Pipeline
Our pipeline applies Grann’s mRNA lipid nanoparticle platform across rare genetic disorders, oncology, and metabolic disease. We are advancing investigational programs at different stages of research and development, with RTT-1 for Rett syndrome as our lead program.
RTT-1: Progress in Rett Syndrome
RTT-1 is our lead investigational mRNA lipid nanoparticle therapy for Rett syndrome, designed to support MECP2 protein expression. Two girls, Eleanor and Indie, have received RTT-1 through single-patient expanded access. Their experiences are helping inform continued development as we prepare for a planned Phase 1 study.
Connect With Grann
We welcome inquiries from patients, families, clinicians, researchers, and potential partners interested in our pipeline or other rare genetic diseases. Contact our team to discuss your questions and opportunities for collaboration.