Ligandal is developing technology to predictively deliver to virtually any cell, tissue or organ.
ligand - a molecule that binds to another (usually larger) molecule.
Ligandal does this by harnessing the power of ligands, biomolecules that stick to other biomolecules, binding to the precise receptor profiles of targeted cell populations. Using a combination of computational techniques, state-of-the-art robotics and a unique nanomaterials platform, Ligandal is able to create extremely tailored gene therapy approaches.
Ligandal's goal is to create a platform for targeting specific cells, tissues and organs and genetically reprogramming our biology to be free of disease, ushering in a new era of genetically tailored, super-personalized medicine with minimal side effects.
ligand - a molecule that binds to another (usually larger) molecule.
Ligandal does this by harnessing the power of ligands, biomolecules that stick to other biomolecules, binding to the precise receptor profiles of targeted cell populations. Using a combination of computational techniques, state-of-the-art robotics and a unique nanomaterials platform, Ligandal is able to create extremely tailored gene therapy approaches.
Ligandal's goal is to create a platform for targeting specific cells, tissues and organs and genetically reprogramming our biology to be free of disease, ushering in a new era of genetically tailored, super-personalized medicine with minimal side effects.
Redefining precision healthcare.
CRISPR must enter a cell's nucleus to perform its function. Shown is super-resolution microscopy of CRISPR ribonucleoprotein (RNP) delivery into the nucleus of a human primary T-cell utilizing Ligandal's cell-specifically targeted gene editing nanomaterials. Green: CRISPR RNP. Blue: nucleus. Red: actin. Scale bar = 500 nanometers.
Shown are Ligandal's peptide-mediated CRISPR gene editing efficiencies of the T cell receptor's TRAC locus in a mixture of CD4+ and CD8+ unstimulated human T cells treated with 54 unique formulations of Ligandal's materials, as measured via Sanger sequencing. Nucleofection controls are shown in wells C11 - F11. Chemically modified gRNA was provided by Synthego.
Shown is Cas9-EGFP (Cas9+) and AF647-tagged peptide (NP+) live cell uptake 1 day post-transfection in unstimulated primary human T cells for CD4+ (top) and CD8+ (bottom) subpopulations grown in co-culture and treated with 90 unique formulations of Ligandal's materials. Measurements were performed with an Attune NxT flow cytometer.
Nucleofection + Lipofection controls are shown in wells G11 for EGFP-Cas9-NLS and F11 for NLS-Cas9-NLS. Negative controls are shown in well H11. Controls lacked the AF647 tag (NP+ tag), however were 26% and 13% CRISPR-EGFP+ at 24h post-transfection for CD4+ and CD8+ subpopulations, respectively, with treatment with Lipofectamine CRISPRMAX followed by nucleofection.
Lipofectamine CRISPRMAX does not perform in these cell types without subsequent nucleofection (data not shown).
Nucleofection + Lipofection controls are shown in wells G11 for EGFP-Cas9-NLS and F11 for NLS-Cas9-NLS. Negative controls are shown in well H11. Controls lacked the AF647 tag (NP+ tag), however were 26% and 13% CRISPR-EGFP+ at 24h post-transfection for CD4+ and CD8+ subpopulations, respectively, with treatment with Lipofectamine CRISPRMAX followed by nucleofection.
Lipofectamine CRISPRMAX does not perform in these cell types without subsequent nucleofection (data not shown).
THE CURRENT STAGE OF DEVELOPMENT.
A NEW PARADIGM FOR MEDICINE.Message us to learn more.
|