Bringing Genetic Therapy to Rare Kidney Diseases
Time: 9:30 am
day: Conference Day Two
Details:
- Developing ELX-02: New small molecule genetic therapy to treat Alport patients with nonsense mutations developed for superior PTC readthrough compared to Gentamicin. Focusing on rare kidney diseases
- Compelling proof of concept in Phase 2 study showing that in only 8 weeks ELX-02 treatment improved morphology by reducing podocyte foot process effacement in Alport Syndrome patients with nonsense mutations
- Planning a larger, longer trial to validate study results
