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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.
Onconova Therapeutics, Inc. (NASDAQ: ONTX) is a clinical-stage biopharmaceutical company focused on discovering and developing novel small molecule drug candidates to treat cancer. Using a proprietary chemistry platform, Onconova has created an extensive library of targeted anti-cancer agents designed to work against specific cellular pathways that are important in cancer cells, while causing minimal damage to normal cells. Onconova has three product candidates in clinical trials and six active pre-clinical programs. Onconova seeks to develop products that improve the outcome for cancer patients by directly addressing the disease, its recurrence, and the burden of side-effects associated with current methods of treatment.
HealthScape is a Tampa, FL-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Celladon is a privately held biotechnology company founded with the goal of developing molecular therapies for cardiovascular diseases. Breakthroughs in the basic understanding of the cardiovascular networks combined with powerful new technologies have provided the foundation for new, rational, and technologically sophisticated approaches to cardiovascular treatments. Our first product candidate is designed to target the key enzyme deficiency in advanced heart failure which regulates calcium cycling and contractility in heart muscle cells.
Kythera Biopharmaceuticals, Inc. is one of the leading companies in the Healthcare, Pharmaceuticals, and Biotech sector.