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Amyloid diseases are a diverse group of disorders, characterized by the deposition of amyloid protein fibrils in vital organs and tissues. There are approximately 30 different types of amyloidosis, each resulting from the misfolding and aggregation of a specific protein, and they are all severely debilitating, progressive, and often fatal.
Cellectar Biosciences is developing agents to detect, treat and monitor a broad spectrum of cancers. Using a novel phospholipid ether analog (PLE) platform technology as a targeted delivery and retention vehicle, Cellectar's compounds are designed to be selectively taken up and retained in cancer cells including cancer stem cells. With the ability to attach both imaging and therapeutic agents to its proprietary delivery platform, Cellectar has developed a portfolio of product candidates engineered to leverage the unique characteristics of cancer cells to "find, treat and follow" malignancies in a highly selective way. I-124-CLR1404 is a small-molecule, broad-spectrum, cancer-targeted PET imaging agent currently being evaluated in a Phase II glioblastoma imaging trial. Additionally, multiple investigator-sponsored Phase I/II clinical trials are ongoing across 11 solid tumor indications. I-131-CLR1404 is a small-molecule, broad-spectrum, cancer-targeted molecular radiotherapeutic that delivers cytotoxic radiation directly and selectively to cancer cells including cancer stem cells. A Phase Ib dose-escalation trial of I-131-CLR1404 in patients with advanced solid tumors was completed in the first quarter of 2014 and results have been submitted to the American Society of Clinical Oncology (ASCO) 2014 Annual Meeting. CLR1502 is a preclinical, cancer-targeted, non-radioactive optical imaging agent for intraoperative tumor margin illumination and non-invasive tumor imaging.
Brain diseases collectively represent one of the greatest challenges of our generation, affecting upwards of 1.5 billion people. However, treatments for many of the most prevalent brain disorders have been dominated by a single class of drugs, such as serotonin targeting antidepressants for MDD, leaving patients with a high degree of unmet medical need given the lack of diverse treatment options and mechanisms of action. We are taking a fundamentally different approach to the way treatments for brain diseases are developed. Our pipeline is comprised of programs pursuing therapeutically relevant targets implicated in Central Nervous System (CNS) diseases, each targeting a novel mechanism of action with best-in-class pharmacology. Our work is supported by our Precision Toolbox, which integrates a suite of translational, clinical, and computational tools to generate insights that can enable precision medicine approaches. Neumora is relentless in its commitment to discovering, developing and commercializing novel therapies for the 1.5 billion people living with brain diseases.
Castle Creek Biosciences, Inc. is a clinical-stage cell and gene therapy company focused on developing and commercializing disease-modifying therapies for patients suffering from rare diseases for which there is a lack of available treatment options. The company`s proprietary autologous fibroblast platform potentially allows for the development of personalized, targeted and redosable cell-based gene therapy product candidates for monogenic and chronic disorders. The company`s most advanced product candidate, dabocemagene autoficel (D-Fi), is currently being evaluated in a Phase 3 clinical trial for the localized treatment of chronic wounds due to recessive dystrophic epidermolysis bullosa (RDEB). The company is also currently evaluating FCX-013 in a Phase 1/2 clinical trial for the treatment of moderate to severe localized scleroderma. In addition, Castle Creek Biosciences is pursuing discovery and potential development of early-stage novel product candidates with the goal of expanding its robust pipeline into other rare diseases and broader indications where there are significant unmet needs. The company operates an in-house, commercial-scale manufacturing facility in Exton, Pennsylvania that benefits from the validated systems and processes previously implemented at the site for manufacture of an FDA-approved cell therapy product. Castle Creek Biosciences, Inc. is a portfolio company of Paragon Biosciences, LLC.
Aegerion Pharmaceuticals is a biopharmaceutical company dedicated to the development and commercialization of innovative therapies for patients with debilitating rare diseases.