Porphyria Clinical Trials

• Aged 18 years or older at the time of signing the informed consent form (ICF)

• Diagnosis of CEP, based on medical history and/or biochemical porphyrin analysis. Sponsor will review the CEP diagnostic criteria (medical history or biochemical porphyrin analysis) of each potential patient, written Sponsor approval before starting the run-in period will be required

• a) Female participants must not be pregnant or lactating at screening or admission

⁃ 3\. b) Female participants must be of nonchildbearing potential or agree to use 2 adequate forms of highly effective method(s) of contraception throughout the entire duration of study participation and for at least 4 weeks after the last dose of any study intervention and have negative pregnancy test results at screening (serum) and admission (urine) 4. Male participants with female partners of childbearing potential must be surgically sterile (successful vasectomy at least 4 weeks prior to Day 1) or agree to use adequate forms of highly effective contraception (together with their female partner) for the duration of the study and for at least 4 weeks after the last dose of study intervention; must also agree to refrain from sperm donation during the study and for at least 4 weeks after the last dose of study intervention 5. Negative pregnancy test (females of childbearing potential) at Screening (Month -6), at Baseline (Day 1) and at every visit site until EoS prior to dosing 6. Able to understand the study aims, procedures, and requirements, and provide written informed consent 7. Able to comply with all study procedures 8. Able and committed to complete study patient diaries and questionnaires