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Regado Biosciences, Inc., is a biopharmaceutical company focused on the discovery and development of novel, oligonucleotide-based actively controllable therapeutics. Our initial focus is on applications in the acute and sub-acute cardiovascular therapeutic area. The company's lead product candidate, REG1, is a two-component system consisting of pegnivacogin, an anticoagulant aptamer specifically targeting coagulation Factor IXa, and its complementary oligonucleotide active control agent, anivamersen. REG1 is currently being evaluated in the REGULATE-PCI trial, a worldwide Phase 3 trial enrolling 13,200 patients with acute coronary syndromes (ACS) undergoing percutaneous coronary intervention (PCI), a hospital-based procedure used to mechanically open or widen obstructed coronary arteries. Regado's actively controllable product candidates have the potential to improve patient outcomes and enhance the patient experience, provide direct therapeutic control to physicians and reduce overall treatment costs.
Cogent Biosciences is a biotechnology company developing real solutions to treat genetically driven diseases. With a focus on rational drug discovery and development, we are leveraging validated biology to advance precision therapies designed to address the true underlying drivers of disease and provide real hope for patients. Cogent`s lead therapeutic candidate, PLX9486 (expected to be named CGT9486 in the future), is a precision kinase inhibitor designed to selectively and potently inhibit the KIT D816V mutation. This mutation is responsible for driving a rare and serious condition called Systemic Mastocytosis which can severely impact many different tissues and organs in the body. We are also studying PLX9486 to treat advanced gastrointestinal stromal tumors (GIST), which have a strong dependence on oncogenic KIT signaling.
Dance Biopharm is a private company focused on developing novel inhaled formulations of medicines to treat diabetes and other serious chronic diseases.
Bio-Path is a biotechnology company developing targeted therapies for acute myeloid leukemia (AML), chronic myeloid leukemia (CML) and other challenging cancers. The company`s lead product candidate, prexigebersen (Liposomal Grb2 Antisense), formerly BP1001, is currently being assessed in a Phase 2 clinical study in 54 people with previously untreated AML who are not eligible for or who have decided to forego intensive induction therapy because of their age or fragile health.
Refuge is leveraging gene engineering technologies known as CRISPR interference (CRISPRi) and CRISPR activation (CRISPRa) to develop therapeutic cells that are programmed to make decisions inside the patient`s body.