ASCEND-ALS: Advanced System for Clinical Evaluation and Novel Drug Design in ALS

Overview

Over 220,000 people live with ALS globally today, a number projected to rise 69% by 2040. Yet diagnosis still takes an average of 12 months after symptoms appear, no effective treatment is widely available, and care at every milestone happens reactively without warning. In a disease where median survival is just 2–5 years, time is the only currency that matters. ASCEND changes that by making both care and treatment personalized. On the clinical side, longitudinal ALSFRS-R trajectories are modeled to predict each patient's individual time to disease milestones months before the crisis, calibrated to their specific rate of functional decline. On the therapeutic side, ASCEND reads a patient's mutation profile and designs a personalized drug from scratch, antisense oligonucleotide sequences tiled across their cryptic exon coordinates. One platform for one patient. Because a world free of ALS begins with treating every patient as the individual they are.

Awards (3)

• Special Award
• Bronze Medal
• Selected for CWSF 2026

Competition history

• CWSF 2026 Disease & Illness

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