Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure

Description

This is a Phase I/II, open label study in up to 12 subjects with telomere biology disorders with bone marrow failure. The study is open to all participants regardless of gender or ethnicity. Subjects who are enrolled but not evaluable will be replaced.

Subjects will sign a consent form prior to any study related procedure and will complete baseline screening assessments. Subjects for this study will not require any preparative regimen such as chemotherapy or radiation.

The study will be conducted in three parts

* Peripheral blood mononuclear cells (PBMNCs) collection; mobilization and apheresis,
* Ex vivo cell processing
* Processed cell infusion and post-infusion safety monitoring,
* Follow-up (Week 2, 3,4,5, Months 1, 2,3,4,5,6,9 and 12)