February 2025

Elixirgen Therapeutics Publishes Early Results Showing Telomere Elongation in First Gene Therapy Trial for Telomere Biology Disorders

Publication /

Data published in NEJM Evidence detail the first clinical test of ZSCAN4 for telomere elongation. Treatment with EXG-34217 resulted in in vivo telomere elongation in a subpopulation of lymphocytes to the normal range for age and increases in absolute neutrophil count in two patients. No treatment-related adverse events were observed. U.S. FDA has recently granted […]

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Elixirgen Therapeutics Granted FDA Orphan Drug Designation for EXG-34217 for Treatment of Telomere Biology Disorders

Press Release /

BALTIMORE, February 20, 2025 – Elixirgen Therapeutics, Inc., a clinical-stage biotechnology company focused on treating rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to EXG-34217, a gene therapy for treatment of Telomere Biology Disorders (TBDs). EXG-34217 is currently being studied in an ongoing Phase 1/2 trial

Elixirgen Therapeutics Granted FDA Orphan Drug Designation for EXG-34217 for Treatment of Telomere Biology Disorders Read More »

Elixirgen Therapeutics Granted FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for EXG-34217 for Treatment of Telomere Biology Disorders

Press Release /

BALTIMORE, Feb. 13, 2025 (GLOBE NEWSWIRE) — Elixirgen Therapeutics, Inc., a clinical-stage biotechnology company focused on treating rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to EXG-34217, a gene therapy for treatment of Telomere Biology Disorders (TBDs). EXG-34217 is currently being studied in

Elixirgen Therapeutics Granted FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for EXG-34217 for Treatment of Telomere Biology Disorders Read More »