Bioprinted Liver Tissue Could Help Treat A1AD, And it Just Got Orphan Drug Status

Organovo Holdings recently stated that its 3D bioprinted therapeutic liver tissue was granted orphan drug status by the Food and Drug Administration (FDA). Taylor Crouch, the company CEO, was excited about the decision. He hopes that the move will allow similar types of technology to enter the development phase. The bioprinted tissue was designed specifically for the treatment of alpha-1 antitrypsin deficiency (A1AD).
This deficiency may result in disease of the liver or lung. The cause has been linked to a mutation in the SERPINA1 gene that results in less A1AT being produced than normal. The likelihood of lung problems increases with smoking or dusty air. Treatment may include bronchodilators or antibiotics when infection in present. In severe cases, a transplant may be necessary. A transplant is also an option when the liver is starting to fail. In people who smoke, live expectancy can drop significantly, but is almost unaffected in those who do not. Many medical experts are convinced that the disease remains undiagnosed in patients with Chronic Obstructive Pulmonary Disease (COPD). To learn more about A1AD, click here.

The orphan drug designation was developed specifically to provide an incentive for pharmaceutical companies to research and design treatments for rare diseases. Previously, few companies chose to pursue rare disease treatments because the demand for them would be lower. The designation allows a seven-year term for the company to exclusively develop the treatment and also includes the waiving of certain charges and fees associated with the development process. This will allow the treatment, if successful, to make its way to the public more efficiently.

Organovo’s unconventional approach to medicine is based around 3D bioprinted tissue, such as the liver that is being developed to treat A1AD. The bioprinted tissue is meant to replicate real organ tissue as accurately as possible. Meant to function as a replacement for actual tissue, the company hopes that it can eventually be used for surgical procedures and transplants. Organovo was the first company to begin conducting research in order to commercialize the bioprinting technology. The company also hoped to be able to print miniature organs that will eliminate the need the use of animals in testing. Read more about it in the Financial Buzz.

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