Webinar Overview
Amyotrophic Lateral Sclerosis (ALS) is a devastating neurodegenerative disease for which there are no curative therapies. Over the last decade, significant advances have been made in the understanding of ALS disease pathology and linked genes, leading to a boom in drug discovery across various therapeutic modalities.
Antisense oligonucleotides (ASOs), short oligonucleotides that modulate gene expression at the transcriptional level, have already proven to be safe, tolerable, and effective in ALS, following the FDA-approval of Tofersen in 2023. However, this drug only addresses a small patient population, leaving a high unmet clinical need that could be me through the discovery of new targets and development of novel ASOs.
In this webinar, you will hear from experts in ASO drug development for ALS to:
- Learn how Amylyx Pharmaceuticals takes a targeted approach to ASO development
- Discover more about the process of ASO design, in vitro screening, and efficacy studies
- Explore the next steps into in vivo proof-of-concept
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Webinar Presenters
Evan Mizerak
Lead, Preclinical Research
Amylyx Pharmaceuticals
Susanne Back, PhD
Senior Manager, CNS Pharmacology
Charles River
Scientific Moderator
Blanca Torroba, PhD
Senior Scientist II
Charles River