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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.
Lead Product(s): AMT-130
Therapeutic Area: Genetic Disease Product Name: AMT-130
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 19, 2023
Details:
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.
Lead Product(s): AMT-130
Therapeutic Area: Genetic Disease Product Name: AMT-130
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 19, 2023
Details:
AMT-191 is a alpha-GLA transgene Transference cell and gene therapy drug candidate, delivered via intravenous infusion for the treatment of Fabry disease.
Lead Product(s): AMT-191
Therapeutic Area: Genetic Disease Product Name: AMT-191
Highest Development Status: IND EnablingProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 29, 2023
Details:
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.
Lead Product(s): AMT-260
Therapeutic Area: Neurology Product Name: AMT-260
Highest Development Status: IND EnablingProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 05, 2023
Details:
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.
Lead Product(s): rAAV5-miHTT
Therapeutic Area: Genetic Disease Product Name: AMT-130
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 21, 2023
Details:
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.
Lead Product(s): Etranacogene Dezaparvovec-drlb
Therapeutic Area: Genetic Disease Product Name: Hemgenix
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: CSL Behring
Deal Size: $2,050.0 million Upfront Cash: $450.0 million
Deal Type: Licensing Agreement June 20, 2023
Details:
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.
Lead Product(s): Etranacogene Dezaparvovec-drbl
Therapeutic Area: Genetic Disease Product Name: Hemgenix
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: HealthCare Royalty
Deal Size: $400.0 million Upfront Cash: $400.0 million
Deal Type: Divestment May 15, 2023
Details:
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.
Lead Product(s): APB-102
Therapeutic Area: Neurology Product Name: APB-102
Highest Development Status: IND EnablingProduct Type: Cell and Gene therapy
Recipient: Apic Bio
Deal Size: $55.0 million Upfront Cash: $10.0 million
Deal Type: Licensing Agreement January 31, 2023
Details:
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.
Lead Product(s): Etranacogene Dezaparvovec-drlb
Therapeutic Area: Genetic Disease Product Name: Hemgenix
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 16, 2022
Details:
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.
Lead Product(s): AMT-260
Therapeutic Area: Neurology Product Name: AMT-260
Highest Development Status: IND EnablingProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 29, 2022
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