For people with ultra-rare genetic diseases, a diagnosis can come with a devastating realization that there may be no treatment in development because the patient population is too small to attract traditional biopharma investment. Nome is working to change that by combining patient-led drug development with AI-enabled scientific and operational support. Steven Ringel, founder and CEO of Nome and founder of the Kazuna Foundation, discusses how the company is helping families and patient organizations identify viable genetic-medicine options, build development plans, and find qualified partners who can help advance individualized therapies toward the clinic.

GLP-1 therapies have dramatically reshaped obesity care, but the next generation of medicines may be judged by more than how much weight patients lose. A central question is whether treatments can selectively reduce the visceral fat associated with cardiometabolic disease while maintaining the muscle needed for physical function, metabolic health, and durable weight management. Erik Ingelsson, chief scientific officer of Wave Life Sciences, discusses the biology behind a new RNA-based approach designed to remove a natural brake on fat breakdown, how the company is thinking about clinical endpoints and patient populations, and why precision approaches to fat loss could change the definition of success in obesity treatment.

T-cell engagers have delivered important advances in blood cancers, but extending their success to solid tumors has proved considerably more difficult. Physical barriers, immunosuppressive tumor microenvironments, and the risk of attacking healthy tissue have constrained the field. Deck Bio is developing multi-target T-cell engagers to address these limitations. Jack Silberstein, the company’s CEO, discusses Deck Bio’s efforts to create therapies that reach more cancer cells, reduce the likelihood that tumors will evade treatment, and minimize harm to healthy tissue.