Company intelligence / Profile preview
Elixirgen Therapeutics, Inc.
Baltimore, Maryland, USAelixirgentx.com ↗
- Ownership
- Private
- Development stage
- Other
01
Overview
Elixirgen’s lead candidate EXG‑34217 is an autologous cell therapy using the ZSCAN4 gene to treat telomere biology disorders. Early Phase I/II data show successful ex vivo and in vivo telomere elongation in patients with TBDs without toxicity or need for immunosuppression—an unprecedented result in this field. The company has received FDA Orphan Drug, Rare Pediatric Disease, and RMAT designations for EXG‑34217. Preclinical work includes Bobcat mRNA™ technology showing promise in restoring dystrophin expression in a mouse model of Duchenne muscular dystrophy.
- Therapeutic areas
- HematologyRare Diseases
- Modalities
- mRNA therapeutics (including self-replicating RNA)Autologous cell therapy (CD34+ hematopoietic stem cells)Gene therapy
- Industry
- Biotech
02
Drug pipeline
5 assets4 more assets in the full pipeline.
03
Partnerships
Hitachi Global Life Solutions – collaboration on microfluidic manufacturing of autologous cell therapies.
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