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Atsena Therapeutics is a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness.
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.
ARIAD is a pharmaceutical company whose vision is to transform the lives of cancer patients with breakthrough medicines.
Abivax is developing therapies that modulate the body`s natural immune system machinery. A clinical-stage company, Abivax leverages its immune enhancing and antiviral platforms to develop drug candidates to treat inflammatory diseases, viral diseases and liver cancer. Abivax is based on innovative research driven by the idea of modulating the body`s natural immune system mechanisms to treat diseases.
The approach is an autologous, cell-based treatment that activates the full array of patient-specific antigens, resulting in both an innate and adaptive immune response. The company`s lead program is in glioblastoma multiforme (GBM), an extremely aggressive form of brain cancer with a significant unmet need. In parallel, the company is also progressing the platform in a select number of additional solid tumors where the unmet need remains great and where initial immunotherapy approaches have often failed to provide a significant benefit.