Sepul Bio by Théa
Sepul Bio, an innovative business unit of Laboratories Théa, is at the forefront of advancing transformative mRNA therapies for inherited retinal diseases. Sepul Bio is carrying forward the groundbreaking work testing 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.
Interested in learning more about the LUNA trial?
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.
Sepul Bio Related Science News
Learn about the Usher Syndrome Coalition’s partnership with Sepul Bio to support enrollment in the LUNA clinical trial, a Phase 2b study testing ultevursen, a potential RNA therapy for USH2A-mediated retinitis pigmentosa.
Sepul Bio launches the LUNA Phase 2b clinical study to test ultevursen, a potential mRNA therapy aimed to help people with Usher syndrome type 2a maintain their vision.
