On May 20th, 2025, individuals with Usher syndrome who are in the USH Trust contact database were invited to join a webinar on Sepul Bio's Phase 2b clinical study called LUNA. The LUNA trial is testing the safety and effectiveness of ultevursen in maintaining vision in individuals with Usher syndrome caused by variants in exon 13 of the Usher syndrome type 2A gene. Below, you will find videos with captions, with Spanish translations, with ASL and British Sign Language translations, text transcripts in multiple languages, and a downloadable pdf of a slide presentation with related content.
The Usher Syndrome Coalition is working closely with Sepul Bio to inform the Usher community and accelerate this research.
English with English captions
Spanish translation with Spanish captions
Ameican Sign Language with English captions and Spoken English
British Sign Language with English captions and Spoken English
Download slides related to this content
Webinar transcripts in multiple languages:
English
Catalan (Catalan)
German (Deutsch)
Español (Spanish)
Français (French)
Italiano (Italian)
한국인 (Korean)
Nederlands (Dutch)
Português (Portuguese)
Русский (Russian)
Background information
The Coalition is pleased to partner with Sepul Bio, an innovative business unit of Laboratories Théa (“Théa”) dedicated to the advancement of mRNA therapies for inherited retinal diseases.
This partnership will work together to help recruit 81 participants worldwide for an exciting trial called LUNA, aimed at stopping vision loss related to retinitis pigmentosa caused by a specific mutation on the USH2A gene. Usher syndrome is the most common genetic cause of combined deafness and blindness. It is a rare condition affecting individuals around the world. USH2A is the most common gene causing Usher syndrome.
The LUNA clinical study was launched in December 2024 to determine whether the investigational RNA therapy, ultevursen, is effective at stopping the progression of vision loss in individuals diagnosed with retinitis pigmentosa due to variants in exon 13 of the USH2A gene. The Phase 2b clinical study will have two groups - one which will receive ultevursen and another which will not receive the treatment (“sham” group). The vision of participants in each group will be measured over two years to determine if ultevursen significantly slowed or stopped vision loss compared with those who did not receive ultevursen. If, after the two years, ultevursen is shown to be effective, all study participants will be eligible to receive treatment. LUNA's goal is to enroll 81 adults and children, making it the only USH2A trial in the world studying both adult and pediatric populations. For more information, please read the latest news here:
Learn more:
Sepul Bio: Transformative RNA therapies for inherited retinal diseases - Théa
Contact information:
Andy Bolan, Director, Patient Advocacy
contact@sepulbio.com
or
Usher Syndrome Coalition
info@usher-syndrome.org
