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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.
We are a biotechnology company creating a new class of targeted gene therapies for debilitating and life-threatening genetic disorders in both central nervous system (CNS) and non-CNS indications. Current gene therapy approaches have shown dramatic efficacy in several rare diseases but are hindered by imprecise targeting. The inability to selectively transduce specific cells and tissues effectively drives administration of higher doses and resulting safety liabilities. We are addressing these concerns with our proprietary adeno-associated virus (AAV) engineering platform that generates capsids optimized to target specific tissues and cells in the disease organ while limiting transduction of tissues and cells not relevant to the target disease. Through our next-generation AAV platform combined with cargo development and state-of-the-art manufacturing, we are tackling monogenetic and sporadic CNS and non-CNS disorders that have previously been challenging to address.
Xcell Bio is a San Francisco-based life science company dedicated to bringing an innovative primary cell control solution to the immunotherapy market to support research, drug discovery, and cell manufacturing. Xcell`s mission is to democratize maintenance, expansion and control of any primary cell type through its bench-top primary cell control systems, reagent kits, software, and standardized protocols. The technology delivers simple control of primary cell types from blood or solid tissue, including a range of immune cells, stem cells, and tumor cells. This patient-centric technology offers a unique and proprietary capability to improve immunotherapy efficacy, safety, and persistence through more predictive and iterative therapy development and rapid, highly-controlled clinical cell manufacturing.
REGENXBIO is a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno‐associated virus (AAV) gene therapy. REGENXBIO`s NAV® Technology Platform, a proprietary AAV gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO`s mission is to transform the lives of patients suffering from severe diseases with significant unmet medical need by developing and commercializing in vivo gene therapy products based on REGENXBIO`s NAV Technology Platform. REGENXBIO seeks to accomplish this mission through a combination of internal development efforts and third‐party NAV Technology Platform licensees.
Gyroscope Therapeutics is a clinical-stage gene therapy company developing gene therapy beyond rare disease to treat diseases of the eye that cause vision loss and blindness. The company was acquired by Novartis, a leading global medicines company, in February 2022.