Bioblast Pharma

Hope should not be rare


Bioblast Pharma Pipeline

A focused pipeline to address unmet therapeutic needs in rare and ultra-rare diseases

Bioblast Pharma has made it a priority to address the significant unmet needs of the rare and ultra-rare disease community focusing our efforts on trehalose 90 mg/mL IV solution, an investigational drug that is currently in Phase 2 development. Bioblast has commenced a scientific and business assessment to determine the suitability of using trehalose IV solution in other orphan diseases.

Given the interest in the scientific and medical community with regard to alternative therapeutic uses of trehalose, we have decided to concentrate our internal development efforts exclusively on this drug candidate, which will enable us to leverage our extensive knowledge and expertise from our R&D and manufacturing efforts.

Bioblast Pharma’s Therapeutic Pipeline

Learn more about Bioblast’s mutant protein stabilization platform