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SAN DIEGO, Jan. 26, 2021 /PRNewswire/
n-Lorem Foundation, the first and only nonprofit organization whose mission is to create experimental antisense oligonucleotide (ASO) treatments for patients who have diseases caused by extremely rare mutations (1-30 patients worldwide) for free, for life, celebrates its first anniversary and the extraordinary progress made in 2020 toward its mission of offering immediate hope and rapid treatment to the millions of patients affected by ultra-rare mutations.
In its first 12 months of operation, n-Lorem Foundation received applications to treat 50 patients, is proceeding to treat nearly 20 of those patients and helped two clinical investigators obtain experimental ASO treatments for nine patients – greatly exceeding applicant and acceptance rate expectations. Leveraging the powerful ASO technology developed at Ionis Pharmaceuticals, the foundation has established quality processes so that only appropriate patients are tr
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.
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CARLSBAD, Calif., Dec. 17, 2020 /PRNewswire/ Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced today that its founder and executive chairman Stanley T. Crooke, M.D., Ph.D., will retire from Ionis and its board of directors effective June 2021. Dr. Crooke is stepping down so that he may focus on his scientific interests and the nonprofit organization he founded, the n-Lorem Foundation. Dr. Crooke will continue to serve as a scientific advisor to Ionis including providing advice regarding Ionis research and development programs and guiding the core antisense research group he founded at Ionis.
/PRNewswire/ n-Lorem Foundation, a nonprofit organization that provides free, lifetime supplies of individualized RNA-targeted medicines to patients living.