About CURE4/6

CURE4/6 is a nonprofit organization dedicated to advancing research and treatment development for retinitis pigmentosa caused by variants in RNU4-2 and RNU6 genes.

RNU4-2 and RNU6 are part of the RNA-splicing system, an essential process that allows cells to correctly process genetic instructions. Recent discoveries have shown that variants in these small nuclear RNA genes can cause inherited retinal degeneration, including retinitis pigmentosa. These discoveries have finally provided answers for families who had lived for years without a clear genetic diagnosis.

Our mission is to accelerate research, connect affected families, and support the development of treatments for these rare retinal disorders.

CURE4/6 is focused on several major goals:

• Building a secure patient registry to identify and connect individuals and families affected by RNU4-2 and RNU6 variants.
• Supporting research to understand how these variants affect retinal cells, including photoreceptors, retinal pigment epithelium, retinal organoids, and other disease models.
• Studying why some people with these variants develop severe retinal disease while others may have milder symptoms or incomplete penetrance.
• Helping classify additional genetic variants similar to the ones already discovered, including variants of uncertain significance.
• Encouraging development of treatments, including antisense oligonucleotides, gene replacement or gene-transfer approaches, drug repurposing, and other therapeutic strategies.
• Supporting preclinical studies in cell systems, retinal organoids, and animal models, with the long-term goal of advancing toward clinical trials.

CURE4/6 was created by families and physicians who believe that rare genetic retinal diseases deserve focused attention, organized research, and a clear path toward treatment. By bringing together patients, families, scientists, clinicians, geneticists, and research partners, we aim to build the foundation needed to move from diagnosis to discovery — and from discovery to therapy.

For affected families, CURE4/6 offers a place to be counted, connected, and heard. For researchers, it provides a way to understand the disease more deeply and identify the patients who may benefit from future studies. For donors and supporters, it offers an opportunity to help transform a newly discovered diagnosis into a treatable condition.