Posted in | Genomics

Boosting Amyotrophic Lateral Sclerosis (ALS) research

Amyotrophic Lateral Sclerosis (ALS) is a destructive neurodegenerative disease for which there is currently no cure. Over the last ten years, researchers have made significant advances in our current understanding of ALS disease pathology and linked genes. As such, there has been a significant surge in the success of drug discovery across numerous therapeutic modalities.

Antisense oligonucleotides (ASOs), which are short oligonucleotides that regulate gene expression at the transcriptional level, have been confirmed to be safe, tolerable, and effective in treating ALS. This was substantiated by the FDA's approval of Tofersen in 2023.

However, this drug only addresses a small patient population, leaving a high unmet clinical need that could be met by discovering new targets and developing novel ASOs.

In this webinar, experts will offer insight into various aspects of ASO drug development for ALS, including:

  • How Amylyx Pharmaceuticals use a targeted approach for ASO development
  • The process of ASO design, in vitro screening, and efficacy studies
  • The next steps into in vivo proof-of-concept


Evan Mizerak, Lead, Preclinical Research, Amylyx Pharmaceuticals

Susanne Back, Ph.D., Senior Manager, CNS Pharmacology, Charles River

Scientific moderator

Blanca Torroba, Ph.D., Senior Scientist II, Charles River

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