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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.
Dendritic NanoTechnologies, Inc. is a Mount Pleasant, MI-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
AJAX Health accelerates medical innovation through capital and capabilities. Created in partnership with private equity leaders KKR and Aisling Capital, AJAX invests and takes an operating role in innovative medical companies that improve lives and bring speed, simplicity and cost efficiency to health care.
Adolor Corporation is a Exton, PA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Loxo Oncology is committed to the discovery, development, and commercialization of targeted cancer therapies with best-in-class potential. Our diverse pipeline reflects the convergence of proven therapeutic technologies with emerging insights into the underlying susceptibilities of cancer and drug resistance. It is an exciting time to develop cancer drugs. Diagnostic methods are yielding strong clues about which cancer features are actionable, “driver alterations,” in contrast to those merely along for the ride as “passenger alterations.” In the last five years, these insights have transformed the standard of care in melanoma, lung cancer and other cancers. Picking a target is just the first step in building a cancer drug. Good chemists build good drugs. The physical properties of a compound have much to do with its ultimate success or failure. Is it stable? Is it soluble? Is it absorbed? Does it reach its target? Does it engage the target? Does it cause toxicity elsewhere in the body before it achieves maximum efficacy in the cancer? In our opinion, drugs that achieve excellent exposure in the body and are highly specific for their intended targets have the highest chances of success. Once an attractive target is selected and a drug with best-in-class properties is constructed, setting up the right clinical development plan is of utmost importance. Our goal is to generate a clinical path that reflects the underlying scientific hypothesis that made the drug interesting in the first place. This often means developing the drug in a population of patients whose tumors possess a specific vulnerability targeted by the drug. Loxo Oncology was built around a team of full-time professionals and engaged scientific advisors who are aligned in choosing the most actionable targets, and pursuing them through disciplined clinical trial approaches. Our license and collaboration agreement with Array Biopharma and our experienced team allow us to construct drugs with best-in-class properties from the ground up.