disorders, including rare central nervous system conditions. The company's pipeline includes XEN496 and XEN1101, both Kv7 potassium channel modulators currently in Phase II clinical trials for the treatment of epilepsy. Additionally, Xenon is advancing XEN901, a selective Nav1.6 sodium channel inhibitor, and XEN007, a central nervous system-acting calcium channel modulator, both in Phase I clinical trials. Through its proprietary discovery platform known as Extreme Genetics, Xenon leverages genetic insights from families with severe phenotypes to identify and characterize single-gene defects that may serve as drug targets. The company has also established a collaboration agreement with Neurocrine Biosciences to develop first-in-class treatments for epilepsy, highlighting its commitment to addressing both rare and more prevalent neurological diseases.

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