VP-001

VP-001 is a first-in-class, disease-modifying RNA therapeutic developed for the treatment of retinitis pigmentosa type 11 (RP11), an inherited retinal dystrophy caused by mutations in the PRPF31 gene. The drug is a cell-penetrating peptide–phosphorodiamidate morpholino oligomer (CPP-PMO) conjugate, also described as a peptide-drug conjugate and antisense oligonucleotide. Its mechanism of action involves downregulating CCR4-NOT transcription complex subunit 3 (CNOT3) protein expression, which normally acts as a negative regulator of pre-mRNA processing factor 31 (PRPF31) transcription. By inhibiting CNOT3, VP-001 upregulates PRPF31 protein production from the healthy allele in patients with RP11, aiming to restore sufficient levels for normal cell function and halt or slow disease progression[1][2][6][8]. The therapy is administered via intravitreal injection and has shown promising safety and efficacy results in early clinical trials[5]. Developed using proprietary delivery technology to enhance cellular uptake into retinal cells—especially retinal pigment epithelium—VP-001 represents an innovative approach targeting the genetic root cause of RP11[6][8].