Clinical trials
Follow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
cell engineering (engineered MSC)
- Development stage
- Preclinical
- Lead developer
- ToolGen
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Stem Cell Therapies → Cell Therapies
- Administration
- Intramuscular, Intravenous
01
Overview
cell engineering (engineered MSC) is an ex vivo gene-edited mesenchymal stem cell (MSC) therapy being developed by ToolGen in collaboration with Cellartgen. The therapy utilizes CRISPR-Cas9 technology to knock out Kelch-like ECH-associated protein 1 (KEAP1), a negative regulator of the antioxidant transcription factor NRF2, in human bone marrow-derived MSCs. This genetic modification activates the NRF2 pathway, reducing intracellular oxidative stress and reprogramming redox and paracrine gene networks. As a result, the engineered MSCs exhibit enhanced cell survival, sustained secretion of proangiogenic cytokines, and improved tissue perfusion and engraftment in ischemic environments. The program is currently in preclinical development (lead optimization) for the treatment of chronic limb-threatening ischemia (CLTI) and other conditions where oxidative stress limits the efficacy of regenerative cell therapies.
- Other names
- engineered MSCengineered MSC (ToolGen)
02
Targets
Cas9 (CRISPR-associated protein 9)KEAP1 (Kelch-like ECH-associated protein 1)NFE2L2 (Nuclear factor (erythroid-derived 2)-like 2)
Beyond the preview
Go deeper on cell engineering (engineered MSC).
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Explore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Trace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Explore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Compare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Connect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on cell engineering (engineered MSC).