| Name | Title | Contact Details |
|---|---|---|
Sarah Yuan |
Chief Technical Operations Officer | Profile |
Martha Rook |
Chief Technical Operations Officer | Profile |
Susan Drapeau |
Vice President and Head of Device and Delivery Engineering | Profile |
GFI LAB is a Rockwood, PA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Vanda is a specialty pharmaceutical company focused on the development and commercialization of novel therapies to address high unmet medical needs and improve the lives of patients.
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.
Brain diseases collectively represent one of the greatest challenges of our generation, affecting upwards of 1.5 billion people. However, treatments for many of the most prevalent brain disorders have been dominated by a single class of drugs, such as serotonin targeting antidepressants for MDD, leaving patients with a high degree of unmet medical need given the lack of diverse treatment options and mechanisms of action. We are taking a fundamentally different approach to the way treatments for brain diseases are developed. Our pipeline is comprised of programs pursuing therapeutically relevant targets implicated in Central Nervous System (CNS) diseases, each targeting a novel mechanism of action with best-in-class pharmacology. Our work is supported by our Precision Toolbox, which integrates a suite of translational, clinical, and computational tools to generate insights that can enable precision medicine approaches. Neumora is relentless in its commitment to discovering, developing and commercializing novel therapies for the 1.5 billion people living with brain diseases.
Nitrome Biosciences has discovered and identified a new class of enzymes.