Stanley Crooke, MD, PhD, talks about his non-profit, which brings the benefits of modern biotechnology to patients with serious, ultra-rare disorders. His pioneering research has led to first-time treatments for patients that at one time had no hope. Dr. Crooke founded the non-profit N-Lorem organization to transform the landscape for rare disease clinical research and address the substantial challenges faced when developing cures for rare genetic diseases. He is also the founder, former CEO, and board member of Ionis Pharmaceuticals, as well as the founder, current CEO, and chairman of the board of non-profit, N-Lorem Foundation. N-Lorem helps identify patients who can benefit from antisense oligonucleotide technology, matches them to a suitable therapy, and finds the appropriate principal investigator to plan and run an N-of-1 trial. N-Lorem also facilitates investigator and FDA collaboration to establish an approved investigator-initiated IND, and it funds treatment. This interview addresses advancements in genome sequencing, newborn screening, regulatory approaches, science, and collaboration with patients —all of which contribute to a more promising drug development landscape.