Sepul Bio Announces First Patient Dosed in Trial Using Ultevursen for RP

Sepul Bio announced the first person has been dosed for the LUNA clinical study, a phase 2b trial using ultevursen for USH2A-associated retinitis pigmentosa (RP). 

USH2A, or Usher Syndrome type 2a, is the most common cause of autosomal recessive retinitis pigmentosa that affects the retina and inner ear. Mutations in exon 13 of the USH2A gene result in both syndromic and non-syndromic forms of retinitis pigmentosa. Currently, there is no treatment for these patients. 

“The initiation of enrollment in the LUNA study is an exciting milestone for the Usher syndrome community,” Krista Vasi, MPA, and Executive Director of the Usher Syndrome Coalition, said in a company press release. The Usher Syndrome Coalition houses the largest international contact database of individuals with Usher syndrome from 76 countries. “We have been eagerly waiting for the re-initiation of the ultevursen program, and today’s announcement reflects renewed hope for the community.” 

The mission of Sepul Bio is to advance RNA therapies for the treatment of inherited retinal disease. The France-based company, a business unit of Laboratoires Théa, developed ultevursen to specifically target the exon 13 mutations that cause vision loss in retinitis pigmentosa. The LUNA study is a two-year double-masked, randomized, sham-controlled study and will enroll 81 adults and children over 8 years of age.