Elixirgen Therapeutics has expanded its rare disease pipeline with a licensing option for EXG-7001 and clinical progress for EXG-34217.
On 26 June, Elixirgen entered an option agreement with Nippon Shinyaku under which Elixirgen will lead development of EXG-7001, a locally administered full-length dystrophin mRNA therapeutic in preclinical development for Duchenne muscular dystrophy; Nippon Shinyaku may obtain exclusive worldwide commercialization rights, will fund development costs, and will make an upfront payment and potential development and sales-based milestone payments, with NS Pharma positioned to market in the United States if the option is exercised and regulatory approval is obtained.
In a separate program, Elixirgen published early Phase 1/2 data on 25 February 2025 showing sustained telomere elongation in blood cells and increases in absolute neutrophil count in two treated patients with telomere biology disorders, with no treatment-related adverse events reported over follow-ups of 24 months and five months.
EXG-34217 uses autologous CD34+ hematopoietic stem cells transiently expressing ZSCAN4 and is described as mutation-independent. The FDA has granted EXG-34217 Orphan Drug, Regenerative Medicine Advanced Therapy, and Rare Pediatric Disease designations, according to the company.
