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great healthworks is a Fort Lauderdale, FL-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
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.
Inspirion Biosciences protects the advancement of science, the safety and security of researchers, and the health of surrounding communities through integrated solutions in risk strategy. Learn more here.
aTyr is a biotherapeutics company engaged in the discovery and development of innovative medicines based on novel biological pathways. aTyr’s research and development efforts are concentrated on a newly discovered area of biology, the extracellular functionality and signaling pathways of tRNA synthetases. aTyr has built a global intellectual property estate directed to a potential pipeline of protein compositions derived from 20 tRNA synthetase genes and their extracellular targets. aTyr’s primary focus is ATYR1923, a clinical-stage product candidate which binds to the Neuropilin-2 receptor and is designed to down-regulate immune engagement in inflammatory lung diseases.
NGM Bio is a research-driven, clinical-stage biopharmaceutical company with a passion to improve the lives of those struggling with serious diseases. Our expertise in drug discovery and translational development, coupled with the significant resources from our partnership with Merck, is driving development of transformational medicines that dramatically improve human health. Our initial R&D focus has been to develop a portfolio of high-impact therapeutics for the underserved patient populations who suffer from cardio-metabolic and liver diseases. We believe that our research expertise – comprehensive and grounded in fundamental, interrelated mechanisms of human biology – is capable of driving the development of new therapies for a wide range of other disease areas as well, such as cancer. NGM Bio`s comprehensive approach to drug discovery focuses on elucidating the biology of hormones, cell receptors and associated ligands. As we investigate these novel biological mechanisms, we assemble a deep understanding of the relationship between the structure and function of proteins of interest. Coupled with our experience in protein and antibody engineering, our approach enables the efficient and targeted generation of potential first-in-class therapeutics with optimized pharmacologic profiles.