Sepofarsen news update Data from InSight, an extension trial for Leber congenital amaurosis 10

During one of ProQR’s Expert Perspectives calls ProQR’s Chief Medical Officer Dr. Aniz Girach reviewed new data from an ongoing clinical trial of sepofarsen with Dr Ian MacDonald, Professor Emeritus from the University of Alberta.

On the left-hand sight a face picture of Dr. Ian MacDonald and on the right-hand side a face picture of Dr. Aniz Girach.

The InSight study

The InSight study is an open label extension study for people that participated in the Phase 1/2 clinical trial of sepofarsen, an investigational RNA therapy for Leber congenital amaurosis 10 (LCA10). In the study these participants were given the opportunity to continue treatment with sepofarsen. During the call on July 20, 2020, Dr. Girach presented preliminary findings from the InSight study, which is ongoing.

Improvements in vision

During the initial Phase 1/2 clinical trial the majority of eleven participants experienced rapid, significant, and durable improvements in vision in the treated eye. Their other eye remained untreated as a control. Read this press release with details about the Phase 1/2 trial results. Nine participants from the Phase 1/2 trial joined the InSight extension study, of which four received treatment in the second eye. After treating the second eye, a consistent response was observed.

The expert’s perspective

During the call IRD expert Dr. Ian MacDonald, Professor Emeritus in the Department of Ophthalmology and Visual Sciences, University of Alberta provides his interpretation of the data and how he thinks these improvements can help patients in their daily life.

ProQR Expert Perspective call

Sepofarsen - RNA therapy for CEP290 mediated Leber congenital amaurosis

Interested in more perspectives from experts? Watch the full video series.