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Mesoblast is a world leader in developing innovative cellular medicines. We have established what we believe is the industry`s most clinically advanced and diverse portfolio of cell-based products with five programs, two of which are partnered, in active Phase 3 clinical studies or Phase 3-ready, and four programs in Phase 2. All our clinical programs target significant, under-served therapeutic areas including cardiac diseases, spine orthopedic disorders, oncology and hematology diseases, and immune-mediated and inflammatory conditions.
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.
Aruvant`s mission is to bring hope to patients living with rare diseases by developing life changing and potentially curative gene therapies, with a near-term focus on sickle cell disease. Aruvant is a part of the Roivant family of companies. We have a talented team with extensive experience in the development, manufacturing and commercialization of gene therapy products. We have an active research program with a lead product candidate in development for individuals suffering from sickle cell disease (SCD). Our lead product candidate, ARU-1801, an investigational lentiviral gene therapy, is currently in a clinical trial as a potential one-time curative treatment for SCD. Preliminary clinical data from the ongoing Phase 1/2 study demonstrated durable efficacy in reducing the negative impacts of SCD. We are preparing for the launch of our pivotal clinical trial in SCD. We continue to look for new people to add to our talented team.
Ortho-McNeil Pharmaceutical, Inc. is a Raritan, NJ-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Providence is a clinical-stage biotechnology company pioneering mRNA therapeutics and vaccines with a focus on infectious diseases and oncology. Initially founded as a cancer vaccines company in 2015, in response to a worldwide need for a COVID-19 vaccine, Providence expanded its focus beyond oncology therapies to develop an mRNA vaccine for COVID-19. Providence works with multiple industry collaborators, universities, nongovernmental agencies and multiple arms of the Government of Canada to discover and develop vaccines and treatments for infectious diseases and cancer. This has resulted in the development of an mRNA vaccine platform that includes a proprietary design algorithm and proprietary, scalable manufacturing processes.