Cure Rare Disease

We are an independent, mission-driven health care services business that focuses on orphan, rare disease, cell and gene therapies — the new frontier of health care. Advanced therapies offer new hope for people living with rare disease. We educate stakeholders who participate in supporting care delivery and therapy access that addresses complex challenges within the rare disease community. Each therapy is as unique as the individual relying on it. We partner with providers as part of a coordinated care team. We move past transactional relationships to serve as a trusted partner. Interested in the latest advances in innovative therapies?

Our goal is to provide novel biopharmaceutical medicines that extend, improve, or enhance the lives of people with rare diseases. We are driven to make an impact for patients and their caregivers—from early detection to therapeutic intervention. We are committed to providing differentiated, high-quality solutions that address unmet medical needs for those who suffer from rare diseases.

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. 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. Our goal is to develop innovative therapies for progressive muscle disorders, with the same boldness and courage of the patients and families. We are driven by this urgency to develop novel precision medicines for severe and rare disorders. 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. 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.

We are highly collaborative and partner with leading pharmaceutical companies and academic institutions that can meaningfully advance these programs to market – and patients. Our team is passionate about applying new technologies that leverage the abundant, available human disease data we have today to build drug discovery programs that meet the needs of all cancer patients.

We find new treatments for unaddressed rare diseases through our innovative methodology called Systematic Serendipity, which creates working partnerships between patient groups and R&D companies.

We are leveraging a toolkit of RNA-targeting modalities, chemistry innovation and deep insights in human genetics to deliver scientific breakthroughs that treat both rare and prevalent disorders. We are advancing a pipeline of first- or best-in-class therapeutics with life-changing potential, including in Alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy, Huntington’s disease, and obesity. We are seeking bold, enterprising individuals to join our team. Our team includes some of the world’s most respected oligonucleotide chemists, drug development experts, and dedicated patient advocates.