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GenSight Biologics is is a clinical-stage biotechnology company discovering and developing novel therapies for neurodegenerative retinal diseases and diseases of the central nervous system. GenSight Biologics` pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics for retinitis pigmentosa, to help preserve or restore vision in patients suffering from severe degenerative retinal diseases. GenSight Biologics` lead product candidate, GS010, is in Phase III trials in Leber`s Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible low vision and legal blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics` product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.
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.
Mission Bio is a company of passionate people dedicated to solving complex biological problems with precision engineering, innovative biochemistry, and supported bioinformatics.
The combined characteristics of a modern machine (with a touch of humanity) and mythical creature symbolizes Lykan Bioscience™ as a company. The team at Lykan Bioscience operates with a highly advanced way of thinking. We offer our clients speed, agility, and control while accelerating their cell and gene therapy products from clinical through to commercial manufacturing. As part of your team, our Manufacturing Services Organization™ (MSO) will transform what is traditionally offered in the CGT industry, as we guide you through your manufacturing journey. Our mission is to partner with our clients, to maximize patient accessibility and expedite time to market for cell-based therapies.
Odonate Therapeutics™ is dedicated to the development of best-in-class therapeutics that improve and extend the lives of patients with cancer. Our initial focus is on the development of tesetaxel, an investigational, orally administered taxane. We are conducting our multinational, multicenter, randomized, Phase 3 study in locally advanced or metastatic breast cancer (MBC), known as CONTESSA. Our goal is to develop an effective chemotherapy choice for patients that provides quality-of-life advantages over current alternatives.