The company develops siRNA-based targeted therapies for nervous system pathologies, focusing on demyelinating diseases like CMT1A. Their platform modulates gene expression at low doses to directly target damaged tissues, offering a novel treatment approach.
Funding
Funding not disclosed
Founders
Product
Problem
Many nervous system pathologies lack effective treatments. Charcot-Marie-Tooth disease type 1A (CMT1A), a prevalent hereditary peripheral neuropathy affecting 1 in 5,000 people, is one such condition with limited therapeutic options.
Solution
MAAsiRNA is developing targeted therapies for nervous system pathologies, with a focus on demyelinating diseases like CMT1A. The company's platform utilizes small interfering RNA (siRNA) conjugated with squalene to modulate gene expression. Squalene facilitates the creation of nanoparticles that can cross cell membranes and directly target damaged tissues following intravenous administration. This approach allows for effective modulation of gene expression at low doses, normalizing overexpression of the targeted mRNA, restoring nerve conduction velocity, and rehabilitating motor activity without observed toxic effects in preclinical studies.
Target Audience
The primary target audience includes individuals affected by Charcot-Marie-Tooth disease type 1A (CMT1A) and potentially other demyelinating diseases, as well as the medical professionals who treat these conditions.
Features
- Tailored siRNA sequences designed to target specific mRNA implicated in nervous system disorders.
- Squalene conjugation for siRNA delivery, leveraging its self-assembling properties to form nanoparticles in aqueous environments.
- Targeted delivery of siRNA to Schwann cells, directly addressing the site of damage in demyelinating diseases.
- Demonstrated efficacy in three transgenic CMT1A mouse models, normalizing mRNA overexpression and restoring nerve function.
- Biocompatible and biodegradable lipid nanoparticles, minimizing potential toxicity.