The Usher Syndrome Coalition announces partnership with Sepul Bio to support clinical trial enrollment for a potential therapy for USH2A-mediated retinitis pigmentosa
Last week, we announced the exciting news that Sepul Bio, an innovative business unit of Théa, successfully dosed the first participant in the LUNA clinical trial. LUNA is a clinical study of ultevursen, a potential therapy to stop the progression of vision loss in individuals with Usher syndrome type 2A due to mutations in exon 13 of the USH2A gene.
Today, we are thrilled to announce that the Usher Syndrome Coalition and Sepul Bio have entered a formal partnership to support this research.
The success of clinical trials relies on having a robust community of potential participants. Without this community, clinical trials will end prematurely. That is why, for years, the Usher Syndrome Coalition has focused its efforts on building the USH Trust, our most powerful tool to connect and support the Usher community worldwide. This new partnership clearly illustrates that Sepul Bio and Théa also acknowledge the necessary involvement of a patient advocacy community, particularly with a rare disease like Usher syndrome. This milestone demonstrates Théa’s commitment and enthusiasm to partner with the Coalition as the center of the global Usher syndrome community.
“At the Coalition, we understand that community participation drives the success of clinical trials, and community is what we’re all about,” said Krista Vasi, MPA, Executive Director. “This partnership underscores the vital role that the Coalition will play in accelerating enrollment in this clinical trial and connecting eligible participants with study sites. Through the Coalition’s USH Trust, we have contact with individuals with Usher syndrome in 78 countries.
Our reach further extends through our team of 80 USH Ambassadors worldwide, who serve as the points of contact for the Usher community in their state or country. These valuable resources strategically position the Coalition as a key community partner of Sepul Bio. We look forward to working alongside the Sepul Bio team as they advance therapies to potentially help children and adults who are living with specific mutations in the USH2A gene.”
LUNA is a two-year study testing ultevursen, an experimental mRNA therapy aimed to help people with Usher syndrome type 2A maintain their vision. The LUNA study will enroll 81 participants, both adults and children over eight years old, who have retinitis pigmentosa caused by genetic changes, or variants, in exon 13 of the USH2A gene. The first active clinical site is in the United States. Sepul Bio plans to open more sites in the coming months, sharing updates as they are confirmed.
Andrew Bolan, Patient Advocacy Director, is a key member of Sepul Bio who transitioned from ProQR. He continues to serve as a vital link in this work. Mr. Bolan will share updates with USH2A-affected individuals and families as significant progress is made. For more information about the LUNA study or to inquire about participation, contact Sepul Bio at contact@sepulbio.com.
The Usher Syndrome Coalition will also share ongoing updates from Sepul Bio with those in our USH Trust. Be sure to sign up so that you are notified of the latest developments. Please take a moment to confirm or update your preferred email address, your city and country, your birthdate, your USH type and subtype, and whether you have genetic confirmation of the diagnosis.
If you do not yet have genetic confirmation of your diagnosis, and you’re interested in participating in this or future clinical trials, now is the time to pursue this testing. Check our page on genetic testing, or check with your trusted healthcare provider. If you have questions, contact the Usher Syndrome Coalition at info@usher-syndrome.org.
