AI and ML: The Future of Target and Drug Discovery
The convergence of artificial intelligence and machine learning in biomedical research is bringing new hope to the ALS community, offering the potential to identify novel therapeutic targets and accelerate drug development in a disease where time is everything.
Artificial intelligence and machine learning now enable the rapid virtual design and testing of thousands of new molecules and the simulation of their interactions with therapeutic targets.
Leading pharmaceutical companies are investing heavily in these tools. Roche has built the largest announced GPU footprint of any pharmaceutical company, embedding accelerated computing into the core of how it discovers and develops new therapies. Eli Lilly partnered with NVIDIA to build a powerful AI supercomputer for medicine discovery and launched its TuneLab platform, backed by over $1 billion in proprietary research data, to give biotech companies access to its AI-enabled drug discovery models.
These topics and more were covered at this year’s Annual Meeting.
A Letter from Amy Easton, PhD, VP of Scientific Programs
The promise of AI in ALS research is real. So is the work left to do. Amy Easton, PhD, breaks down where the field stands.
Artificial intelligence is reshaping how we find drug targets, but what does that mean for ALS specifically?
Current & Future Impact of AI/ML on Novel Target Discovery in ALS
At the 2026 Target ALS Annual Meeting, moderator Puneet Batra led a forward-looking roundtable with leaders from biopharma, AI-driven biotech, and venture capital to explore how AI and machine learning are already influencing target discovery in neuroscience — and where the field is headed next.
More from the field
AI and Machine Learning: A Turning Point for ALS Research
Aj Kaykas, Chief Exploration Officer and Head of Neuroscience at insitro, makes the case that Target ALS’s high-quality, consistently formatted data sets represent a unique opportunity for machine learning (ML) to transform ALS research. Having previously worked at the Allen Institute and Novartis on data-intensive biology projects, Kaykas explains how researchers can now “slice across” multi-omics data and patient trajectories to identify meaningful patterns.
Using AI as a Hypothesis Engine to Accelerate ALS Discovery
Annalisa Pawlosky, Senior Staff Research Scientist at Google, introduces an innovative application of artificial intelligence to ALS research. Her team’s “agentic model” system, Co-Scientist, functions as a novel hypothesis generator by synthesizing scientific literature to surface unique mechanisms and connections, backed by web search and evidence-based reasoning. Hear how AI is being positioned not to replace scientists, but to help them ask better questions and find connections across the rapidly growing body of ALS research.
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