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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.
Provention Bio, Inc. is a clinical-stage biopharmaceutical company developing novel therapeutics aimed at intercepting and preventing immune-mediated diseases. -We seek technologies or targets designed to predict, preempt or intervene before immune disease begins, re-appears or progresses. -We are creating a new biopharmaceutical category for autoimmunity. We leverage a transformational drug development strategy that sources, repositions and advances candidates that: -Target the interception or prevention of immune-mediated disease -Have been underdeveloped or deprioritized because of insufficient clinical trial efficacy or for strategic reasons -Have demonstrated proof-of-mechanism by preventing or intercepting immunopathologic pathways We believe that our deep understanding of immune-mediated pathophysiology, experience in translational medicine, and expertise in the design of rapid go/no-go clinical trials enables us to identify and evaluate potential therapeutic candidates for acquisition or in-licensing.
RAPT Therapeutics is a clinical stage immunology-based biopharmaceutical company focused on discovering, developing and commercializing oral small molecule therapies for patients with significant unmet needs in oncology and inflammatory diseases. Utilizing its proprietary discovery and development engine, the company develops highly selective small molecules that are designed to modulate the fundamental immune responses underlying these diseases. RAPT has rapidly discovered and advanced two unique drug candidates each targeting CCR4, including our lead oncology drug candidate, FLX475, now in clinical development and our lead inflammation drug candidate, RPT193, expected to enter the clinic in the second half of 2019. The company is also pursuing other discovery targets including GCN2 and HPK1 for the treatment of cancer.
Eastern Maine Medical Center Federal Credit Union aka EMMC FCU is a Bangor, ME-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Cyclacel Pharmaceuticals, Inc. is a biopharmaceutical company developing oral therapies that target the various phases of cell cycle control for the treatment of cancer and other serious diseases. Professor Sir David Lane, a recognized leader in the field of tumor suppressor biology, who discovered the p53 protein, founded the Company in 1996. In 1999, Cyclacel Pharmaceuticals was joined by Professor David Glover, a recognized leader in the mechanism of mitosis or cell division, who discovered, among other cell cycle targets, the mitotic kinases, Polo and Aurora, enzymes that act in the mitosis phase of the cell cycle. Sapacitabine (CYC682), Cyclacel`s most advanced product candidate, is the subject of SEAMLESS, a Phase 3 trial, which has completed enrollment and is being conducted under an SPA with the FDA as front-line treatment for acute myeloid leukemia (AML) in the elderly, and other indications including myelodysplastic syndromes (MDS). Cyclacel`s pipeline includes an oral regimen of seliciclib in combination with sapacitabine in a Phase 1 study of patients with Homologous Recombination (HR) repair-deficient breast, ovarian and pancreatic cancers, including BRCA positive tumors, and CYC065, a novel CDK2/9 inhibitor in a Phase 1 study of patients with solid tumors with potential utility in both hematological malignancies and solid tumors. Cyclacel`s strategy is to build a diversified biopharmaceutical business focused in hematology and oncology based on a development pipeline of novel drug candidates. Cyclacel Pharmaceuticals` corporate headquarters are in Berkeley Heights, New Jersey, where our business development and medical and regulatory functions are also located. The company`s primary research facility is located in Dundee, Scotland. Dundee is the main location of our translational research and preclinical activities.