09.30.20

ProQR Announces Virtual Presentations at Euretina and AAOpt

Source: ProQR Therapeutics

ProQR Therapeutics announced virtual presentations at the Ophthalmology Futures Retina Forum, European Society of Retina Specialists (Euretina) congress and the Annual Meeting of the American Academy of Optometry (AAOpt).

ProQR’s presentation at Ophthalmology Futures Retina Forum

Presentation title: Advancements in Inherited Retinal Diseases Management

Presenter: Aniz Girach, MD, Chief Medical Officer of ProQR

Date: September 30, 2020

ProQR’s presentations at Euretina

Presentation title: Phase 1b/2 trial results of intravitreal sepofarsen RNA therapy in Leber congenital amaurosis 10 (LCA10)

Presenter: Stephen R. Russell, MD, principal investigator of the ProQR Phase 1/2 clinical trial of sepofarsen Professor and Director of Vitreoretinal Diseases and Surgery Service, Department of Ophthalmology and Visual Sciences, University of Iowa

Date: The poster presentation will be available on the Euretina Virtual Congress portal starting October 2, 2020.

Presentation title: RNA antisense oligonucleotide sepofarsen in Leber Congenital Amaurosis type 10 (LCA10): disease severity and patient response based on genotype

Presenter: Bart P. Leroy, MD, PhD, principal investigator of the ProQR Phase 1/2 clinical trial of sepofarsen and Chairman and Head of the Department of Ophthalmology at the Center for Medical Genetics at the Ghent University

Date: The poster presentation will be available on the Euretina Virtual Congress portal starting October 2, 2020.

ProQR’s presentation at AAOpt

Presentation title: Survey of Early Genetic Diagnosis of Inherited Retinal Diseases (IRDs) and Patient Referrals: The important role of Optometrists in the early management of blinding genetic diseases

Presenter: Ramiro S. Maldonado, MD, Assistant Professor of Ophthalmology at Shriners UK Advances Eye Care at the University of Kentucky

Date: The poster presentation will be available on the AAOpt Virtual Platform starting October 7, 2020. A live questions and answers session will be held on Friday October 9, 2020, 7:30-9:30 PM EDT.

About Sepofarsen

Sepofarsen (QR-110) is being evaluated in the pivotal phase 2/3 Illuminate trial and is a first-in-class investigational RNA therapy designed to address the underlying cause of Leber congenital amaurosis 10 due to the p.Cys998X mutation (also known as the c.2991+1655A>G mutation) in the CEP290 gene. The p.Cys998X mutation leads to aberrant splicing of the mRNA and non-functional CEP290 protein. Sepofarsen is designed to enable normal splicing, resulting in restoration of normal (wild type) CEP290 mRNA and subsequent production of functional CEP290 protein. Sepofarsen is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union and received fast-track designation and rare pediatric disease designation from the FDA as well as access to the PRIME scheme by the EMA.

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