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Ambys Medicines is a biotechnology company focused on discovering and developing regenerative and restorative therapies for people with advanced liver disease. Founded by world-renowned experts in liver disease and regenerative medicine, Ambys is pioneering the application of novel modalities including cell and gene therapy, and gain-of-function drug therapy, to meet the urgent need for treatments that have the potential to restore liver function and prevent the progression to liver failure across multiple liver diseases that are untreatable or poorly treated today. Ambys was launched in 2018 by Third Rock Ventures and Takeda Pharmaceuticals with $140M in starting capital and is headquartered in South San Francisco, Calif.
Minerva Neurosciences, Inc. (NASDAQ: NERV) is a clinical-stage biopharmaceutical company focused on the development of a portfolio of product candidates to treat central nervous system (CNS) diseases. Our goal is to transform the lives of patients with improved therapeutic options. Leveraging extensive domain expertise, we have identified and acquired or in-licensed a portfolio of development-stage proprietary compounds with what we believe are innovative mechanisms of action. Our executive management team includes globally recognized specialists in central nervous system diseases addressing significant unmet medical needs. Our strategy is based on the following key principles: • Develop differentiated products based on biological and clinical insights related to the unmet needs of patients; • Conduct proof-of-principle trials in geographic areas with well characterized and validated patient populations where we have access to highly trained physicians with experience in conducting CNS-related trials; • Leverage the randomized, double-blind, placebo-controlled data from these trials to advance the clinical development of our products in multiple regulatory jurisdictions; • Selectively explore collaborations with leading pharmaceutical companies to maximize the value of our current product candidate portfolio, particularly in connection with the initiation of pivotal Phase III clinical trials and subsequent regulatory review, approval and commercialization; • Apply our management team`s expertise and current intellectual property portfolio to identify and explore additional indications relating to our current portfolio of compounds and to acquire additional product candidates
Amira Pharmaceuticals is a San Diego, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Mavupharma is developing non-nucleotide, conditional modulators of the STING pathway to treat cancer and infectious disease.
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.