Engineering iPS-Derived MSCs with Enhanced Homing and Anti-Inflammatory Properties for the Treatment of DMD
Abstract
Rationale: Duchenne muscular dystrophy (DMD) is a hereditary, X-linked condition caused by a mutation in the dystrophin gene that leads to progressive muscle fiber degeneration and weakness. The diagnosed incidence of DMD is estimated to be 17.24 per 100,000 live male births in the United States, making it the most common and severe type of muscular dystrophy. Currently, no cure exists for DMD, and treatment is primarily supportive. DMD results in inflammation and muscle damage due to the absence of the structural protein dystrophin. This constant inflammation also prevents muscle repair. It is believed that, by controlling the inflammation, the DMD disease process can be delayed, and muscles can be allowed to repair themselves. Factor Bioscience uses its novel, patent-protected technologies to develop a type of cell known as induced-pluripotent-stem-cell-derived mesenchymal stromal cells (iMSCs). iMSCs outperformed bone marrow-derived mesenchymal stromal cells and demonstrated anti-inflammatory and antibacterial properties when tested in animals. This project will use additional aspects of Factor’s core technologies to engineer these cells to better home to damaged muscles and more strongly suppress inflammation, and we will show this in a mouse model of DMD. In DMD patients, these cells will allow muscle repair and improved motor function. Applicability: The research proposed in this application will help patients with DMD directly and can also be used to enhance other cutting-edge DMD therapies. In addition to being a more effective alternative to current DMD management, Factor Bioscience’s proposal presents a more efficacious, robust, well-characterized, and cost-effective cell therapy option. This project will also create a platform to create even more potent engineered induced pluripotent stem cell (iPSC)-derived-MSC (EiMSCs) with additional mechanisms of action for DMD and other muscle pathologies. Following this research step, Factor Bioscience plans to move to test EiMSCs in additional animal models (in vivo work) to assess safety and effectiveness before moving to first-in-human clinical work. With successful outcomes, Factor Bioscience are aiming for EiMSCs to be a viable DMD treatment option in clinical trials by 2026.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Jan 04, 2024
- Source ID
- HT94252310787
Entities
People
- Christopher Rohde
Organizations
- United States Army