Lempo Therapeutics

Lempo Therapeutics is a biotechnology company pioneering a one-time, curative gene editing therapy to prevent immune-mediated organ damage. Developed in collaboration with Prof. Donald B. Kohn of UCLA, our novel CRISPR-Cas9 platform permanently knocks out the MPO gene in autologous hematopoietic stem cells. This precision approach eliminates the Myeloperoxidase (MPO) protein, a critical driver of oxidative stress and inflammation implicated in severe vascular and autoimmune disorders—including ANCA-associated vasculitis, cardiovascular diseases, and neurodegenerative conditions.

We have completed comprehensive translational work, demonstrating >90% editing efficiency and a clean safety profile with no off-target effects. Following positive FDA feedback in a Pre-IND session and a strategic review, we have prioritized MPO-ANCA Glomerulonephritis (AAGN) as our lead indication. We are now advancing through final IND-enabling studies with a roadmap to file an IND and initiate our First-in-Human clinical trial within 12 months.