Introduction
The Alpha-1 Project (TAP), the venture philanthropy initiative of the Alpha-1 Foundation, is singularly focused on ridding the world of the effects of Chronic Obstructive Pulmonary Disease (COPD) and liver disease caused by Alpha-1 Antitrypsin Deficiency (Alpha-1). Alpha-1 is a rare genetic disorder that can lead to potentially fatal chronic lung disease in adults and chronic liver disease in children and adults.
It is calculated that approximately 100,000 people in the US have the severe form of Alpha-1. Unfortunately less than 10,000 persons have actually been diagnosed with the disease even though the diagnosis of Alpha-1 can be confirmed by a simple blood test.
Drug Therapy Development Pipeline
While currently available therapies may halt the progression of lung disease, there are currently no effective treatments for the liver disease and a cure for Alpha-1 remains elusive. Academic research, to a great extent supported by the Alpha-1 Foundation, has identified novel drug targets but is not in the business of drug development. Large pharmaceutical companies on the other hand have the means of introducing new drugs to the market but until now have been reluctant to invest in new drug development for rare diseases.
Why The Alpha-1 Project? Why Now?
The gap between basic academic research and the marketing of new drugs, the principal impediment to drug development for rare diseases, can be bridged by a partnership between biotechnology companies and voluntary health advocacy (VHA) organizations. Venture philanthropy has emerged as the ideal model for this partnership by linking expertise in biotechnology with the resources of VHA’s including academic researchers, donors and patient populations for new drug testing.
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