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AVROBIO`s vision is to bring personalized gene therapy to the world. We aim to prevent, halt or reverse disease throughout the body with a single dose of gene therapy designed to drive durable expression of therapeutic protein, even in hard-to-reach tissues and organs including brain, muscle and bone. Our ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease. AVROBIO is powered by our industry leading plato® gene therapy platform, our foundation designed to deliver gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario.
At Mineralys Therapeutics we aim to redefine hypertension diagnosis, management and treatment. We are driven to break the “trial and failure” approach to hypertension management and bring a personalized approach to the management of hypertension.
Zynerba (NASDAQ: ZYNE) is dedicated to improving the lives of people with rare and near rare neuropsychiatric disorders where there is a high unmet medical need by pioneering the development and commercialization of next-generation pharmaceutically-produced cannabinoid therapeutics formulated for transdermal delivery. Cannabinoids interact with specific receptors throughout the body to produce pharmacologic effects, primarily in the CNS and immune systems. Transdermal delivery has the potential to reduce adverse effects associated with oral dosing because it allows the drug to be absorbed through the skin directly into the bloodstream, avoiding the gastrointestinal tract and lessening the opportunity for GI-related adverse events. It also avoids first-pass liver metabolism, potentially enabling lower dosage levels of active pharmaceutical ingredients with a higher bioavailability and an improved safety profile. Zynerba`s lead patent-protected product candidate in clinical development is ZYN002: ZYN002 is the first and only pharmaceutically-produced cannabidiol (CBD) formulated as a permeation-enhanced gel for transdermal delivery. Through a proprietary combination of our pharmaceutically-produced CBD and permeation enhancers, we believe we can effectively deliver CBD through the layers of the epidermis and into the circulatory system. The company is currently assessing ZYN002 in Fragile X syndrome and certain adult and pediatric refractory epilepsies. These include adult refractory focal epilepsy and developmental and epileptic encephalopathies (DEE), a heterogeneous group of rare and ultra-rare epilepsy syndromes associated with severe cognitive impairment and behavioral disturbances.
Adventus Group is a Freeport, IL-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
A W Research Laboratories is a Brainerd, MN-based company in the Healthcare, Pharmaceuticals, and Biotech sector.