Eikonoklastes Therapeutics is developing ET-101, a first-in-class gene therapy that overexpresses Caveolin-1 in neurons to enhance neuromuscular signaling and function, targeting Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative diseases. This approach aims to provide a disease-modifying treatment for ALS, a condition with limited existing therapies and significant patient need.
Funding
$3.2M 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
Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative diseases lack effective treatments to address the progressive loss of neuromuscular signaling and function. Current therapies offer limited benefits, leaving a significant unmet need for disease-modifying approaches.
Solution
Eikonoklastes Therapeutics is developing ET-101, a gene therapy platform designed to enhance neuromuscular signaling and function by overexpressing Caveolin-1 in neurons. ET-101 utilizes an AAV9 vector with a synapsin promoter for neuron-specific expression and intraspinal delivery to minimize systemic exposure. By increasing Caveolin-1 levels, ET-101 aims to provide a neuroprotective effect, enabling nerve cells to compensate for damage caused by neurodegenerative processes. This approach has demonstrated significant improvements in functional capability, quality of life, and survival in preclinical ALS models. The company is focused on advancing ET-101 through clinical trials, with potential applications in other neurodegenerative diseases beyond ALS.
Target Audience
The primary target audience includes patients with ALS and other neurodegenerative diseases, as well as clinicians and researchers focused on developing novel therapies for these conditions.
Features
- AAV9-based gene therapy for targeted delivery of Caveolin-1 to neurons
- Synapsin promoter ensures neuron-specific gene expression
- Intraspinal delivery minimizes systemic exposure and potential toxicity
- Overexpression of Caveolin-1 promotes neuroprotection and enhances neuromuscular signaling
- Demonstrated improvement in motor function and survival in preclinical ALS models
- Potential application for ALS, Alzheimer’s, Parkinson’s, Huntington’s, and Multiple Sclerosis
- Orphan Drug Designation from the FDA for ALS treatment