Drug intelligence / Profile preview

MSC-AAT

Development stage
Unknown
Lead developer
Kyttaro
Modality
Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies, Stem Cell Therapies → Cell Therapies
Administration
Intravenous, Parenteral
01

Overview

MSC-AAT is a genetically engineered cell therapy platform consisting of mesenchymal stem cells (MSCs) modified to overexpress alpha-1 antitrypsin (AAT). Developed by Kyttaro Therapeutics (and associated with Exollent Therapeutics), this candidate leverages the innate immunomodulatory and regenerative properties of MSCs alongside the potent anti-inflammatory and cytoprotective effects of AAT. AAT is a serine protease inhibitor that protects tissues from proteolytic damage and modulates immune responses by promoting the expansion and function of regulatory T cells (Tregs) and regulatory B cells, while suppressing pro-inflammatory Th1 and Th17 responses. The therapy is primarily being investigated for the treatment of Graft-versus-Host Disease (GvHD), Type 1 Diabetes, and various inflammatory lung and joint diseases, aiming to induce immune tolerance and protect target tissues from autoimmune destruction.

Other names
AAT-MSCsalpha-1 antitrypsin-expressing mesenchymal stem cellsalpha1 antitrypsin-expressing mesenchymal stem cellsalpha 1 antitrypsin-expressing mesenchymal stem cellsAAT-overexpressing MSCs

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