Delivery, Use and Therapeutic Applications of the CRISPR-Cas Systems and Compositions for Genome Editing Getting Gene-Editing Tools Safely Into the Right Cells

This patent concerns the delivery and therapeutic use of CRISPR-Cas systems, the gene-editing tools that let scientists make precise changes to DNA. The focus is less on the molecular scissors themselves and more on how to package, deliver and optimise them so they reach the intended cells and edit the genome accurately and safely.

The Problem

Genome editing with CRISPR has transformed biology, but turning it into a therapy depends on a hard practical question: how do you get the editing machinery into the right tissue inside a living body, in the right amount, without unwanted effects? Editing tools that work beautifully in a dish can struggle in a patient, where delivery, specificity and toxicity all become concerns. Off-target edits, weak delivery to the target organ, and immune or toxic responses are all obstacles between a promising laboratory result and a usable treatment.

What This Invention Does

The patent provides delivery systems and methods for getting CRISPR components into cells and tissues, together with vectors and vector systems that encode one or more parts of a CRISPR complex. It covers how to design and use these vectors, how to target particular tissues or organs as sites for delivery, and how to direct the formation of the CRISPR complex inside cells so that target recognition is more specific and toxicity is avoided. The overall aim is to edit or modify a chosen site in the genome in order to alter or improve a disease or condition. By claiming the delivery and optimisation layer, the patent addresses the part of the gene-editing problem that sits between the tool and the patient.

Key Features

• Targeted delivery. Methods describe directing the editing components to specific tissues or organs.
• Vector systems. Viral vectors and other carriers encode the CRISPR components for delivery into cells.
• Enhanced specificity. The approach guides CRISPR complex formation to improve accurate target recognition.
• Toxicity avoidance. Optimisation is aimed at reducing unwanted effects in the edited cells.
• Therapeutic orientation. The end goal is editing a genomic site to treat or improve a disease or condition.

Who Is Behind It

The applicants are The Broad Institute, Inc. and the Massachusetts Institute of Technology, two of the foremost research organisations in genome editing. The named inventors include Feng Zhang, a central figure in the development of CRISPR-based tools, along with Le Cong, David Benjamin Turitz Cox, Matthias Heidenreich, Randall Jeffrey Platt and Lukasz Swiech. This Australian application is a divisional of an earlier filing and has been withdrawn.

Why It Matters

CRISPR-based therapies are moving from research into approved treatments, and the intellectual property around how editing tools are delivered and optimised is as commercially important as the editing chemistry itself. Tracking the status of foundational filings like this one matters to anyone following the gene-editing landscape in Australia, including the fact that this particular application has been withdrawn.

Related Concepts

• CRISPR gene editing – the core technology this patent helps deliver.
• Cas9 – a key enzyme used to cut DNA at a chosen site.
• Viral vector – a common vehicle for delivering editing components into cells.
• Gene therapy – the broader field of treating disease by changing genetic material.
• Genome editing – the practice of making precise changes to DNA.

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AU 2026201901 was published in the Australian Official Journal of Patents on 2 April 2026 and was subsequently withdrawn. Patent applications represent inventions that are sought to be protected and do not necessarily reflect commercially available products.