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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.
Cogent Biosciences is a biotechnology company developing real solutions to treat genetically driven diseases. With a focus on rational drug discovery and development, we are leveraging validated biology to advance precision therapies designed to address the true underlying drivers of disease and provide real hope for patients. Cogent`s lead therapeutic candidate, PLX9486 (expected to be named CGT9486 in the future), is a precision kinase inhibitor designed to selectively and potently inhibit the KIT D816V mutation. This mutation is responsible for driving a rare and serious condition called Systemic Mastocytosis which can severely impact many different tissues and organs in the body. We are also studying PLX9486 to treat advanced gastrointestinal stromal tumors (GIST), which have a strong dependence on oncogenic KIT signaling.
Tarcine is a Thousand Oaks, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Sengenics is a functional proteomics company that leverages its patented KREX technology to discover autoantibody biomarker signatures for prediction of drug response and severe immune-related adverse events (irAEs). KREX™ was invented and patented by Professor Jonathan Blackburn whilst he was a member of the faculty at the University of Cambridge. Jonathan is the CSO of Sengenics. The key application of KREX™ is the discovery of autoantibody biomarker signatures for prediction of drug response and severe immune-related adverse events (irAEs). KREX™ can also be used to identify autoantibody biomarkers that may be used to diagnose cancer, autoimmune or neurodegenerative conditions years before conventional diagnostic tests. Some autoantibodies that are identified as diagnostic biomarkers may be protective and have potential in themselves as therapeutic biomolecules.
Ceribell is a fast growing venture-backed medical device start-up company founded by two faculty members at Stanford University. Our primary mission is to improve neurological care for emergency and ICU patients. Ceribell is focused on making EEG widely accessible, more efficient, and more cost-effective to improve the diagnosis and treatment of patients at risk for seizures. The System can be set up by any healthcare provider within minutes. Now with Clarity, Ceribell offers 24/7 continuous bedside EEG monitoring and alert.