December 12, 2024

First participant dosed in Phase 2b clinical trial of ultevursen for USH2A-associated RP

The image shows a scientist using a microscope in a laboratory setting, with a text overlay that reads "RESEARCH UPDATE" in bold, yellow letters. The background is slightly blurred, highlighting the focused work of the scientist.

We are thrilled to share that Sepul Bio, an innovative business unit of Laboratories Théa, will carry forward the groundbreaking work started by ProQR Therapeutics. On December 12, 2024, Sepul Bio announced the first clinical participant has been dosed in the LUNA clinical study. LUNA is a Phase 2b clinical study for ultevursen in individuals with Usher syndrome or non-syndromic RP due to variants in exon 13 of the Usher syndrome type 2a gene.

LUNA, also called SB-421a-006, is a two-year study testing ultevursen. Ultevursen is 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 specific 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. 

How does ultevursen work?

Ultevursen is an RNA-based therapy that targets messenger RNA (mRNA). As covered in our Science Education series, DNA is transcribed into RNA, which is then translated into proteins. mRNA carries the genetic instructions to make specific proteins. Stopping the translation process can prevent a protein from being made. This is what happens with variants in exon 13 of the USH2A gene.

Antisense oligonucleotides (AONs or ASOs) are short, custom pieces of DNA or RNA that bind to mRNA to block translation. Sepul Bio’s ultevursen uses these custom RNA fragments to bind specifically to exon 13 of the USH2A gene. This prevents the mutated exon from being translated (a process called exon skipping), while the rest of the mRNA is processed normally. The result is a functional USH2A protein, even with the skipped mutation.

Sepul Bio

As a dedicated business unit of Théa, Sepul Bio is at the forefront of advancing transformative mRNA therapies for inherited retinal diseases, with a particular emphasis on the further development of two cutting-edge ophthalmic products—ultevursen and sepofarsen. 

The Sepul Bio team was established in December 2023 and has worked hard to restart the ultevursen program. The clinical team looks forward to quickly enrolling the study, to advance ultevursen’s development and bring this potential novel therapy to the USH2A patient community as quickly as possible.

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. Interested individuals can send inquiries to contact@sepulbio.com. 

This is also a good time to make sure you are a part of our USH Trust to be kept informed on this and other clinical trials. The USH Trust is our most powerful tool for supporting research. Make sure that you have updated your information in our USH Trust. If you suspect your information is outdated, take a moment to update your preferred email address, your birthdate, your type and subtype, and whether you have genetic confirmation of this diagnosis.

Disclaimer: The information contained on the Usher Syndrome Coalition website and other online properties is provided for your information only. The Coalition is neither a medical service nor a scientific organization. It provides information concerning the various types of Usher syndrome that includes information developed or derived from unrelated third parties over which the Coalition has no involvement or control. The Usher Syndrome Coalition does not independently, nor in conjunction with others, attest to, deny, or comment upon the merits of the accuracy of the information provided by unrelated third parties. No reliance on medical or scientific information contained on this website should be taken by any person. You should always consult with and be guided by your Physician’s advice when considering treatment based on research results.