About IntraBio Glossary
IntraBio is a biopharmaceutical company with a late-stage drug pipeline that includes novel treatments for genetic and neurodegenerative diseases. Their clinical programs leverage the expertise of their scientific founders from the University of Oxford and University of Munich, the preeminent experts and pioneers in discovering and developing small molecule drugs that modulate lysosomal function and intracellular calcium signaling.
With their successful track record of drug development and commercialization, IntraBio’s team translates research in the fields of lysosomal biology, autophagy, and neurology into orphan drugs and treatments that will significantly improve the lives of patients, their caregivers, and families.
ASMD – Acid Sphingomyelinase (Niemann-Pick Type A/B & Type B)
EMA – European Medicines Agency (European equivalent of FDA)
ERT – Enzyme Replacement Therapy
FDA – United States Food and Drug Administration
HDAC – Histone Deacetylase Inhibitors
ICV – Intracerebroventricular
IND – Investigational New Drug
i-IND – Individual Investigational New Drug
IRB – Institutional Review Board
NICHD – National Institute of Child Health and Human Development
NIH – National Institutes of Health
NPC – Niemann-Pick Disease Type C Disease
TRND – Therapeutics for Rare and Neglected Diseases