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Find Clinical Drug Pipeline Developments & Deals by Uniqure
AMT-130 is uniQure’s first clinical program focusing on the CNS incorporating its proprietary miQURE® platform. Itos under phase 1/2 clinical development for the treatment of Huntington’s Disease.
AMT-130 is an mHTT production inhibitor cell and gene therapy drug candidate, which is currently being evaluated for the treatment of Huntington’s disease.
AMT-191 is a alpha-GLA transgene Transference cell and gene therapy drug candidate, delivered via intravenous infusion for the treatment of Fabry disease.
AMT-260 is an AAV9 gene therapy product that locally delivers miRNA silencing technology to target the GRIK2 gene and suppress aberrantly expressed GluK2 containing kainate receptors, which is investigated for refractory mesial temporal lobe epilepsy.
AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging our proprietary miQURE™ silencing technology, which is investigated for Huntington’s Disease.
The licensing agreement provided CSL Behring with exclusive global rights to Hemgenix (etranacogene dezaparvovec), uniQure’s first approved gene therapy for hemophilia B in the United States, EU and European Economic Area, and the UK, for patients with hemophilia B.
The divestment aims to sell a portion of the royalty rights due to uniQure from CSL Behring from the net sales of Hemgenix (etranacogene dezaparvovec-drbl), the first and only FDA-approved gene therapy for hemophilia B, to HealthCare Royalty and Sagard Healthcare.
Under the agreement, uniQure acquires global rights for the development and commercialization of APB-102, adding to its pipeline of gene therapies to treat neurological disorders.
Hemgenix (Etranacogene dezaparvovec), is a one-time gene therapy for the treatment of appropriate adults with hemophilia B that reduces the rate of annual bleeds after a single infusion.
AMT-260 is an AAV gene therapy that uses an AAV9 vector and combines uniQure’s miQURE and linQURE technologies to deliver multiple miRNAs that reduce the expression of the GRIK2 gene.
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