uniQure NV is at the forefront of gene therapy, focusing on innovative treatments for genetic disorders, particularly those affecting the liver and central nervous system (CNS). Their proprietary platform utilizes adeno-associated virus (AAV) vectors, specifically the AAV5 serotype, to deliver therapeutic genes that can either restore the expression of missing proteins or silence disease-causing genes. This approach is exemplified in their development of AMT-130, a gene therapy targeting Huntington's disease, which aims to inhibit the production of the mutant huntingtin protein. The company’s technology is designed to provide long-lasting therapeutic effects from a single administration, with ongoing clinical trials demonstrating promising safety and efficacy profiles.

uniQure's modular technology platform allows for the efficient development and manufacturing of gene therapies for various diseases by utilizing a consistent set of components, such as capsids and promoters, while only needing to modify the disease-specific gene cassette. This innovative approach not only reduces the time and cost associated with developing new treatments but also enhances the potential for re-dosing and cross-administration of therapies. The company has received multiple designations from regulatory bodies, including the FDA's Regenerative Medicine Advanced Therapy (RMAT) designation for AMT-130, highlighting its commitment to addressing significant unmet medical needs in the field of gene therapy.

Netherlands

uniQure is delivering on the promise of gene therapy - single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with severe genetic diseases.

Central Nervous System (CNS)

Genetic Diseases

Hematologic Diseases

Uniqure NV

Hemophilia

Rare diseases

Rare genetic disease

Etranacogene dezaparvovec, also known as AMT-061, consists of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua). AAV5-based gene therapies have been demonstrated to be safe and well tolerated in many clinical trials, including four uniQure trials conducted in 25 patients in hemophilia B and other indications. No patient treated in clinical trials with the Company’s AAV5-based gene therapies has experienced any cytotoxic T-cell-mediated immune response to the capsid. Additionally, preclinical and clinical data show that AAV5-based gene therapies may be clinically effective in patients with pre-existing antibodies to AAV5, thereby potentially increasing patient eligibility for treatment compared to other gene therapy product candidates. Etranacogene dezaparvovec has been granted Breakthrough Therapy Designation by the United States Food and Drug Administration and access to Priority Medicines (PRIME) regulatory initiative by the European Medicines Agency.
Gene therapy

HEMGENIX® (etranacogene dezaparvovec-drlb) (AMT-061) 

Genetic disorder

Huntington's disease

Our gene therapy product candidate&nbsp;AMT-130&nbsp;consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging our proprietary miQURE™ silencing technology. The therapeutic goal is to inhibit the production of the mutant protein (mHTT). Using AAV vectors to deliver micro-RNAs directly to the brain for non-selective knockdown of the huntingtin gene represents a highly innovative and promising approach to treating Huntington’s disease.
Gene therapy

AMT-130 

Epilepsy

Mesial temporal lobe epilepsy

AMT-260

Fabry disease

AMT-191 

Amyotrophic lateral sclerosis

AMT-162 is an investigational AAVrh10-based gene therapy that expresses a miRNA designed to knock down the expression of the mutated SOD1 protein. Patients with SOD1-ALS express a misfolded SOD1 protein. This misfolded protein is toxic to motor neurons causing degeneration which, over time leads to muscle weakness, loss of function and eventually death. AMT-162 may offer a novel one-time, intrathecally administered approach to slowing or halting the progression of SOD1-ALS. AMT-162 has been granted both Orphan Drug status and Fast Track designation by the U.S. Food and Drug Administration.

Uniqure NV

Company Details