Expansion Therapeutics develops oral small molecules that target RNA to treat severe RNA-mediated diseases, particularly major neurological disorders. By utilizing the SMiRNA technology, the company aims to provide effective therapeutic options for conditions that currently lack adequate treatment.
Funding
$85M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Many neurological disorders are caused or exacerbated by RNA dysfunction, yet effective treatments that directly target RNA are lacking. Current therapeutic options often fail to address the underlying molecular mechanisms of these RNA-mediated diseases.
Solution
Expansion Therapeutics develops orally available small molecule drugs designed to selectively bind to and modulate RNA targets implicated in severe neurological conditions. Their SMiRNA technology platform identifies and optimizes small molecules that can correct aberrant RNA function, leading to potential disease-modifying therapies. By directly targeting RNA, Expansion Therapeutics aims to address the root cause of these disorders, offering a more precise and effective treatment approach compared to traditional methods. This approach has the potential to create transformative medicines for diseases with limited or no existing treatment options.
Target Audience
The primary target audience includes patients suffering from severe RNA-mediated neurological diseases and the healthcare professionals who treat them.
Features
- SMiRNA technology platform for identifying small molecules that bind to specific RNA targets.
- Focus on developing orally available drugs for improved patient convenience and compliance.
- Targeting RNA dysfunction to address the underlying cause of neurological disorders.
- Development of disease-modifying therapies with the potential to slow or reverse disease progression.