This is a Phase I/II, open label, single center study to assess the safety and tolerability of EXG34217 in bone marrow failure patients with telomere biology disorders.
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Number of participants with adverse events -Safety by Incidence of Treatment-Emergent Adverse Events
Timeframe: Multiple times for the duration of the study (baseline through Month 12)
Number of participants with a change in in physical examination
Timeframe: Multiple times for the duration of the study (baseline through Month 12)
Number of participants with a change in Electrocardiography (ECG)
Timeframe: Multiple times for the duration of the study (baseline through Month 12)
Number of participants with a change in clinical laboratory evaluations
Timeframe: Multiple times for the duration of the study (baseline through Month 12)
Number of participants with a change of Immunogenicity
Timeframe: Multiple times for the duration of the study (baseline through Month 12)