Published: Jan 10, 2021 By Gail Dutton
n-Lorem Foundation has taken on a challenge that many nations consider too great: treating patients with ultra-rare diseases (which affect 30 or fewer people) for free, for life. It received FDA guidance January 5
th and is in the midst of preparing the first package of investigational new drug (IND) applications – usually with an n-of-1, for individual patients. Patient dosing is expected to begin later this year.
“No one has ever done this before,” Stanley Crooke, M.D., Ph.D., founder, CEO, and chairman of the board of n-Lorem Foundation, told BioSpace. “I expected we’d have 5 applications our first year. We had 48, and are moving to treat 23 people.” Experiments are planned for patients at Harvard and 10 at Columbia University for an aggressive mutation of ALS. While forming n-Lorem, “We helped two investigators obtain antisense oligonucleotides for several patients,” he added.