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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.
RuiYi is focused on the discovery and development of novel biologic therapeutics that have the potential to be disruptive globally. In addition to RYI-018 and RYI-008, RuiYi has a growing pipeline of monoclonal antibodies from its internal discovery efforts utilizing a series of the Company's technologies, including the iCAPS (intramembranous Conformation Antigen Presenting System). Targets include a select subset of G protein coupled receptors (GPCR), where specificity in binding is critical but has proven difficult to achieve with small molecule modulators. RuiYi's executive management team has offices in La Jolla, California, and RuiYi's discovery efforts and research facility are located in in the Zhangjiang Hi-Tech Park in Pudong, Shanghai, China.
Asterias Biotherapeutics is a leading biotechnology company in the emerging field of regenerative medicine. The company’s proprietary cell therapy programs are based on its immunotherapy and pluripotent stem cell platform technologies. Asterias is presently focused on advancing three clinical-stage programs which have the potential to address areas of very high unmet medical need in the fields of oncology and neurology.
Aerpio Therapeutics is a Cincinnati, OH-based company in the Healthcare, Pharmaceuticals, and Biotech sector. Aerpio Therapeutics is a biopharmaceutical company focused on the development of novel therapeutics for vascular disorders with a concentration on diseases of the eye.
Aldeyra Therapeutics, Inc., is a biotechnology company focused primarily on the development of products to treat diseases thought to be related to endogenous free aldehydes, a naturally occurring class of toxic molecules. The company has developed NS2, a product candidate designed to trap free aldehydes. Aldeyra plans to begin clinical testing of NS2 in 2014 for the treatment of Sjögren-Larsson Syndrome and acute anterior uveitis. NS2 has not been approved for sale in the U.S. or elsewhere.