Aevitas Therapeutics

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Aevitas Therapeutics focuses on advancing gene therapies of short-form human factor H (sFH) to restore complement regulation in dry age-related macular degeneration (Dry AMD), atypical hemolytic uremic syndrome (aHUS), and other conditions characterized by complement dysregulation. Their sFH platform involves an engineered, functional short-form factor H with its transgene cloned into adeno-associated viruses (AAV) for delivery by gene therapy. Avista's goal is to develop innovative gene therapies for retinal diseases, including rare, inherited conditions with a significant impact on patients' quality of life. They utilize a computationally guided scAAVengr platform to create and validate a toolkit of proprietary AAV vectors that address key challenges in effective gene therapies, such as cell-type specific delivery, reduced dosages, and efficient expression, through a minimally invasive intravitreal approach. Vita is a cell engineering company specializing in induced pluripotent stem cell technology to engineer specific cell types intended to replace those that are defective in patients. They are advancing their lead program, VTA-100, for the treatment of limb-girdle muscular dystrophy (LGMD2A), and developing proprietary cellular therapies through a dual development strategy, starting with autologous-derived cells before transitioning to a universal hypoimmunogenic cell line.