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

[HT-17-1] Clinical application siRNA for neurological diseases

^○Muthiah Manoharan (Alnylam Pharmaceuticals, Inc.)

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.