Sepul Bio begins clinical trial of ultevursen for USH2A-associated RP

The new clinical trial, known as LUNA, is a two year randomized sham-controlled study that will enroll 81 adults and children over the age of eight years across sites around the globe.

Ultevursen was originally developed by biotechnology company ProQR and proceeded through the early stages of clinical testing with encouraging results. It was then acquired by Laboratoires Théa, of which Sepul Bio is an innovation-focused subsidiary.

Ultevursen is a type of RNA-based therapy called an antisense oligonucleotide (AON). It precisely targets a particular disease-causing sequence of DNA in a section of the USH2A gene called exon 13, in effect putting a patch over the faulty genetic sequence and allowing the retinal cell to produce functional protein. Ultevursen is injected into the jelly of the eyeball.

Mutations in the USH2A gene can cause Usher syndrome type 2, where RP occurs with hearing loss, or non-syndromic RP with no hearing involvement. Ultevursen can potentially address sight loss in either case, but will only work for those individuals whose condition is caused by mutations in exon 13 of the gene. This can be confirmed by a genetic test report.

Sepul Bio will share UK trial site details with Retina UK as soon as possible. In the meantime, you can address questions directly to Sepul Bio via contact@sepulbio.com . If you need clarification of your genetic test result and whether it indicates an USH2A exon 13 mutation, please contact your ophthalmologist, clinical geneticist or genetic counsellor.