Second gene therapy for Huntington’s disease

Huntington’s disease is an obvious target for gene therapies. Caused by long repeats in a single specific gene and the slow accumulation of its mutant protein, the disorder could be treated with any method capable of suppressing RNA sequences.

Team from UniQure company will try their method of micro-RNA targeting faulty intermediates between genes and proteins. The cure will be delivered with AAV5 (adeno-associated virus serotype 5).

Clinical trial (NCT04120493) recruits 26 patients with early-manifested Huntington’s disease. Gene therapy will consist of a single administration, followed by close monitoring of many outcomes. The primary goal of the trial is safety assessment. Treatment efficiency will be evaluated with a surrogate measure for Huntington’s disease severity – the level of mutant huntingtin protein in cerebrospinal fluid.

It should be noted that, contrary to prevailing media coverage, this is not the first gene therapy for Huntington’s disease. Between 2015 and 2019, clinical trial (NCT02519036) used interfering RNAs (antisense oligonucleotides) to successfully lower the level of mutant huntingtin, in multiple administrations and without the use of viral delivery.

Photo: Jens Maus.

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