MREO - Newer Treatments In Alpha-1 Antitrypsin Deficiency: $2.5 Billion/Year Market Size By 2032
2024-03-14 17:34:14 ET
Summary
- Alpha-1 antitrypsin deficiency, or AATD, is an inherited disease that affects the lung, liver, and skin.
- The current treatment landscape involves smoking cessation, COPD treatment, and augmentation therapy with AAT.
- Newer therapies in development include elastase inhibitors, novel augmentation therapy, RNA editing, gene therapy, and RNA interference.
Background of the disease and the current treatment landscape
Alpha-1 antitrypsin deficiency, AATD, is an inherited disease affecting the lung, liver, and, rarely, skin. Alpha-1 antitrypsin, AAT, is a protease inhibitor of the proteolytic enzyme elastase. Deficiency of AAT may be inherited (due to mutations in SERPINA1 gene) or induced by smoking. Reduced levels of AAT (below 11 micromol/L) result in lung damage due to elastase enzyme, causing emphysema. Emphysema in AATD is usually bibasilar (bases of lung lobes). Liver disease due to AATD is characterized by chronic liver disease, cirrhosis, and liver cancer. Skin involvement is typically manifested as panniculitis....
Newer Treatments In Alpha-1 Antitrypsin Deficiency: $2.5 Billion/Year Market Size By 2032