Patients are at the core of everything we do.
We recognize that for patients with rare and debilitating diseases, every day without an effective treatment is a day too late. We are driven by this urgency to evolve our understanding of rare progressive muscle disorders. Our goal is to develop novel precision medicines to help protect and enhance muscle function. Our intimate knowledge of muscle biology and biophysics combined with our ability to identify and design muscle specific precision small molecules has enabled us to rapidly advance our clinical and preclinical research pipeline. We continue to build a leading, global biopharmaceutical company focused on rare disorders involving muscle dysfunction in order to develop transformative precision medicines to treat and possibly, someday cure rare muscle disorders.
We are focused on the discovery, development and commercialization of innovative treatments for severe, rare muscle disorders for which there is significant unmet medical need. We are currently testing our lead drug candidate for muscular dystrophy in clinical studies. Guided by our drug discovery approach to target the muscle as an organ, we are developing novel therapeutic approaches using small molecules that target myosin to treat rare muscle disorders. Our platform utilizes custom-built high throughput and translatable systems that measure integrated muscle function in whole organ extracts to identify small molecule precision medicines regulating key proteins in muscle tissue.
Our vision is to improve the lives of patients and families suffering from rare muscle disorders by building the world’s leading muscle-focused, precision medicine company. Key components of our strategy to achieve this vision include:
Engaging with patients and their families and physicians to develop trusted relationships, transparent communications, and become a leader in the muscular dystrophy community;
Leveraging clinical and regulatory precedents and our experience in rare diseases to rapidly advance EDG-5506 through clinical development in Duchenne and Becker muscular dystrophy (Duchenne, Becker) and potentially other muscle diseases;
Evaluating strategic collaborations and asset acquisition opportunities to accelerate development and commercialization timelines as well as potentially expand our pipeline within our core therapeutic areas.