Blood disorders often require lifelong treatment involving routine drug infusions or blood transfusions. They can also be associated with frequent visits to physician offices, infusion centers or even the emergency room. Patients with these disorders often experience debilitating pain, disability, reduced life expectancy and quality of life. Potential gene therapies may offer long-term benefits and even cures for patients with blood disorders with a single administration. Since they may drastically reduce the burden of the current standard of care, they may also:

• Reduce the need for patients to miss work for treatment and/or symptoms, leading to economic benefits for patients
• Dramatically reduce existing treatment burdens for patients
• Result in savings for the health care system