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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.
Improving lives by enhancing precision cancer diagnostics.
Expansion Therapeutics is a drug discovery and development company pursuing the vast potential of small molecule medicines for RNA-mediated diseases.
We have developed a high-sensitivity next-generation sequencing-based system for the detection of cancer mutations in miniscule concentrations of circulating cell-free DNA (cfDNA) in blood. Our technology addresses the significant barriers associated with harnessing the information contained in cfDNA by combining our proprietary techniques in molecular biology and computational algorithm for error suppression. With our technology, nanogram quantities of highly-fragmented cfDNA can be amplified more than 1000-fold, and a panel of tens to hundreds of cancer-related genes can be screened with a sensitivity at the single-digit molecular level.
Sherlock Biosciences aims to disrupt molecular diagnostics with better, faster, affordable tests. With our unique Engineering Biology platforms, we are on the cusp of solving challenges ranging from faster pathogen detection and simpler testing for cancer to improved food safety. We envision a world where our products will enable users to make more effective decisions in any environment, whether in hospitals, industrial settings, the developing world, or at home. Our team and founders include Engineering Biology pioneers with world-leading expertise in CRISPR and Synthetic Biology, diagnostic industry veterans, and disease-area authorities. Together, they provide an unparalleled set of capabilities that are transforming molecular diagnostics in clinical and non-clinical settings.