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Pareto Biotechnologies has pioneered development of a revolutionary synthetic biology platform that produces naturally derived, high value “designer molecules.” Pareto’s technology is the result of over a decade of academic and private sector research and intellectual property focused on one particular polyketide pathway. Building on Dr. Joe Noel’s groundbreaking research at the Salk Institute, this pathway – previously dismissed as the “poor man’s pathway” – is the foundation of Pareto’s platform, capable of generating known products and novel molecules. In the near term, Pareto will introduce “designer molecules” as cosmetics, flavors and fragrances including molecules with properties unlike anything previously experienced. In the long term, Pareto’s molecules will be designed to enhance nutraceuticals, pharmaceuticals, and industrial commodities. Pareto’s potential is limitless. Join us as we ignite a molecular revolution
AVROBIO`s vision is to bring personalized gene therapy to the world. We aim to prevent, halt or reverse disease throughout the body with a single dose of gene therapy designed to drive durable expression of therapeutic protein, even in hard-to-reach tissues and organs including brain, muscle and bone. Our ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease. AVROBIO is powered by our industry leading plato® gene therapy platform, our foundation designed to deliver gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario.
Bio-Kinetic Clinical Applications is a Springfield, MO-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
NICO gives hope to patients with primary and secondary brain tumors, cysts and hemorrhagic strokes.
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.