Huntington's Disease Program
Precision neurotherapeutic strategies and molecular disease modeling for a monogenic disease with no current cure.
The problem we are solving.
Huntington's disease is one of medicine's cruelest inheritances — a single CAG-repeat expansion in the HTT gene that guarantees the disease's arrival, often decades before symptoms appear. Current therapies manage symptoms; none alter the underlying course.
The genetic certainty of Huntington's is also its therapeutic opportunity. The mutation is known. The protein is known. The pathogenic mechanism — toxic gain of function from the expanded mutant huntingtin protein — is increasingly understood. What remains is the engineering: a therapy that lowers mutant HTT in the brain, safely, durably, and at the right point in the disease course.
How we tackle it.
The Huntington's program integrates two of MACRO HRD's core capabilities: brain-mapping platform technologies for biomarker discovery and disease-progression staging, and BBB-facilitating delivery systems developed for the GBM Immunotherapy program. The strategic intent is precision neurotherapeutic design — matching intervention timing and modality to where each patient sits in the molecular disease course.
GEDM-3DQ provides the longitudinal modeling layer: tracking individual disease trajectories against multimodal biomarker signals, identifying intervention windows, and supporting the kind of adaptive trial design that monogenic disorders with long prodromal phases demand.
What makes this real.
Part of an integrated platform.
“When a disease is genetically certain, intervention can begin before symptoms — if we know when, and how.”