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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.
The future is different. We are building sensing and thinking machines, with synthetic neurobiology at its core. Machines with extremely dense sensing and computation based on carbon. In our systems, everything can be engineered with care for sustainab...
BioCryst Pharmaceuticals, Inc. is one of the leading companies in the Healthcare, Pharmaceuticals, and Biotech sector.
At Spark Therapeutics, we don`t follow footsteps. We create the path. We were born of innovation, springing from the curiosity, imagination and dedication of remarkable scientists and healthcare visionaries. Our mission is seemingly impossible to others, but not to us: Challenge the inevitability of genetic disease by discovering, developing and delivering treatments in ways unimaginable – until now. Since our founding, we have been committed to bringing a wide range of expertise to build a fully integrated gene therapy company focused on inherited retinal diseases (IRDs), neurodegenerative diseases, as well as diseases that can be addressed by targeting the liver, such as hemophilia. We are seeking talented individuals with diverse experiences, abilities and interests who have the curiosity, courage and drive to reimagine a new health care paradigm. Join us on a journey through uncharted territory – seeking to bring gene therapy for genetic diseases to people worldwide. The resilience of the people we serve is our inspiration to break barriers, as we strive to turn genes into medicine for those with inherited diseases. Spark is a member of the Roche Group.
NuProbe brings together cutting-edge innovations in molecular engineering with leading scientific expertise and management to take on one of the world`s greatest challenges — non-invasive early detection of rare genetic variants in patients. NuProbe will take a multi-platform approach to apply these breakthrough technology capabilities in precision medicine to cancer diagnostics, NIPT, and infectious disease screening, building on scientific findings licensed by Wyss Institute, Harvard University.