A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease

Key Inclusion Criteria:

Part 1 (Healthy participants)

• Healthy male participants and female participants of non-childbearing potentialbetween 18-55 years of age

• In good health as determined by the investigator's assessment of medical history,physical examination, vital signs, ECG, and laboratory tests

• Participants must weigh at least 50 kg at screening and first baseline (admission)and must have a body mass index (BMI) within the range of 18.0-32.0 kg/m2 inclusive.

Part 2 (Sickle Cell Disease)

• Male and female participants with a diagnosis of sickle cell disease

Key Exclusion Criteria:

Part 1 (Healthy participants)

• QTcF ≥ 450 msec (as a mean value of triplicates)

• History of arrhythmias

• History of significant illness which has not resolved within two (2) weeks prior toinitial dosing

• Women of child-bearing potential (WOCBP)

Part 2 (Sickle Cell Disease)

• Current use of hydroxyurea/hydroxycarbamide (HU/HC)

• QTcF ≥ 450 msec (as a mean value of triplicates)

• History of arrhythmias

Other protocol-defined inclusion/exclusion criteria may apply.