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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.
BioAgilytix is a leading bioanalytical contract research organization (CRO) that supports the development of novel therapeutic biologics and biosimilars. Located in Research Triangle Park, North Carolina, BioAgilytix specializes in large-molecule bioanalysis providing critical insight into the safety, efficacy, and mechanism of action of drug candidates throughout the clinical trial process. Specifically, BioAgilytix offers assay development, validation, and sample analysis services for immunoassays and cell-based assays under non-GLP, spirit-of-GLP, and GLP, as well as product release testing under GMP. BioAgilytix is an engaged and energetic partner committed to delivering exceptional value and veteran expertise through a collaborative process that consistently results in successful projects.
TYME is an emerging biotechnology company developing cancer therapeutics that are intended to be broadly effective across tumor types and have low toxicity profiles.
Orna Therapeutics is a biotechnology company dedicated to designing and delivering a new class of fully engineered circular RNA (oRNA) therapeutics with the potential to change the way we treat disease. Orna`s proprietary platform combines novel technology to create oRNAs that drive protein expression with validated and unique delivery solutions.
Concuity is a Vernon Hills, IL-based company in the Healthcare, Pharmaceuticals, and Biotech sector.