Investor Overview

About Us

Bioblast Pharma is a publicly traded, clinical-stage biotechnology company (NASDAQ - "ORPN") committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases.

Our therapeutic platforms are based on deep understanding of the disease-causing biological processes, and can potentially offer solutions for several diseases that share the same biological pathology.

Given the interest in the scientific and medical community regarding alternative therapeutic uses of our lead drug candidate, trehalose 90mg/mL IV solution, Bioblast has decided to postpone work on our other therapeutic platforms and to concentrate internal development efforts exclusively on trehalose.

The company is led by a management team experienced in the development and commercialization of disease therapeutics.

Corporate Presentations

Developing Novel Therapies for Orphan Neurological Diseases


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