The IGI simultaneously pursues two tracks: It targets monogenic diseases where cures are achievable now for immediate impact. In parallel, it invests in the foundational science needed to tackle highly complex diseases like Alzheimer's and solid tumors, building a portfolio for the long term.

Instead of diversifying across diseases, Kenai is building deep expertise in Parkinson's. Its pipeline addresses different patient needs: replacing lost cells (lead program), repairing existing damaged cells (002), and targeting inherited forms (003), creating a comprehensive disease franchise.

Tackling monumental challenges, like creating a biologic effective against 800+ HIV variants, is not a single-shot success. It requires multiple iterations on an advanced engineering platform. Each cycle of design, measurement, and learning progressively refines the molecule, making previously impossible therapeutic goals achievable.

Instead of targeting individual gene mutations in diseases like ALS, condensate science focuses on shared cellular structures where genetic risks converge. This approach creates a broader therapeutic target, potentially treating more patients with diverse genetic profiles.

Brad Ringeisen translates his experience at DARPA to the Innovative Genomics Institute by scoping near-impossible challenges with aggressive timelines and fostering a belief that the goal is achievable. This injects a sense of mission-driven urgency typically absent in academic research, now powered by philanthropy.

Gene editing pioneer David Liu is developing a platform that could treat multiple, unrelated genetic diseases with a single therapeutic. By editing tRNAs to overcome common nonsense mutations, one therapy could address a wide range of conditions, dramatically increasing scalability and reducing costs.

CZI set an audacious goal to cure all disease. When scientists deemed it impossible, CZI's follow-up question, "Why not?" revealed the true bottleneck wasn't funding individual projects, but a systemic lack of shared tools, which then became their core focus.

The Innovative Genomics Institute is tackling rare diseases by creating a standardized platform. By keeping elements like the delivery vehicle and enzyme constant and only changing the guide RNA, they aim to create a repeatable 'bucket trial' process for developing hundreds of cures, not just one-offs.

Unlike using genetically identical mice, Gordian tests therapies in large, genetically varied animals. This variation mimics human patient diversity, helping identify drugs that are effective across different biological profiles and addressing patient heterogeneity, a primary cause of clinical trial failure.