Past Project
Nanoheme
2023 / 2025
About
The work, in plain language.
Hemophilia A is a rare genetic condition in which patients lack functional factor VIII, a key blood-clotting protein. Standard care requires multiple intravenous injections per week, and existing AAV-based gene therapies struggle to package the large factor VIII sequence and carry a risk of oncogenic mutagenesis from viral integration.
Nanoheme designed optimized lipid nanoparticles for the delivery of an mRNA gene therapy encoding factor VIII. mRNA-LNP systems, popularized by the COVID-19 vaccines, sidestep the integration risk, do not require a promoter, and have a smaller delivery volume per dose than AAV.
The work focused on synthesis and characterization of LNPs with adequate packaging capacity, encapsulation efficiency, and a favorable immunogenicity profile for repeated dosing.
Project archive
Images from the original project page.
These images are preserved in the same order as the legacy McGill BioDesign page.
View the original project pageNanoheme project archive, image 1 of 4.
1 / 4
Team
Led by.
Every project starts with a lead who carries it from idea to build. Want to lead the next one?
Related