The Data Safety Monitoring Board (DSMB) is composed of distinguished, independent experts in antisense oligonucleotide (ASO) technology, clinical trial design and evaluation, and drug development. Many are also dedicated clinicians who care for patients with rare diseases. This board provides unbiased safety oversight by reviewing accumulated data from all investigator-initiated studies involving n-Lorem’s ASO medicines.

n-Lorem's DSMB is chaired by Dr. Eugene Schneider, Executive Vice President and Chief Clinical Development and Operations Officer at Ionis Pharmaceuticals.

On This Episode We Discuss:

• 2:20 – What is the Data Safety Monitoring Board at n-Lorem?
• 6:50 – Monitoring the data from each and every treated n-Lorem patient is essential
• 9:40 – The DSMB consists of a collection of physicians and other experts
• 13:20 – As the number of n-Lorem patients grows, the DSMB’s responsibilities have expanded significantly—yet members remain deeply committed to supporting the mission
• 17:05 – The DSMB’s goal is to avoid any ASO-related serious adverse events (SAEs) and minimize adverse events
• 19:20 – During his time as Chair of the DSMB, Eugene has learned lessons in patience and humility
• 21:00 The judgements of monitoring boards can and will affect lives

Links: n-Lorem 2025 Nano-rare Patient Colloquium: https://www.nlorem.org/nano-rare-patient-colloquium-2025/

Donate to support n-Lorem programs: https://www.nlorem.org/donate/

Hongene Biotech: https://www.hongene.com/