ProQR (PRQR) Announces Publication in Nature Medicine for Sepofarsen in Leber Congenital Amaurosis 10

ProQR Therapeutics N.V. (Nasdaq: PRQR), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today announced the publication in the scientific journal Nature Medicine titled “Durable vision improvement after a single treatment with antisense oligonucleotide sepofarsen: a case report” describing durable vision improvement after treatment with sepofarsen in a clinical trial for CEP290 mediated Leber congenital amaurosis 10 (LCA10).

“We are excited to share these findings with the scientific and medical community,” said lead author Professor Artur V. Cideciyan, Ph.D., one of the co-investigators at the Scheie Eye Institute of the University of Pennsylvania. “Treatment with sepofarsen resulted in substantial, concordant and enduring improvement in more than a dozen different subjective and objective measurements of visual function as well as retinal structure.”

“We are highly encouraged by the data showing sepofarsen had such a beneficial effect on this child’s vision,” said Aniz Girach, MD, Chief Medical Officer of ProQR, “The visual field improvements observed indicate that RNA therapy could potentially be used to treat early stage disease, which is enabled by the broad distribution of intravitreal RNA therapy throughout the retina.”

Published results highlight a patient who is homozygous for the c.2991+1655A>G mutation in CEP290 and was part of a larger cohort in the Phase 1/2 clinical trial. The patient was studied for 15 months after intravitreal treatment with sepofarsen. Concordant measures of visual function and retinal structure including visual acuity, light sensitivity and visual fields, reached a substantial efficacy peak near three months after injection and remained better than baseline at 15 months.

You May Also Be Interested In

• Acrivon Therapeutics (ACRV) Reports Initial Positive Clinical Data for ACR-368 and Pipeline Program Progress Today at Corporate R&D Event
• Petros Pharmaceuticals (PTPI) Expands Online Distribution for Prescription STENDRA
• Edgewise Therapeutics Inc. (EWTX) Granted EU Orphan Drug Designations for Sevasemten