ProQR to Present at the Retinal Cell and Gene Therapy Innovation Summit and ARVO 2022
ProQR Therapeutics announced several presentations at the 7th Annual Retinal Cell and Gene Therapy Innovation Summit being held Friday, April 29, 2022 and the Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) being held May 1-4, 2022, both in Denver, CO, U.S.
Presentations at the Retinal Cell and Gene Therapy Innovation Summit
Presentation title: AON treatment for CEP290-LCA: Efficacy, safety, and durability
Presenter: Dr. Artur Cideciyan, University of Pennsylvania
Presentation type: Pre-recorded talk
Session: Session 4: Antisense Oligonucleotide Therapy
Date: April 29, 2022 at 4:00pm MDT
Presentation title: Efficacy and safety of sepofarsen, an intravitreal RNA antisense oligonucleotide, for the treatment of CEP290-associated inherited retinal disease called Leber congenital amaurosis (LCA10): A randomized, double-masked, sham-controlled, phase III study (ILLUMINATE)
Presenter: Dr. Bart Leroy, Ghent University Hospital
Presentation type: Oral presentation
Session: Session 4: Antisense Oligonucleotide Therapy
Date: April 29, 2022 at 4:15pm MDT
Presentations at ARVO
Presentation title: Long-term safety and efficacy of sepofarsen in a Ph1b/2 INSIGHT extension trial in CEP290-associated Leber congenital amaurosis (LCA10)
Presenter: Dr. Stephen R. Russell, Iowa University
Presentation type: Oral presentation
Date: May 1, 2022 at 3:36pm MDT
Presentation title: QR-1011 corrects splicing in the Stargardt disease type 1-causing variant ABCA4 c.5461-10T>C
Presenter: Melita Kaltak, ProQR Therapeutics
Presentation type: Oral presentation
Date: May 4, 2022 at 10:34am MDT
Presentation title: Efficacy and safety of sepofarsen, an intravitreal RNA antisense oligonucleotide, for the treatment of CEP290-associated Leber congenital amaurosis (LCA10): a randomized, double-masked, sham-controlled, Phase 3 study (ILLUMINATE)
Presenter: Dr. Bart Leroy, Ghent University Hospital
Presentation type: Poster presentation
Date: May 4, 2022 at 3:00pm MDT
“CEP290-mediated Leber congenital amaurosis 10, or LCA10, is a difficult to treat ultra-rare inherited retinal disease for which there is currently no treatment,” said Dr. Bart Leroy, Head of the Ophthalmology Department and Professor of Ophthalmology and Ophthalmic Genetics at Ghent University in Belgium and Attending Physician at The Children's Hospital of Philadelphia. “After missing the primary endpoint in the Illuminate Phase 2/3 clinical trial, sepofarsen showed an encouraging efficacy signal across multiple endpoints in post-hoc analyses comparing sepofarsen treated and sham treated eyes to their corresponding untreated contralateral eyes. I look forward to continuing to work with ProQR on this investigational treatment.”