New Publication Provides an Unprecedented Look at the Largest Reported Cohort of Nano-Rare Patients Evaluated for Individualized ASO Therapies
First-of-its-kind perspective from the n-Lorem Foundation provides a very broad review of n-Lorem’s experience in more than 300 patient applications and reports excellent safety profile and meaningful clinical benefit in treated patients receiving individualized ASO therapies
n-Lorem, a nonprofit foundation that discovers, develops and provides personalized experimental antisense oligonucleotide (ASO) medicines to patients with nano-rare mutations (1-30 known worldwide) for free, for life, announced today the publication in Nucleic Acids Research [https://doi.org/10.1093/nar/gkag504] that details its experience with the first 329 nano-rare patient applications for treatment and a summary of the results to date in nano-rare patients treated with experimental ASOs medicines provided by n-Lorem.
n-Lorem has created the largest safety database of nano-rare patients treated that includes more than 50 nano-rare patients treated with bespoke ASO medicines discovered and developed at n-Lorem, more than 300 doses administered across all routes of administration (subcutaneous, intravitreal, intrathecal), and more than 55 patient years of safety data. Amongst treated patients, one patient has been treated for longer than three years, six patients for longer than two years and 26 patients for more than one year. In all treated patients, n-Lorem has observed an excellent safety and tolerability profile with no ASO-related serious adverse events.
