Experimental infusion aims to fix bone marrow failure in rare telomere disease
Disease control
Ongoing
This early-stage trial tests a single infusion of EXG34217 in 12 people aged 12 and older who have bone marrow failure due to telomere biology disorders. The main goal is to check safety and tolerability, while also looking at changes in telomere length and blood cell counts. Bec…
Phase: PHASE1 • Sponsor: Elixirgen Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:02 UTC