uniQure Announces Dosing of First Patient in Phase I/II Clinical Trial of AMT-162 for the Treatment SOD1-ALS
“We are pleased to announce the first patient dosing of AMT-162, our investigational gene therapy for the treatment of SOD1-ALS, a debilitating, degenerative and fatal disease,” stated
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
The EPISOD1 Phase I/II clinical trial of AMT-162 will be conducted in
About Amyotrophic Lateral Sclerosis (ALS) caused by mutations in Superoxide Dismutase 1 (SOD1)SOD1-ALS is a rare, progressive and fatal neurodegenerative disease that results in the loss of motor neurons in the brain and spinal cord. As ALS progresses, individuals experience muscle weakness and atrophy causing them to lose the ability to move their arms and legs, speak, swallow and eventually breathe, causing respiratory failure. Based on a 2021 study in Neuroepidemiology, there are an estimated ~170,000 individuals with ALS globally with SOD1 mutations representing 2% within that population. According to the
About uniQure
uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. The approvals of uniQure’s gene therapy for hemophilia B – an historic achievement based on more than a decade of research and clinical development – represent a major milestone in the field of genomic medicine and ushers in a new treatment approach for patients living with hemophilia. uniQure is now advancing apipelineof proprietary gene therapies for the treatment of patients with
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