Grann Pharmaceuticals
mRNA Therapeutics for Protein Replacement
At Grann Pharmaceuticals, innovation is our foundation. We are advancing mRNA lipid nanoparticle therapeutics for diseases associated with missing or deficient proteins. Our goal is to translate scientific research into treatment options for patients and families facing serious unmet medical needs.
Our Vision
The Challenge
Some genetic conditions disrupt the production or function of proteins essential to healthy development and everyday life.
Our Approach
We develop mRNA lipid nanoparticle therapeutics designed to deliver instructions that cells can use to produce a protein of interest.
Our Science
Explore the science behind our mRNA lipid nanoparticle platform and its application across our investigational programs.
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.
Commitment to Rare Disease
At Grann Pharmaceuticals, our commitment to rare disease begins with the patients and families who inspire our work. We collaborate with patient advocacy organizations, clinicians, and researchers to advance investigational mRNA therapies for Rett syndrome and other rare genetic conditions. Together, we aim to turn scientific progress into meaningful treatment possibilities for communities with significant unmet needs.
Investors & Partnerships
We welcome investment and strategic collaborations to advance our mRNA lipid nanoparticle platform and investigational programs across rare genetic disorders, oncology, and metabolic disease. Connect with our team to discuss opportunities to support our next stages of research and clinical development.