Bioblast Pharma

Hope should not be rare

About Us

About Bioblast Pharma

Focused on finding therapeutic solutions for patients in need

A dedication to pioneering treatments for rare and ultra-rare diseases

Founded in 2012, Bioblast Pharma is a publicly traded, clinical-stage biotechnology company (NASDAQ: ORPN) built on the premise of discovering, developing, and delivering clinically meaningful therapies for patients with rare diseases. The company is led by a team of passionate and experienced individuals who share a single goal—improving the lives of patients with rare and ultra-rare genetic diseases.

Bioblast Pharma approaches scientific discovery with focus and fervor, concentrating efforts on the underlying causes of disorders in order to design first-in-class disease modifying therapies that may effectively change the course of the targeted disease.

Given the interest in the scientific and medical community regarding alternative therapeutic uses of our lead drug candidate, trehalose 90 mg/mL IV solution, Bioblast has decided to concentrate internal development efforts exclusively on trehalose. Our hope is to address high unmet medical needs and offer solutions for multiple diseases that share the same biological pathology.

A commitment of unity with the rare and ultra-rare genetic disease community

With innovation as a guiding principle, Bioblast Pharma is committed to maintaining the highest scientific integrity to ensure our mission never wavers. That’s why we are working collaboratively with practitioners, patients, patient advocacy groups, and caregivers, to not only provide groundbreaking medications, but also to deliver the comprehensive support services patients deserve.

Learn about Bioblast’s mission