Enabling Technologies

Hero enabling technologies

New technologies to advance gene therapy delivery, efficacy and safety.

uniQure has long been a leader in developing technologies for advancing gene therapy. Our AAV technology engine, built with more than 20 years of experience, drives our gene therapies, and we remain focused on developing innovations designed to address some of the major challenges in AAV gene therapy.

New Capsids to Deliver Gene Therapies to the CNS and Liver

These challenges include how to deliver gene therapies– how to deliver them to the right cells, how to get enough cells transduced, how to dose through preexisting neutralizing antibodies, how to re-dose if patients don’t respond appropriately, and how to dose in a way that is non-invasive.

We are working on a set of technologies we call QUREDose® for administering multiple doses of a gene therapy. We have shown in large animal models the ability to administer a first dose of a gene therapy and then follow it by administering a second dose of the therapy. We believe this could improve gene therapy and enable a wide variety of new applications. This includes increasing the precision of our AAV dosing, re-treating patients with partial responses, and treating growing tissues in children and adults.

Our Smart AAV technology represents a new generation of capsids that improve the delivery of gene therapies to the central nervous system (CNS). These capsids use antibodies and peptides to target specific tissues and cells and to cross the cross the blood-brain barrier.

Transporting Genes to Transform Lives

We are focused on developing the next generation of cargos, the core of a gene therapy, for effective gene knockdown, correcting genes, and delivering biologics.

Our growing portfolio of innovative, leading-edge cargo technologies include the following:

miQURE® is our proprietary gene silencing platform. It is designed to degrade disease-causing genes, without overloading the cellular machinery and causing off-target toxicity.

LinQURE® is our technology to deliver multiple microRNAs in a single AAV, enabling more effective gene silencing and the simultaneous modulation of multiple signaling pathways in a cell.

GoQURE® is our technology for simultaneously knocking down a disease gene and replacing with a healthy copy. It combines our miQURE technology with the ability to safely and efficiently express a gene in the same AAV vector.

AbQURE® is our technology for delivering a therapeutic antibody, both systemically from the liver and into the CNS from brain cells. This could change the therapeutic landscape by allowing the possibility of a one-shot delivery of biologics for serious diseases.

Our technology platform enables our pipeline

Liver-Directed QUREDose Smart AAV miQURE LinQURE GoQURE AbQURE
Hemophilia B
Fabry Disease
CNS Diseases QUREDose Smart AAV miQURE LinQURE GoQURE AbQURE
Huntington’s Disease
Temporal Lobe Epilepsy
Parkinson’s Disease
ALS
Alzheimer’s Disease

Learn more about uniQure’s miQURE technology in the animation below:

Gene Therapy Manufacturing Expertise

uniQure is a leader in addressing the challenges of gene therapy manufacturing technologies. Our ambition is to develop new technologies that increase the robustness and reliability of manufacturing and decrease the cost of goods so we can bring our gene therapies to much larger populations.

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