Ultevursen, a treatment developed by Sepul Bio, a subsidiary of Laboratoires Théa, commences its Phase 2b clinical trial on a single participant for USH2A-related retinitis pigmentosa in the USA.
In a significant step forward for individuals affected by retinitis pigmentosa (RP), Sepul Bio, a business unit of Thea, a leading European pharmaceutical company specializing in eye care products, has announced the first clinical participant has been dosed in the LUNA clinical study.
The LUNA study is a Phase 2b study for ultevursen, an investigational RNA therapy. This study is a double-masked, randomized, sham-controlled study that will enroll 81 adults and children (over eight years of age) who have RP due to variants in exon 13 of the USH2A gene. The study's primary focus is on assessing the safety and efficacy of ultevursen in individuals with RP due to mutations in exon 13 of the USH2A gene.
Ultevursen is an antisense oligonucleotide product designed to target mutations in exon 13 of the USH2A gene. Mutations in this gene are the most common cause of autosomal recessive RP and syndromic RP, and there is currently no treatment for these patients. Exon 13 mutations in the USH2A gene targeted by ultevursen cause vision loss in approximately 16,000 individuals in the Western world.
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—ultevursen and sepofarsen. Sepofarsen is a separate antisense oligonucleotide product that targets Leber congenital amaurosis 10 (LCA10).
The initiation of enrollment in the LUNA study was supported by Activate, a fellowship program that helps scientists start their own companies. At Sepul Bio, Activate plays a role in the advancement of RNA therapies for inherited eye diseases by providing entrepreneurial training, mentorship, and connections to industry and funding, facilitating the development and progression of these therapies.
Retinitis pigmentosa (RP) is a clinically and genetically heterogeneous condition, and mutations in over 100 genes have been implicated. Thea, with its subsidiary Sepul Bio, is working diligently to address this vast spectrum of inherited retinal diseases.
Thea, based in Clermont-Ferrand, France, comprises about 2000 collaborators. Thea has expanded by opening more than 35 affiliates and offices in Europe, North Africa, North and South America, and the Middle East. Thea's products are available in 75 countries.
This clinical trial marks a significant milestone in the quest for a cure for RP, offering hope to thousands of individuals affected by this condition. The results of the LUNA study could potentially pave the way for the approval of the first therapy for the treatment of RP due to mutations in exon 13 of the USH2A gene.
