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According to the World Health Organization, tuberculosis (TB) causes approximately 1.5 million deaths worldwide each year. More than one-third of the world’s population has tested positive for Mycobacterium tuberculosis (Mtb), the bacterium that causes TB, but are not yet ill (latent infection). Of these, approximately ten percent are expected to progress to active TB infection. Treatment is lengthy, and the drugs often come with serious side effects. This has led to poor treatment compliance and allowed the rise of drug-resistant TB strains. 

On July 20, 2012, a committee of the European Medicines Agency recommended for regulatory approval the first gene therapy using an adeno-associated virus vector to treat lipoprotein lipase deficiency (LPLD), a very rare genetic disease. With a normal diet, patients lacking sufficient levels of lipoprotein lipase have abnormally high serum triglycerides and high levels of very low-density lipoprotein (VLDL), resulting in acute severe pancreatitis and chronic conditions associated with high levels of VLDL, such as cardiovascular diseases. 

Malaria is a life-threatening disease transmitted through the bite of mosquitoes infected with malaria parasites. In 2012, there were an estimated 219 million cases of malaria and an estimated 660,000 deaths, mostly among young children in sub-Saharan Africa. With the effectiveness of current drugs diminishing as resistant strains of malaria have emerged, new drugs are urgently needed. The plasmodial surface anion channel (PSAC) found on the surface of red blood cells infected with malaria parasites offers an opportunity to develop new drugs to treat and prevent malaria.

In April 2013, President Obama unveiled the “BRAIN” initiative, which called on the scientific community to better understand the human brain in an effort to treat, prevent, and cure neurological diseases. For example, anxiety, depression, substance abuse, and post-traumatic stress are a few pervasive neuropsychiatric diseases that afflict more than 150 million people in developed countries, and approximately 15 million of those are in the U.S.

Niemann-Pick disease, type C (NPC) is a lethal, neurodegenerative disorder that affects children. Presently, no therapies for NPC are approved by the Food and Drug Administration (FDA). Several studies have suggested the potential use of 2-hydroxypropyl-β-cyclodextrin (HPBCD) to treat NPC, but the critical studies and data required for an Investigative New Drug (IND) application to evaluate HPBCD were not available.

On March 10, 2015, the Food and Drug Administration (FDA) approved Unituxin™ (dinutuximab) as part of first-line therapy for pediatric patients with high-risk neuroblastoma, a rare cancer that most often occurs in young children. This approval was the result of a collaborative effort between the National Cancer Institute (NCI), the Children’s Oncology Group, and United Therapeutics Corporation (UTC) for the first approved therapy for pediatric high-risk neuroblastoma.

On March 10, 2015, the U.S. Food and Drug Administration (FDA) approved Unituxin™ (dinutuximab) as part of first-line therapy for pediatric patients with high-risk neuroblastoma, a rare cancer that most often occurs in young children. This approval was the result of a collaborative effort among the National Cancer Institute (NCI), the Children’s Oncology Group, and United Therapeutics Corporation (UTC) for the first approved therapy for pediatric high-risk neuroblastoma.