Seeing Is Believing: A Gene Therapy First

Ligandal’s Bioinspired Nanomaterials Deliver CRISPR into Nucleus of a T-Cell

SAN FRANCISCO, May 23, 2018 (GLOBE NEWSWIRE) -- For the first time, a ligand-targeted nanoparticle has been successfully used to deliver CRISPR into the nucleus of a T-cell.

“From the start, CRISPR and other genetic therapies have suffered from the classic ‘last-mile’ problem,” said Andre Watson, founder, chairman and CEO of Ligandal. “For all their power to heal, genetic therapies are ineffective at best if they cannot be delivered to the precise cellular subtypes and locations inside cells.

“Now they can be,” Watson said.

Ligandal’s bioinspired nanomaterials utilize peptide targeting ligands, which bind to precise cellular surface profiles of targeted cell populations, to target CRISPR and other genetic materials into T-cells or other cells. The ligand targeting capacity is an industry first for these gene therapy modalities: Ligandal’s technology can selectively target T-cells with manifold greater efficiency than other blood cell populations.

“In part, the flexibility and ease of this platform is enabled by state-of-the-art peptide synthesis and fluid handling robotics, driven by a back-end machine-learning approach to rapid ligand and nanomaterials discovery,” Watson said. “Of course, none of these robotics or machine learning approaches would be meaningful without our breakthroughs in bioinspired nanomaterials design and synthesis.”

In addition to Ligandal’s breakthroughs in high-throughout biological nanomaterials screening, the company has demonstrated that its first-in-class ligand-targeted particles are efficiently performing gene edits.

In a representative image, Ligandal documented the delivery of CRISPR to a T-cell nucleus via 3D super-resolution microscopy. Such an image has never been shown for a nanoparticle delivering a gene editing tool, and Ligandal is the first to demonstrate ligand-targeting of CRISPR via a non-viral construct. Furthermore, these particles are serum stable, forming the basis of a breakthrough approach to in vivo gene therapy.

“This is just the start,” Watson said. “Our novel imaging pipeline coupled to our leading genomics, peptide synthesis, gene editing and biological nanomaterials expertise will ramp up production by giving us real-time quantification of nanoparticle uptake and gene-editing efficiency for thousands of nanoparticle variants per week. Most importantly, this infrastructure is forming the basis of the first diagnostically-responsive nanomaterials lab the world has ever seen. We are pleased to have brought on leading scientists in the field to drive us forward on our mission of creating curative, scalable, cost-effective genetic therapies for humanity at large.”

Ligandal is now working to program T-cells to target markers of viruses and cancer cells, as well as developing a novel set of nano-immunotherapies with no industry precedent. Current data shows that Ligandal’s approach can differentially target human primary T-cells within mixed blood cell populations, delivering CRISPR and other genetic tools with high efficiency and performing gene edits. The company has also generated compelling data in bone marrow and additional blood models.

Watson gave thanks to Shuailiang Lin, PhD, for his novel genome engineering work; Ryan Spencer, PhD, for his peptide engineering breakthroughs; Matthew Dobbin, PhD’s for his novel computational imaging pipeline; and Kola Awe for his computational robotics and nanomaterials design pipeline. The company also thanks additional team members involved in the project.

Overall, Ligandal is developing a complete platform to unlock the human genome to tackle multiple complex diseases to free humanity from the pain and suffering caused by each disease.

Based in San Francisco, Ligandal is a nanomedicine company developing unique curative genetic therapies with a personalized nanotechnology platform. Visit to learn more.

Contact: Vinh Vuong
+1 814-357-5445