Safety Updates
As leaders in ASO technology, we will provide updates on safety to the nano-rare community. These updates may pertain to an n-Lorem program, or they may not be associated with an n-Lorem program at all. We believe that by sharing information in a timely manner together with our guidance on ASO discovery, development, manufacturing, dose and dose schedule, we help the community by holding ASO developers to the same high standard we demand of ourselves.
Expanding our Expertise
Leaders in the discovery and development of ASO technology
Though the discovery and development of ASOs may sound simple, it is not. It requires significant expertise, knowledge, experience and automation that n-Lorem has. ASOs are complex molecules that require rigid selection criteria and careful consideration of dosing parameters.
At n-Lorem, we are open to collaborations and encourage all academic investigators to work with us to take advantage of our knowledge and experience. Furthermore, the clinical administration of experimental ASOs must be done professionally by investigators knowledgeable about ASOs who have significant experience in the develop of experimental medicines.
Latest Safety Updates
October 14, 2025
In accordance with our commitment to safety and the professional management of all of our patients treated by an n-Lorem ASO, on October 14, 2025, we issued a safety bulletin describing the possible ASO-related adverse event that we believe is an isolated observation unique to this patient.
The possibly related adverse event was observed in a patient with a toxic gain-of-function mutation in KIF1A who is treated with an allele-selective PS ASO designed for that patient. This patient has been treated for almost three years, demonstrated significant benefit with regards to a broad range of neurodegenerative manifestations of KIF1A-associated disease (KAND) and continues to demonstrate benefit. The adverse event is mild, transient fevers associated with intrathecal dosing of the ASO. After completing a thorough analysis to ascertain the potential cause of the fever, several discussions with experts, the treating physician and the data safety monitoring board, and after the physician discussed the situation with the family, we submitted a full report to the FDA. Because the patient continues to demonstrate meaningful benefit, we proposed continued treatment with added focused monitoring. The FDA agreed with our plan.
Based on the evidence available today, we believe that the fever represents an ASO-KAND interaction that appears to be unique to the severe syndrome presented by this patient.
December 2022
In December 2022, a study conducted by Roche was unblinded and reported, which triggered the reissue of the below safety update. The new data was from a controlled clinical study conducted by Roche in approximately 800 patients with Huntington’s disease treated with an antisense oligonucleotide (ASO) of the same chemical class as n-Lorem compounds. The ASO in question was not developed by n-Lorem, but given the seriousness of this event and in an effort to provide as much guidance to our investigators as possible, we provided a safety update to our investigators making them aware of these data.
August 1, 2022
On August 1, 2022, we issued a safety update to a broad group of physicians and scientists.
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