Sepul Bio Doses First Patient in Phase 2b Trial of Ultevursen for USH2A-Associated Retinitis Pigmentosa

Sepul Bio has announced the dosing of the first patient in the LUNA clinical study, a Phase 2b trial evaluating ultevursen for the treatment of USH2A-associated retinitis pigmentosa (RP). This milestone represents renewed hope for individuals affected by this debilitating genetic condition.

Understanding USH2A-Associated Retinitis Pigmentosa

USH2A (Usher Syndrome Type 2a) is the most common cause of autosomal recessive retinitis pigmentosa, a condition affecting the retina and inner ear.

Key Facts:

       • Mutations in exon 13 of the USH2A gene result in syndromic and non-syndromic forms of RP.

       • Symptoms include progressive vision loss, often accompanied by hearing impairment.

       • Currently, there are no approved treatments for these patients.

About Ultevursen

Ultevursen, developed by Sepul Bio, is an RNA therapy designed to specifically target mutations in exon 13 of the USH2A gene. By addressing the underlying genetic cause, ultevursen aims to preserve vision in patients with RP.

LUNA Clinical Trial Overview

The LUNA study is a two-year, double-masked, randomized, sham-controlled Phase 2b trial evaluating the safety and efficacy of ultevursen.

Key Details:

       • Participants: 81 adults and children aged 8 years and older.

       • Objective: Assess the potential of ultevursen to slow or halt the progression of vision loss in patients with USH2A-associated RP.

Community Support

Krista Vasi, MPA, Executive Director of the Usher Syndrome Coalition, highlighted the significance of the trial:

“The initiation of enrollment in the LUNA study is an exciting milestone for the Usher syndrome community. We have been eagerly waiting for the re-initiation of the ultevursen program, and today’s announcement reflects renewed hope for the community.”

The Usher Syndrome Coalition, a global organization supporting individuals with Usher syndrome, maintains the largest international contact database of affected individuals from 76 countries.

Sepul Bio’s Commitment

As a business unit of Laboratoires Théa, France-based Sepul Bio is dedicated to advancing RNA therapies for inherited retinal diseases. The launch of the LUNA study aligns with the company’s mission to address significant unmet medical needs in retinal health.

Conclusion

The dosing of the first patient in the LUNA study marks a significant step forward in the development of ultevursen as a potential treatment for USH2A-associated retinitis pigmentosa. With its focus on exon 13 mutations, Sepul Bio is bringing hope to patients and families affected by this challenging condition.