AntagomiR develops anti-miR oligonucleotides targeting upregulated microRNAs in myotonic dystrophy patients, addressing the underlying toxic DMPK and insufficient MBNL levels. Their fatty acid-conjugated delivery system enables effective treatment with a significantly reduced active dose directly to muscle tissue.
Funding
$58.1M 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
Myotonic dystrophy is characterized by upregulated microRNAs that lead to toxic DMPK levels and insufficient MBNL, disrupting normal muscle function. Current treatments do not directly address the underlying genetic causes of the disease.
Solution
Arthex Biotech develops anti-miR oligonucleotides designed to target and modulate the upregulated microRNAs present in myotonic dystrophy patients. Their lead investigational program utilizes a first-in-class mechanism of action to impact the root cause of the disease by addressing toxic DMPK and insufficient MBNL levels. The company employs a proprietary fatty acid-conjugated delivery system to enhance drug delivery directly to muscle tissue, enabling effective treatment with a significantly reduced active dose. This targeted approach aims to restore normal muscle function by correcting the underlying molecular imbalances associated with myotonic dystrophy.
Target Audience
The primary target audience includes patients suffering from myotonic dystrophy and medical professionals specializing in neuromuscular disorders.
Features
- Anti-miR oligonucleotides designed to target upregulated microRNAs in myotonic dystrophy.
- Fatty acid-conjugated delivery system for improved drug delivery to muscle tissue.
- Reduced active dose requirements due to targeted delivery.
- First-in-class mechanism of action impacting the root cause of myotonic dystrophy.