Hideki Mochizuki,
Professor and Chairman,
Osaka University Graduate School of Medicine
Dr. Mochizuki is currently Professor and Chair of the Department of Neurology at Osaka University in Osaka, Japan. Prior to his present position, he served as Professor and Chair of Neurology at Kitasato University in Kanagawa, Japan. He obtained his MD and PhD degrees from Juntendo University in Tokyo, and his neurology residency at Tokyo Metropolitan Neurological Hospital. He then completed a postdoctoral fellowship in the Developmental and Metabolic Neurology Branch of NINDS / NIH in Bethesda, Maryland under the tutelage of Roscoe Brady. He returned to Japan to serve on the faculty of Neurology at Juntendo University for eleven years, rose through the academic ranks until his appointment as Chairman first at Kitasato University and now at Osaka University.
The Recent State of Oligonucleotide Therapeutics for Neurological Disorders
Friday, 7 October 2022 at 09:00
Add to Calendar ▼2022-10-07 09:00:002022-10-07 10:00:00Europe/LondonThe Recent State of Oligonucleotide Therapeutics for Neurological Disorders3D-Bioprinting, Biofabrication, Organoids and Organs-on-Chips Asia 2022 in Tokyo, JapanTokyo, JapanSELECTBIOenquiries@selectbiosciences.com
Oligonucleotide therapeutics have a wide range of potential applications in the clinic owing to their highly different modes of action such as antisense, ligands and protein inhibitors. In the field of neurology, ASOs that modulate splicing of SMN2 and Dystrophin pre-mRNA were approved for spinal muscular atrophy and Duchenne muscular dystrophy, respectively. siRNA encapsulated in lipid nanoparticle that degrades TTR mRNA was also available for hereditary ATTR amyloidosis. Many others are being developed worldwide, targeting neurological disorders caused by gene mutations and neurodegenerative diseases such as Huntington’s disease, amyotrophic lateral sclerosis (ALS), Parkinson’s disease (PD), multiple system atrophy (MSA) and Alzheimer’s disease. Beyond pre-clinical trials, several clinical trials of oligonucleotide therapeutics are ongoing for neurological diseases, whereas phase III trials of tominersen (for Huntington’s disease) and tofersen (for ALS) closed due to failing to attain their primary efficacy endpoints in 2021. We have been developing ASO suppressing SNCA expression as a potential therapy for MSA and PD. In this symposium, we will review the recent state of oligonucleotide therapeutics for neurological diseases including basic science, approved drugs and clinical trials.
Add to Calendar ▼2022-10-06 00:00:002022-10-07 00:00:00Europe/London3D-Bioprinting, Biofabrication, Organoids and Organs-on-Chips Asia 20223D-Bioprinting, Biofabrication, Organoids and Organs-on-Chips Asia 2022 in Tokyo, JapanTokyo, JapanSELECTBIOenquiries@selectbiosciences.com