Understanding and managing acute central nervous system (CNS) effects is essential when developing therapeutic oligonucleotides. Recent research reveals two transient CNS responses following intrathecal administration – rapid neuronal activation and delayed, reversible sedation – each driven by distinct factors such as dose, sequence and chemical composition. Standardized behavioral frameworks now enable consistent evaluation across species, helping researchers interpret neurotoxic responses more effectively.
Join this webinar to explore how these insights can improve assessment of safety and tolerability in oligonucleotide-based therapies targeting the CNS.
Meet Our Speaker
Paymaan Jafar-Nejad
Executive Director, Neurology Research
Paymaan Jafar-Nejad, MD, is a scientific leader in RNA therapeutics and currently serves as Executive Director at Ionis Pharmaceuticals, Inc., a pioneering biotechnology company in RNA-targeted drug discovery and development. He brings more than two decades of experience in neuroscience and RNA biology, including over a decade focused on oligonucleotide therapeutics, and has contributed to a substantial body of scientific publications in the field.
Dr. Jafar-Nejad earned his medical degree from Tehran University of Medical Sciences and completed postdoctoral training in Dr. Huda Zoghbi’s laboratory at Baylor College of Medicine, where he investigated mechanisms of neurodegeneration. At Ionis, he has led research programs in neurology and rare diseases, advancing RNA-based therapies into clinical trials for ALS, Angelman syndrome, genetic epileptic encephalopathies, and related disorders. In recognition of this work, he and his colleagues received the 2023 Lalji & Family ALS Endowed Award for Innovative Healing from the Healey & AMG Center at Massachusetts General Hospital.
