A framework designed for discovery
Overview
CREATE combines mRNA construct design, GMP manufacturing infrastructure development, and coordinated preclinical-to-clinical translation to advance three novel categories of mRNA-LNP therapeutics for autoimmune disease. The work integrates discovery science, in vitro and animal studies, and early-phase clinical trial preparation in close coordination with CECTA.
Experimental / Computational Methods
Design and testing of non-inflammatory, targeted mRNA-LNP and IAJD-based constructs across three therapeutic categories — antigen-specific Treg induction, pathogenic effector cell modification, and regulatory T cell immunomodulation; GMP-condition manufacturing of lead constructs; in vitro and animal study validation; and establishment of regulatory and logistical frameworks for Phase I and II clinical trials.
Data Sources / Models Used
In vitro functional datasets validating mRNA-LNP construct activity across therapeutic categories; animal model efficacy and safety datasets; immunoprofiling insights from CECTA clinical trial patients informing construct design; and GMP manufacturing development data from the Penn RNA Institute infrastructure.
Analytical / Translational Focus
Advancement of three novel mRNA-based autoimmune therapeutic categories from construct development through GMP manufacturing and Phase I human trials, targeting type 1 diabetes, lupus, multiple sclerosis, and other severe autoimmune conditions. With the Penn GMP facility operational within 12–18 months and established industry partnerships, CREATE is positioned to deliver the first-ever human trials of these mRNA autoimmune therapeutics.
Powering the science
Ali Naji, MD, PhD, Colton Consortium Member
Jonathan E. Rhoads Professor of Surgical Science II, Department of Surgery, Perelman School of Medicine, University of Pennsylvania
Drew Weissman, MD, PhD, Colton Consortium Member
Roberts Family Professor in Vaccine Research, Department of Medicine, Perelman School of Medicine, University of Pennsylvania
Peter Merkel, MD, MPH, Colton Consortium Member
Professor of Medicine and Epidemiology, Department of Medicine, Perelman School of Medicine, University of Pennsylvania