Value Proposition
· Disease-Modifying Mechanism: Elevates fibrinogen-like protein 1 (FGL1) in the brain to inhibit proteinopathies (spread of toxic proteins in the brain).
· Versatile Delivery Options: Flexible treatment strategies including gene therapy, exosome delivery, and blood exchange to deliver FGL1.
· Dual application: Functions as both a therapeutic agent and a blood biomarker for aging-associated neurodegeneration risk.
· Broad Disease Coverage: Addresses multiple neurodegenerative diseases such as Parkinson’s disease (PD), Lewy body dementia (LBD), Alzheimer’s disease (AD), and other neurological disorders involving protein misfolding.
Unmet Need
· Neurodegenerative diseases such as PD, LBD, and AD progress through the prion-like spread of misfolded proteins, which is accelerated by aging and currently lacks effective, disease‑modifying interventions.
· Current care focuses on symptomatic management (motor and cognitive symptom control) and lacks validated blood biomarkers for risk prediction and treatment monitoring.
· Therefore, there is a strong need for FGL1-based therapeutics and blood biomarkers to be developed to block upstream prion-like propagation and address early risk prediction and treatment monitoring.
Technology Description
· Johns Hopkins researchers have discovered that FGL1, a protein that naturally declines with age, plays a crucial protective role against neurodegenerative diseases by preventing the spread of toxic proteins in the brain.
· Elevating FGL1, through various methods including AAV-mediated gene delivery, exosome-mediated protein delivery, or blood exchange approaches, prevents the prion‑like spread of misfolded proteins (α‑synuclein) between brain cells.
· Preclinical models of PD and LBD demonstrate elevation significantly reduces pathological protein spread and preserves dopamine neurons.
Stage of Development
· Preclinical proof-of-concept demonstrated in vitro and in vivo; mechanism-of-action studies planned.
Data Availability
· Data available upon request.
Publications: N/A
