Inspiration:
- Limited effective treatments for ALS.
- Neuroinflammation contributes to disease progression.
- Need for targeted immune regulation within the CNS.
What It Does:
- Stabilizes Treg identity through TSDR demethylation.
- Enhances CNS migration through CXCR3-A overexpression.
- Maintains Treg function with low-dose IL-2.
- Targets neuroinflammation in ALS. How We Built It
- Patient leukapheresis and Treg isolation.
- Ex-vivo genetic and epigenetic engineering.
- cGMP-compliant cell expansion and quality control.
- Intrathecal administration and IL-2 support.
Challenges We Ran Into:
- Maintaining stable FOXP3 expression.
- Ensuring effective CNS trafficking.
- Balancing Treg functionality and safety.
- Addressing manufacturing complexity and cost. Accomplishments We're Proud Of
- Integrated three complementary engineering strategies.
- Combined targeted delivery with immune regulation.
- Incorporated an iCasp9 safety switch.
- Established a biomarker-based monitoring strategy.
What We Learned:
- FOXP3 expression alone does not guarantee full Treg functionality.
- Epigenetic stability is essential for Treg identity.
- Chemokine signaling influences immune-cell migration.
- Cell therapy requires careful safety and feasibility planning.
What's Next for NEURO-Treg:
- Validate therapeutic efficacy in preclinical ALS models.
- Optimize cell manufacturing and reduce production costs.
- Evaluate long-term safety and Treg persistence.
- Progress toward early-phase clinical trials.
- Monitor outcomes using CSF-pNFH and ALSFRS-R.
Built With
- therapeutics
