Sepul Bio, an innovative business unit of Théa, completed enrollment in the Phase 2b LUNA Clinical Trial of Ultevursen for USH2A-associated retinitis pigmentosa
Clermont-Ferrand, France, Thursday 16 Jul 2026 - Sepul Bio, an innovative business unit of Laboratories Théa, is dedicated to the advancement of therapies for the treatments of inherited retinal diseases and is at the forefront of advancing transformative RNA therapies. Sepul Bio today announced the enrollment of the last participant in the Phase 2b LUNA clinical trial for Ultevursen, in patients with Usher Syndrome Type 2A (USH2A) associated retinitis pigmentosa (RP) or non-syndromic RP.
“Achieving the last enrolled participant in the Phase 2b LUNA clinical trial represents a fantastic milestone for USH2A research," said Dr. Robert Koenekoop, MD, PhD. Professor, Department of Pediatric Surgery, Faculty of Medicine and Health Sciences, McGill University. “This moment takes us one step closer in our search for therapies for this condition of high unmet need. The LUNA clinical trial, being conducted across 2C active clinical sites in 10 countries, is exploring the impact of RNA therapies on individuals with USH2A-associated RP due to variants in the exon 13 of the USH2A gene.”
“We are thrilled to see full enrollment of the LUNA clinical trial," said Krista Vasi, MPA, Executive Director of the Usher Syndrome Coalition. "The Usher Syndrome Coalition has supported the LUNA trial from protocol review through recruitment, connecting eligible families through the USH Trust, the largest international contact database of individuals with Usher syndrome, and amplifying this trial for families living with an USH2A exon 13 variant. This milestone brings our partnership full circle as we now enter the waiting period for data at the end of this trial. Each enrolled trial is a meaningful step forward toward treatments for our community, and one day, therapies that can change the lives of children, adults, and families living with Usher syndrome type 2A."
“There are currently no treatments for inherited retinal diseases like Usher Syndrome,” said Professor Katarina Stingl, Head of the Clinic for Hereditary Retinal Degeneration, Center for Rare Eye Diseases, University Hospital Tübingen. “I am delighted the LUNA clinical trial has now fully enrolled, as it marks a moment in Ultevursen’s clinical development where we can look forward to emerging data and real results for the USH2A community. I look forward to working with the Sepul Bio team to hopefully bring forward a treatment which can slow the disease progression or even reverse it in families affected by USH2A-associated RP.”
Retinitis pigmentosa (RP) is caused by one or more variants (“defects") in a gene in the DNA, which is then copied to the RNA. This mutation disrupts production of an important protein in the retina, called usherin, which is believed to cause photoreceptor death and thus vision loss. Ultevursen, an investigational RNA therapy, will be evaluated for safety and efficacy in this clinical trial. Ultevursen is designed to overcome this genetic defect and to restore this important protein, which may slow or stop some effects of RP.
Ultevursen is designed to target specifically mutations in exon 13 of the USH2A gene. Hybridization of Ultevursen to the pre-messenger ribonucleic acid (pre-mRNA) modulates the ribonucleic acid (RNA) splicing process, which leads to the exclusion of exon 13 from the mRNA. The exclusion of exon 13 results in the production of a functional (shorter) usherin protein. It is hypothesized that the production of usherin protein in the retina can potentially prevent or slow the progression of photoreceptor degradation and, as such, preserve visual function in patients.
About the Phase 2b LUNA study
LUNA, or SB-421a-006, is a two-year double-masked, randomized, sham-controlled trial of Ultevursen for adults and children (over eight years of age globally, and 12 years of age in Europe) who have retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.
Available safety and efficacy data from previous Ultevursen clinical studies support its therapeutic potential.
About Sepul Bio
As an innovative business unit of Théa, Sepul Bio is at the forefront of advancing transformative RNA therapies for inherited retinal diseases, with a particular emphasis on the further development of two cutting-edge ophthalmic products, Sepofarsen and Ultevursen.
Sepofarsen targets Leber congenital amaurosis 10 (LCA10), a rare genetic ailment causing severe visual impairment, while Ultevursen is designed to halt vision loss in individuals with a mutation in exon 13 of the USH2A gene suffering from retinitis pigmentosa.
Our programs are driven by the vision of a future where patients with inherited eye diseases have treatment options for their eye condition. Through ongoing research and rigorous development, Sepul Bio hopes to bring new therapies to patients. www.sepulbio.com
About Théa
Théa is the leading independent European pharmaceutical company specialized in the research, development, and commercialization of eye care products. Based in Clermont Ferrand, France, this family-owned and run company comprises about 2400 collaborators and has expanded by opening more than 35 affiliates and offices in Europe, North Africa, North and South America, and the Middle East. Its products are available in 75 countries. www.thea.com
Contact
Andrew Bolan
Patient Advocacy Director - Sepul Bio andrew.bolan@sepulbio.com