Our lead compound is being developed to treat systemic lupus erythematosus (SLE), an autoimmune disease in which the body’s immune system mistakenly attacks normal tissues in the body. Clinical presentation and symptoms vary widely and can include fatigue, arthritis, chest pain, kidney damage, and neurologic symptoms. Patients with SLE face a higher risk of cardiovascular illness and death.
SLE is considered an orphan disease in the US. “Orphan” status allows the drug sponsor to benefit from a range of incentives for the development of these products, including:
- Tax credits on clinical research
- Technical assistance during the filing of a marketing application, simplification of administrative procedure, reduction of the waiting period, and reduction of the amount of registration fees
- Marketing exclusivity of 7 years after marketing approval is granted
Other Rare Diseases
Along with our program in SLE, we are focused on addressing a range of rare diseases where mTORC1 activity is known to be elevated. These include serious neurodegenerative diseases along with several rare genetic disorders. We are dedicated to bringing our science to bear to fight these diseases that greatly reduce both lifespan and quality of life.