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Dive Brief:
UniQure can begin treating patients with the highest dose of its experimental Huntington s disease gene therapy, announcing Thursday that clinical trial monitors found no safety concerns after reviewing data from the first 10 participants who received lower doses.
The safety panel s go-ahead clears the way for the Massachusetts-based company to enroll another 16 patients. The trial is primarily assessing the safety of the gene therapy, called AMT-130, but will also measure biomarkers, cognition and motor function.
Huntington s disease drug development has seen two major setbacks, with Roche halting dosing of partner Ionis Pharmaceutical s drug tominersen and Wave Life Sciences canceling two separate programs. UniQure may have more signs of how well AMT-130 works later this year when brain scans and other biological tests of the first patients treated in the trial will be available.
Operator
Ladies and gentlemen, thank you for standing by, and welcome to the Solid Biosciences update call. [Operator instructions] Please be advised that today s conference is being recorded. I would now like to hand the conference over to your speaker today, Mr. Tim Palmer, corporate communications manager at Solid Biosciences.
Sir, you may begin.
Corporate Communications Manager
Good morning. Thank you, operator. Before we get started, I would like to remind everyone that during this conference call, we may make forward-looking statements, including statements about the company s financial results, financial guidance, future business strategies and operations and product development and regulatory progress, including statements about the ongoing IGNITE DMD clinical trial. Actual results could differ materially from those discussed in these forward-looking statements due to a number of important factors, including uncertainty inherent in the clinical development and regulatory
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