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

Summary

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.

Timeline

Start

2021-04-08

Primary completion

2026-10-08

Completion

2027-10-08

Publications

• Myers KC, Davies SM, Lutzko C, Wahle R, Grier DD, Aubert G, Norris K, Baird DM, Koga M, Ko AC, Amano T, Amano M, Yu H, Ko MSH. Clinical Use of ZSCAN4 for Telomere Elongation in Hematopoietic Stem Cells. NEJM Evid. 2025 Mar;4(3):EVIDoa2400252. doi: 10.1056/EVIDoa2400252. Epub 2025 Feb 25.

Drugs

Indications