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.
Blood-Brain Barrier
Grann Pharmaceuticals Inc. has put forth continued research efforts into developing a lipid nanoparticle delivery system capable of crossing the Blood Brain Barrier (BBB). Current research indicates that any particle < 100 nm - 150 nm has the capability to cross the BBB (Nance et al., 2012).
Here at Grann Pharmaceuticals, we have tailored our lipid nanoparticles to an average size of 80 nm to successfully deliver mRNA and small molecules across the BBB. This has been observed in multiple animal trials spanning across several different species of mice.
Our goal at Grann Pharmaceuticals is to apply this lipid nanoparticle technology in therapeutics for CNS disorders and neurological diseases such as Obesity, Rett Syndrome, GABA-A Variants, Alzheimer’s and cancer.
