Next Generation Scalable Technology for Rare Disease Research

Explore cutting-edge scalable technologies for rare disease research in this symposium session from the Broad Institute's Ladders to Cures Accelerator program. Discover how functional genomics and machine learning are being applied to rare cancer research through Gabe Griffin's presentation on innovative approaches to understanding and treating uncommon malignancies. Learn about rapid drug identification methods for rare disorders through JT Neal's discussion of pooled image-based profiling techniques that accelerate therapeutic discovery. Examine multiplexed optical pooled screening approaches for proteinopathy therapeutic development as presented by Matt Brown, focusing on scalable methods to identify treatments for protein misfolding diseases. Gain insights into how these next-generation technologies are being scaled to accelerate progress toward treatments and cures for patients with rare genetic diseases, representing collaborative efforts between scientists, physicians, patients, and industry partners in the rare disease research community.