[HT-17] Hot Topics 17
Update of Oligonucleotide Therapy for CNS diseases；From Basic to Clinic

[HT-17-3] Antisense oligonucleotides for the treatment of neurodegenerative diseases

^○Holly Kordasiewicz, Dan Norris, Anne Smith, Roger Lane, Frank Bennett, Eric Swayze (Ionis Pharmaceuticals, USA)

Following antisense oligonucleotide, nusinersen/Spinraza for SMA, first siRNA drug, givosiran/Fitusiran for TTR amyloidosis (familial amyloid neuropathy) has been approved in Japan, and SOD1 and C9ORF72 for ALS, tau for Alzheimer disease, PSP/CBD, synuclein for Parkinson disease, DLB and MSA, and Huntinting for Huntington disease are also started for clinical stages. This rapid and splendid progress of oligonucleotide therapy are now a main stream for development of DMT to neurological diseases. In this symposium, basic chemistry, delivery and biology of oligonucleotide drug are reviewed, and current clinical status for approved oligonucleotide drugs for neurological diseases are to be updated.

Dr. Holly Kordasiewicz is Vice President of Neurology Research at Ionis Pharmaceuticals, a company that specializes in RNA therapeutics. Dr. Kordasiewicz heads a team focused on identifying antisense oligonucleotide therapeutics for currently untreatable neurodegenerative diseases, including drugs now in clinical trials for ALS, Alzheimer’s disease, Parkinson’s disease and Huntington’s disease. Dr. Kordasiewicz joined Ionis 8 years ago after working on the preclinical validation for Ionis’ Huntington’s disease program as a post-doctoral fellow in the laboratory of Dr. Don Cleveland at the University of California at San Diego. Dr. Kordasiewicz began her work on understanding neurodegenerative diseases at the University of Minnesota, where she received her Ph.D. in Neuroscience.