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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.
AbSci is a global leader in biomanufacturing technologies. The company`s SoluPro™ expression platform achieves ultra-high yields of soluble and active protein products and enables ultra-high efficiency purification processes.
Precision Diagnostics, a leader in clinical laboratory testing and medication adherence monitoring, is transforming healthcare through the delivery of comprehensive, insightful clinical data that can help improve patient outcomes and manage costs. Spec...
Genprex (Nasdaq: GNPX) is a clinical-stage gene therapy company focused on developing life-changing technologies for patients with cancer and diabetes. Genprex`s technologies are designed to administer disease-fighting genes to provide new treatment options for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches for patients with cancer and other serious diseases.
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.