Ricardo Dolmetsch, PhD, President of Research and Development at uniQure, discusses what he thinks physicians should know about gene therapy.
Huntington disease is an inherited, neurodegenerative disorder that usually begins to be symptomatic starting at around 40 years of age. Persons with Huntington disease suffer from a progressive movement disorder, cognitive decline, psychiatric disturbances, and behavioral symptoms. All symptoms progress which eventually leads to immobility, dementia, and premature death.
Recently, Uniqure announced positive safety and biomarker data from 10 patients enrolled in the low-dose cohort of the ongoing phase 1/2 clinical trial of AMT-130, the first-ever AAV gene therapy for Huntington’s disease.
In light of this positive data from the gene therapy trial, Dr. Dolmetsch describes what he thinks doctors should know about gene therapy. The first concept is that many doctors do not realize gene therapy is often a “one and done” therapy that has the potential to dramatical change the health care system. The second concept Dr. Dolmetsch thinks more physicians ought to understand is that many gene therapies have very positive safety profiles, even if as a group, gene therapies are often designated as unsafe.
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