Owakihi

Owakihi is a private biotechnology company dedicated to developing transformative therapies for patients with rare and orphan diseases, with a primary focus on central nervous system (CNS) disorders. The company was founded by Sean Nolan, the former President and CEO of AveXis, who led the development and sale of the landmark gene therapy Zolgensma to Novartis. Owakihi leverages the extensive experience of its leadership team in genetic medicines and orphan drug commercialization to identify and advance high-potential therapeutic candidates. Headquartered in Illinois, the firm operates as an incubator-style venture, focusing on lean development cycles for innovative modalities. Its mission is centered on providing life-changing treatments for populations with significant unmet medical needs. The company's name is derived from a Lakota word meaning "to be able," reflecting its commitment to patient empowerment.