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.
Enabling Technologies
New technologies to advance gene therapy delivery, efficacy and safety.
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.
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.
Our technology platform enables our pipeline
| CNS Diseases | Smart AAV | miQURE | LinQURE | GoQURE |
|---|---|---|---|---|
| Huntington’s Disease | ||||
| Temporal Lobe Epilepsy | ||||
| ALS | ||||
| Alzheimer’s Disease |