Aeglea is committed to developing engineered human enzymes for treating rare diseases and cancers associated with abnormal amino acid metabolism.
Generics & 505(b)(2) drugs
Belrose Pharma focuses on rare diseases and uses its proprietary “releasable linker” conjugation technology and other techniques to improve the clinical efficacy of both novel therapeutics and ...
BioBlast Pharma manufactures Trehalose, a therapeutic to potentially treat a number of devastating PolyA/PolyQ diseases. Trehalose acts as a chemical chaperone to protects against pathological ...
SGK agonists for the treatment of rare, ophthalmological diseases
Fibrodysplasia ossificans progressiva treatments
Orphan inflammatory-fibrotic diseases
Orphan regulatory affairs, risk evaluation, and streamlined project management
Early-stage research on 505(b)(2) products
Rare disease treatment
Orphan Disease Treatments
Biotherapeutics for orphan genetic diseases
Orphan Indicated Small Molecule