Neubase Enters Gene Editing Research Agreement with Global Healthcare Company

Precision medicine biotech NeuBase Therapeutics has entered a research agreement with an undisclosed top 10 global healthcare company that will evaluate NeuBase’s PATrOL technology for three monogenic genetic diseases.

Photo: Dietrich Stephan, CEO of NeuBase

The PATrOL platform is a peptide nucleic acid (PNA)-based technology designed to address disease at the root of causality to help patients with rare and common diseases by editing, upregulating, or downregulating gene function. PATrOL gene editing is differentiated in that it does not require bacterial enzymes (e.g., CRISPR-Cas9), which potentially increases fidelity and reduces immunogenicity to provide a safer solution to patients for in vivo gene editing.

“Our PATrOL technology enables us to create a series of peptide nucleic acids designed to target a genetic mutation and recruit the cell’s own high fidelity nucleic acid repair machinery to resolve the mutation,” said Dietrich Stephan, CEO of NeuBase.

Under the terms of the agreement, NeuBase and the healthcare company will collaborate on the evaluation of drug candidates for three undisclosed indications. The healthcare company will have the exclusive opportunity, subject to certain terms and conditions, to license and develop the drug candidates created under this research evaluation agreement.

Author: Rare Daily Staff