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Stealth BioTherapeutics is a privately held biopharmaceutical company committed to bringing mitochondria therapies to patients to treat both common and rare diseases. As a key common element in a variety of serious, debilitating diseases, mitochondria – the cell’s energy source – offer a promising, and yet untapped target to modify diseases with significant unmet treatment needs. Stealth is expanding its clinical development program to additional therapeutic areas, including cardio-renal diseases, ophthalmic disorders and orphan mitochondrial diseases. By defining the broad potential of its mitochondrial platform and therapies, Stealth is leading mitochondrial medicine.
Anokion, a spin-off from the Ecole Polytechnique Fédérale de Lausanne (EPFL), is focused on applying the company`s antigen-specific immune tolerance technology to reduce the immunogenicity of therapeutic proteins and to treat autoimmune and allergic diseases. As a platform technology, Anokion`s approach to antigen-specific tolerance can be translated to virtually any protein in numerous clinical indications.
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.
Gel-Del is a Saint Paul, MN-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
QED Therapeutics, a subsidiary of BridgeBio Pharma, is a biotechnology company focused on precision medicine for FGFR-driven disorders.