Allele-Specific Protein-Based Therapeutics for Myotrophic Lateral Sclerosis Associated with C9ORF72 Repeat Expansions
Abstract
Enzerna Biosciences is developing a novel treatment approach addressing the underlying disease mechanism of familial ALS. By modifying defective RNA molecules found in the neurons of ALS patients, Enzerna s technology will ameliorate the underlying cell pathologies that lead to neuronal death. As opposed to symptomatic treatment, Enzerna is treating the fundamental disease process to improve patient outcomes. Enzerna s therapeutic will help patients with an inherited form of the disease known as familial ALS. Mutation in the C9orf72 gene is the most common cause of familial ALS (~40% of cases) and is inherited dominantly, meaning only one copy of the gene is needed to cause disease. Patients with C9orf72 mutation also tend to have earlier disease onset and reduced survival. Currently, no disease-modifying treatments are available to these patients. Enzerna s treatment has the potential to change the disease trajectory for these patients by mitigating their underlying neurodegeneration, improving neuron survival and the patient s quality of life. Enzerna intends to develop this therapeutic as a Food and Drug Administration-regulated drug administered directly into the spinal cord. The benefit of our therapeutic is fundamental disease process modification to preserve patients neurons, mitigating motor symptoms seen in patients. The primary contribution of this study is to develop a novel therapeutic mechanism to treat ALS. Enzerna s technology can provide patient-specific modification to mutant RNA molecules in patients neurons. This targeted approach has never been attempted for ALS.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Dec 28, 2022
- Source ID
- W81XWH2210334
Entities
People
- Joseph Ruiz
Organizations
- United States Army