Sepul Bio, an innovative business unit of Théa, Doses First Participant in Phase 3 HYPERIONclinical trial of sepofarsen for CEP290-associated Leber Congenital Amaurosis Type 10

Sepul Bio: Théa’s Innovative Unit Doses First in Phase 3 HYPERION Trial for LCA10 Clermont-Ferrand, France, Wednesday 1 October 2025 - Sepul Bio, an innovative business unit of Théa, dedicated to the advancement of RNA therapies for inherited retinal diseases, has today announced the dosing of its first participants in the Phase 3 HYPERION clinical trial for sepofarsen, in individuals with CEP290- associated Leber Congenital Amaurosis Type 10 (LCA10). “ There are currently no effective treatments for inherited retinal diseases like CEP290-associated LCA10, ” says Dr. Artur Cideciyan , PhD, Research Professor of Ophthalmology and Co-Director, Center for Hereditary Retinal Degenerations at the Scheie Eye Institute of the University of Pennsylvania. “ Together with Dr. Tomas Aleman, MD, we have studied this childhood blindness for two decades. We are now excited to participate in the HYPERION phase 3 clinical trial and dose one of the first participants. The HYPERION study is evaluating the potential positive impact of sepofarsen, an RNA therapy, on the vision of individuals with LCA10. We know that the c.2991+1655A>G (p.Cys998X) variant in the CEP290 gene stops retinal cells from producing an essential protein needed for vision. By addressing this variant specifically, sepofarsen aims to restore cell function in the retina, offering the LCA10 community potentially life-changing therapies .” LCA is a rare genetic eye disorder that primarily affects the retina, which is the lightsensitive tissue at the back of the eye. It is a congenital condition, meaning it is present from birth, and causes severe vision impairment or blindness. It results from genetic mutations that disrupt the function of the retina’s photoreceptor cells. Common symptoms, such as loss of acuity, nystagmus (involuntary eye movements), nyctalopia and impaired peripheral vision, typically emerge in infancy. LCA encompasses various subtypes, each linked to specific genetic mutations and different levels of visual impairment. “ The launch of the HYPERION study is an exciting milestone for the LCA community,” said Courtney Coates, Executive Director, Hope in Focus. “We have been eagerly waiting for the re-initiation of the sepofarsen program, and today’s announcement reflects renewed hope for the community. As a community partner, we look forward to working alongside the Sepul Bio team as they advance RNA therapies to potentially help children, adults, and families affected by CEP290- associated LCA10. ” “CEP290- associated LCA10 is a devastating retinal disease resulting in blindness, for which there are no therapies,” said Dr. Bart Leroy, Head of the Ophthalmology Department at Ghent University Hospital and Professor of Ophthalmology and Ophthalmic Genetics at Ghent University, Ghent, Belgium. “ We are proud to continue to bring forward sepofarsen as a medicine through the new HYPERION clinical trial. We are delighted to be part of the first wave of clinical sites to dose participants. By directly targeting this defect, sepofarsen is designed to restore retinal cell activity and bring the possibility of transformative therapies to the LCA10 community .” About the Phase 3 HYPERION study HYPERION, or SB-110-007, is a two-year double-masked, randomized, placebo-controlled, paired-eye study to evaluate the efficacy, safety and tolerability of sepofarsen in 32 participants with LCA10 due to the c.2991+1655A>G (p.Cys998X) mutation in the CEP290 gene. About Sepul Bio As a dedicated 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 LCA10, a rare genetic ailment causing severe visual impairment, while ultevursen is designed to slow vision loss in individuals with a mutation in exon 13 of the USH2A gene suffering from retinitis pigmentosa. 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 ClermontFerrand, France, this family-owned and run company comprises more than 2000 collaborators and has expanded by opening 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 Published on 10/1/25 Share : {"@context":"https:\/\/schema.org","@type":"Article","url":"\/en_WW\/g\/news\/sepul-bio-an-innovative-business-unit-of-thea-doses-first-participant-in-phase-3-hyperionclinical-trial-of-sepofarsen-for-cep290-associated-leber-congenital-amaurosis-type-10","publisher":{"@type":"Organization","name":"Laboratory Th\u00e9a","logo":"\/static\/img\/Logo-Thea.svg"},"headline":"Sepul Bio, an innovative business unit of Th\u00e9a, Doses First Participant in Phase 3 HYPERIONclinical trial of sepofarsen for CEP290-associated Leber Congenital Amaurosis Type 10","mainEntityOfPage":"\/en_WW\/g\/news\/sepul-bio-an-innovative-business-unit-of-thea-doses-first-participant-in-phase-3-hyperionclinical-trial-of-sepofarsen-for-cep290-associated-leber-congenital-amaurosis-type-10","image":"\/uploads\/medias\/sepul-x-thea-68dd411b44626066946805.png","datePublished":"October 1, 2025 17:03","author":[{"@type":"Organization","name":"Laboratory Th\u00e9a","url":"https:\/\/www.laboratoires-thea.com\/"}],"description":"Sepul Bio: Th\u00e9a\u2019s Innovative Unit Doses First in Phase 3 HYPERION Trial for LCA10","category":"Th\u00e9a"} Other news Théa Théa welcomes G5 Santé in Clermont-Ferrand to discuss the future of the healthcare industry Théa welcomed G5 Santé in Clermont-Ferrand for discussions on the competitiveness, innovation and strategic sovereignty of the healthcare industry in France and Europe. 9/11/26 Théa Théa and Unither launch new ophthalmic production facility Théa and Unither Pharmaceuticals announce a strategic long-term industrial partnership to develop a new ophthalmic production facility in Gannat, France. 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