Eikonoklastes Therapeutics is an early stage biopharmaceutical company focused on rapidly advancing innovative medicines for numerous areas of significant unmet clinical need, starting with neurodegenerative diseases. We are iconoclasts who are passionate about finding new and better ways to treat disease.
Our first-in-class gene therapy moves beyond single defective gene replacement. It is a transformational, disease-modifying gene supplement therapy with a unique mechanism of action that provides extra copies of a critical gene to cause overexpression of a critical neurotrophic factor that is essential for neuromuscular signaling and function. Our initial focus is on Amyotrophic Lateral Sclerosis (ALS, Lou Gehrig’s Disease), a rare, yet debilitating nerve disease that causes progressive muscle degeneration and eventual death. An estimated 5,000 new patients in the U.S. are diagnosed each year with ALS, and there are few treatment options beyond supportive care. Our dynamic discovery and development approach allows us to focus on accelerated approvals and outsized returns for ALS and potentially other neurodegenerative diseases such as Parkinson’s, Huntington’s, Alzheimer’s, traumatic brain injury, and more.
