While AI excels where large, clean datasets exist (like protein folding), it struggles with modeling slow, progressive diseases like Alzheimer's or obesity. These are organ-level phenomena, and the necessary data doesn't exist yet. In vivo platforms are critical for generating this required foundational data.
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The next leap in biotech moves beyond applying AI to existing data. CZI pioneers a model where 'frontier biology' and 'frontier AI' are developed in tandem. Experiments are now designed specifically to generate novel data that will ground and improve future AI models, creating a virtuous feedback loop.
AI models trained on descriptive data (e.g., RNA-seq) can classify cell states but fail to predict how to transition a diseased cell to a healthy one. True progress requires generating massive "causal" datasets that show the effects of specific genetic perturbations.
Despite AI's power, 90% of drugs fail in clinical trials. John Jumper argues the bottleneck isn't finding molecules that target proteins, but our fundamental lack of understanding of disease causality, like with Alzheimer's, which is a biology problem, not a technology one.
The progress of AI in predicting cancer treatment is stalled not by algorithms, but by the data used to train them. Relying solely on static genetic data is insufficient. The critical missing piece is functional, contextual data showing how patient cells actually respond to drugs.
Current AI for protein engineering relies on small public datasets like the PDB (~10,000 structures), causing models to "hallucinate" or default to known examples. This data bottleneck, orders of magnitude smaller than data used for LLMs, hinders the development of novel therapeutics.
The next frontier in preclinical research involves feeding multi-omics and spatial data from complex 3D cell models into AI algorithms. This synergy will enable a crucial shift from merely observing biological phenomena to accurately predicting therapeutic outcomes and patient responses.
Unlike math or code with cheap, fast rewards, clinically valuable biology problems lack easily verifiable ground truths. This makes it difficult to create the rapid reinforcement learning loops that drive explosive AI progress in other fields.
The bottleneck for AI in drug development isn't the sophistication of the models but the absence of large-scale, high-quality biological data sets. Without comprehensive data on how drugs interact within complex human systems, even the best AI models cannot make accurate predictions.
Traditional methods like crystallography are slow and analyze purified proteins outside their native environment. A-muto's platform uses proteomics and AI to analyze thousands of protein conformations in living disease models, capturing a more accurate picture of disease biology and identifying novel targets.
The founder of AI and robotics firm Medra argues that scientific progress is not limited by a lack of ideas or AI-generated hypotheses. Instead, the critical constraint is the physical capacity to test these ideas and generate high-quality data to train better AI models.
