Elixirgen Therapeutics, Inc.

FDA Grants RMAT Designation to Elixirgen's Novel Gene Therapy for Rare Telomere Disorders

3 months ago

• Elixirgen Therapeutics' gene therapy EXG-34217 receives FDA's Regenerative Medicine Advanced Therapy designation for treating Telomere Biology Disorders with bone marrow failure. • The therapy, currently in Phase 1/2 trials, uses modified hematopoietic stem cells expressing ZSCAN4 protein to address telomere elongation in patients with these rare genetic conditions. • RMAT designation provides accelerated development and review pathways, including Fast Track and Breakthrough Therapy benefits, for this treatment targeting disorders affecting 1 in 1 million Americans.