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 | Rett Syndrome
RTT-1 is an investigational mRNA lipid nanoparticle therapeutic designed to support MECP2 protein expression. Two girls have received RTT-1 through single-patient expanded access, with third-patient dosing upcoming and Phase 1 development planned.
Clinical monitoring, laboratory assessments, and caregiver observations are informing continued development. These individual observations do not establish safety or efficacy.
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.