A novel platform to deliver nucleic acids to Schwann cells
to cure peripheral nerve diseases
Our delivery platform is built on lipid nanoparticles (LNPs), a clinically validated system known for its redosability, scalability, and ability to carry large, genetic material also called payloads. Leveraging our unique in vivo local delivery approach, we are able to precisely target Schwann cells in specific anatomical locations, overcoming one of the most significant challenges in LNP therapeutics.
Our payloads are engineered using a rational design strategy that combines synergistic components into a dual-action mechanism, where the whole outperforms the individual parts. By integrating complementary elements, our system delivers potent and durable responses in peripheral nerve tissue.
Lead program: Neurofibromatosis type 1
Neurofibromatosis type 1 (NF1) is a genetic disorder caused by mutations in the NF1 gene, affecting over 3 million people worldwide. It often causes learning and developmental difficulties, skeletal abnormalities, skin pigmentation changes, and vision problems.
A hallmark of NF1 is the growth of tumors along nerve sheaths, which can lead to significant pain, disability, and a high risk of cancer. Currently, only two drugs have been approved to treat these tumors, both with limited efficacy and high dropout rates due to significant side effects.
Neurofibromatosis type 1 (NF1) is a genetic disorder caused by mutations in the NF1 gene, affecting over 3 million people worldwide. It often causes learning and developmental difficulties, skeletal abnormalities, skin pigmentation changes, and vision problems.
A hallmark of NF1 is the growth of tumors along nerve sheaths, which can lead to significant pain, disability, and a high risk of cancer. Currently, only two drugs have been approved to treat these tumors, both with limited efficacy and high dropout rates due to significant side effects.
NF1-corrected cell (dark green)(light green)
Our solution
CureAge is developing the first curative therapy for NF1 designed to treat peripheral tumors in specific body locations.
Our minimally invasive therapy uses Lipid Nanoparticles (LNPs) to deliver genetic treatment directly to tumor Schwann cells, aiming to restore NF1 gene function and eliminate surrounding tumor cells.
This dual approach ensures that all tumor cells are addressed attacking the disease at its root.
With only a few doses per year, the therapy is intended to shrink existing tumors, prevent new ones from forming, and provide a durable, potentially curative effect.
We are currently testing our solution in cellular and animal models.