Test Subject Participates in Phase 2b Trial of Ultevursen, a Sepul Bio product, for USH2A-related Retinitis Pigmentosa in Collaboration with Laboratoires Théa
Leading European Eye Care Company Begins Clinical Trial for Rare Eye Disease
In a significant development for the treatment of a rare genetic eye condition, Sepul Bio, a business unit of Laboratoires Théa, has announced that the first participant has been dosed in the LUNA clinical study. This trial is assessing the safety and efficacy of ultevursen, an investigational RNA therapy, in individuals with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.
Laboratoires Théa, the leading independent European pharmaceutical company specializing in eye care products, is spearheading this initiative. Based in Clermont-Ferrand, France, the company employs approximately 2000 collaborators and has expanded its reach by opening more than 35 affiliates and offices across Europe, North Africa, North and South America, and the Middle East. Their products are available in 75 countries, and more information about the company can be found on their website at https://www.laboratoires-thea.com/en.
Usher Syndrome type 2a, a condition that affects the retina and the inner ear, is the most common cause of autosomal recessive RP and syndromic RP. There is currently no treatment for patients with these mutations. RP, a clinically and genetically heterogeneous condition with over 100 genes implicated, can lead to vision loss and blindness.
In this context, Sepul Bio, as a dedicated business unit of Théa, is focused on 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. Ultevursen is an antisense oligonucleotide product designed to target mutations in exon 13 of the USH2A gene, while sepofarsen targets Leber congenital amaurosis 10 (LCA10).
The LUNA clinical study, also known as SB-421a-006, is a two-year double-masked, randomized, sham-controlled study. It 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 trial's aim is to evaluate the safety and efficacy of ultevursen in slowing down the progression of RP in these patients.
While the available search results do not specify which organization coordinated the initiation of the LUNA study for treating RP caused by mutations in exon 13 of the USH2A gene, this trial marks a significant step forward in the quest for a treatment for Usher Syndrome type 2a and related retinal diseases. The results of this study could potentially provide hope for the millions of people worldwide affected by these conditions.
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