Drug intelligence / Profile preview

MSC-NG01

Development stage
Phase 2
Lead developer
NeuroGenesis
Modality
Patient-derived iPSCs → iPSCs → Pluripotent Stem Cells → Stem Cell Therapies → Cell Therapies, Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Autologous CAR-T → CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies, Mesenchymal Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Tumor-Infiltrating Lymphocytes (TILs) → Native Immune Cells → Adoptive Cell Transfer → Cell Therapies
Administration
Intrathecal, Intravenous
01

Overview

MSC-NG01 (also referred to as NG-01) is an autologous cell therapy consisting of a proprietary subpopulation of bone marrow-derived mesenchymal stem cells (MSCs) with high immunosuppressive, neuroprotective, and regenerative potential. The therapy is developed for the treatment of progressive forms of multiple sclerosis (MS), particularly secondary progressive MS. The process involves harvesting bone marrow from the patient, expanding the selected MSCs ex vivo, and then administering them back to the patient—primarily via intrathecal injection into the cerebrospinal fluid. This targeted delivery allows the cells to migrate to sites of injury in the central nervous system where they exert anti-inflammatory effects, secrete neurotrophic and remyelinating factors, and promote tissue repair. Clinical trials have demonstrated that repeated intrathecal administration leads to significant reductions in biomarkers associated with neurodegeneration (notably neurofilament light chain [NfL] and glial fibrillary acidic protein [GFAP]), stabilization or improvement in neurological function—including mobility and cognition—and a favorable safety profile with only mild adverse events such as headache or back pain[2][3][4][5][6][7][8][9].

Brand names
NG CELL
Other names
Autologous bone marrow derived human stromal cellsNG01-MSCsNG-01-MSCsNG 01-MSCs

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