Therapeutic Agents for Rare Genetic Diseases
Company Founded Year
Company Number of Employees
Biotechnology & Biopharmaceutical
Bioblast Pharma is a biotechnology company that develops therapies for patients with rare and ultra-rare genetic diseases. The company is building a diverse portfolio of product candidates with the potential to treat patients with incurable rare diseases.
Cabaletta is a mutant protein-stabilizing platform based on a small repurposed molecule. Mutant unstable cellular proteins are the cause of several genetic diseases known as PolyA/Poly Q, including OPMD, SBMA, and SCA3.
BBrm is a read-through platform based on a small repurposed molecule. The platform enables the read-through (or bypassing) of genetic defects, called nonsense mutations or stop codons, that interfere with normal protein formation.
mPRT is a mitochondrial protein replacement platform based on biological fusion proteins. The platform is aimed at mitochondrial protein deficiency diseases and is capable of replacing missing or mutated proteins in mitochondria and restoring cell function.
In March 2019, EnlivexEnlivex Therapeutic, which is developing a treatment for graft versus host disease, a deadly side effect of bone marrow transplants, has announced the completion of its merger into Nasdaq stock exchange shell Bioblast Pharma.