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Alexion is a global biopharmaceutical company focused on developing and delivering life-transforming therapies for patients with devastating and rare diseases. Our shared purpose to serve patients in need drives us to find answers, change the world, and create a legacy. Alexion`s three highly innovative therapies treat patients with four severe and ultra-rare diseases, and we are committed to developing additional therapies that have the potential to transform patients` lives. Today, Alexion is advancing the most robust rare disease pipeline in the biotech industry, which, in addition to our complement and metabolic clinical programs, includes more than 30 diverse preclinical programs across a range of therapeutic modalities. Alexion has more than 3,000 employees serving patients in 50 countries. Our global headquarters and research operations are based in New Haven, Connecticut and our EMEA headquarters are in Zürich, Switzerland. We also have global supply chain and operations headquarters in Ireland, manufacturing facilities in the United States, and local and regional operations in countries around the world. Alexion was ranked number 5 by Science magazine in its Top Employer Survey and has also been ranked as one of the top companies on the Forbes "World`s Most Innovative Companies" list every year since 2012.
Kura Oncology is a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer. Our pipeline consists of small molecule drug candidates that target cancer signaling pathways where there is a strong scientific and clinical rationale to improve outcomes by identifying those patients most likely to benefit from treatment. Our lead drug candidate is tipifarnib, a farnesyl transferase inhibitor, which is currently being studied in multiple Phase 2 clinical trials in solid tumor and hematologic indications. Our pipeline also includes KO-947, an ERK inhibitor, currently in a Phase 1 dose-escalation trial, and KO-539, a menin-MLL inhibitor, currently in preclinical development.
Adverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs for serious ocular and rare diseases. Adverum is evaluating its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of its lead indication, wet age-related macular degeneration.
The mission of 4D Molecular Therapeutics is to design and develop transformative gene therapy products using our proprietary technology to create novel adeno-associated virus variants to be utilized as treatments for serious unmet medical conditions. Our products are unlocking the full potential of gene therapy to treat, and potentially cure, genetic diseases. A large number of patients and diseases, who previously were not treatable by gene therapy, will be addressable by 4D products. 4D has one of the deepest and most diverse product pipelines in the gene therapy field. Adeno-associated virus (AAV) vectors have emerged as a favored delivery vehicle for gene therapy in the human body. They can deliver the genes for therapeutic proteins to accessible tissues in the body and are generally considered safe. Several AAV gene therapy products are in late-stage clinical development, and one product is approved in the EU (Glybera, Uniqure). However, these first-generation AAV vectors have limited utility for the majority of diseases. Many of these common AAV were discovered as laboratory contaminants and monkey infections, for example; they have not been customized as targeted medicines. In contrast, 4D customized AAV vectors, and the products made from them, are designed to unlock the full potential of gene therapy. Through 4D`s Therapeutic Vector Evolution, we are able to create customized vectors solutions to overcome the hurdles identified with first-generation AAV vectors. 4D is creating the ultimate gene therapy products to cure genetic diseases using new customized delivery vehicles (vectors) to shuttle genes into the cells in any organ in the body. This 4D “library” of vectors will soon include several for the liver, several for the brain, several for the heart, the eye, muscle, and so on. Doctors will be able to hand pick a vector for any patient based on the organ that is diseased.
Tarcine is a Thousand Oaks, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.