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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.
Celcuity is a biotech company developing state-of-the-art cell-based assays using our proprietary functional cellular analysis platform.
EduQuest is a Clarksburg, MD-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Sierra Oncology is a clinical stage drug development company advancing targeted therapeutics for the treatment of patients with unmet medical needs in hematology and oncology. Our lead drug candidate, momelotinib, is a potent, selective and orally-bioavailable JAK1, JAK2 & ACVR1 inhibitor with a differentiated therapeutic profile in myelofibrosis encompassing a range of meaningful anemia benefits, including eliminating or reducing the need for frequent blood transfusions, as well as achieving substantive spleen and constitutional symptom control. We are also advancing SRA737 and SRA141. SRA737, is a potent, highly selective, orally bioavailable small molecule inhibitor of Checkpoint kinase 1 (Chk1). We are pursuing an innovative development plan for SRA737, which is currently being evaluated in two Phase 1/2 clinical trials in patients with advanced cancer. SRA737-01 is intended to evaluate SRA737`s potential to induce synthetic lethality as monotherapy, while SRA737-02 is intended to evaluate the combination of SRA737 potentiated by subtherapeutic, low dose gemcitabine. Concurrently, we are conducting preclinical research evaluating SRA737 in combination with other DDR-targeted agents, including PARP inhibitors, as well as with immuno-oncology therapeutics, that may guide a potential next wave of clinical development for our asset, possibly further broadening its therapeutic utility. SRA141, a potent, selective, orally bioavailable small molecule inhibitor of cell division cycle 7 kinase (Cdc7). Cdc7 is a key regulator of DNA replication and is involved in the DDR network, making it a compelling emerging target for potential treatment across a broad range of tumor types. An Investigational New Drug Application (IND) submission to the U.S. Food and Drug Administration (FDA) is being prepared in order to commence clinical trials with this drug candidate.
Nova Biologics. is a Carlsbad, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.