NEURO61

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

Wed. Sep 2, 2020 10:45 AM - 12:15 PM Room 1 (OKAYAMA CONVENTION CENTER 4F 405)

Chair：TakanoriYokota（Department of Neurology, Tokyo Medical and Dental University）,TatsushiToda（Department of Neurology, Graduate School of Medicine, The University of Tokyo）

[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.

[HT-17-2] BBB-penetrating heteroduplex oligonucleotide

○Tetsuya Nagata (Department of Neurology and Neurological Science, Tokyo Medical and Dental University, Japan)

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.

[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.

[HT-17-4] Basic biology and clinical application of ASO to SMA

○Kentaro Sahashi (Department of Neurology, Nagoya University, Japan)

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.