Today we announced a strategic collaboration and global exclusive license agreement with nonprofit organization @n_lorem Foundation to develop an antisense oligonucleotide medicine for people living with ReNU syndrome, a serious and rare neurodevelopmental condition that is caused by variants in the RNU4-2 gene and currently has no approved medicines.

ReNU syndrome was identified as a distinct condition in 2024 by an international team of geneticists, following the work of families, advocates and other researchers to raise awareness and accelerate understanding of the genetic variant.

As we continue to build out our pipeline, we are excited to partner with the antisense experts at n-Lorem to leverage our leadership in genetic medicines to develop this potential first-in-disease treatment for ReNU syndrome.

Read more in our press release: http://ms.spr.ly/6012vCgaa