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MBTC-AT-EVs (Genetic)
- Development stage
- Preclinical
- Lead developer
- Stemmedicare
- Modality
- Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
01
Overview
MBTC-AT-EVs (Genetic) is an extracellular vesicle (EV)-based therapeutic candidate being developed by Stemmedicare for the treatment of genetic diseases, specifically Neurofibromatosis and Spinal Muscular Atrophy. The drug is part of Stemmedicare's proprietary EV platform, which utilizes vesicles to deliver therapeutic cargo or mediate biological signaling. While specific molecular targets or the nature of the cargo (e.g., RNA, protein) have not been publicly detailed, the program aims to address the underlying pathology of these genetic conditions. As of early 2024, the asset is in the preclinical stage of development, focusing on efficacy verification for its intended genetic indications.
- Other names
- MBTC-AT extracellular vesicles
02
Targets
LDH (L-lactate dehydrogenase A chain)PPARGC1A (Peroxisome proliferator–activated receptor gamma coactivator 1-alpha)
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