🎉 Exciting news from Inverna Therapeutics! 🚀 We’re honored to announce that Inverna has been awarded a grant from the Michael J. Fox Foundation for Parkinson’s Research (MJFF) as part of the LRRK2 Inv...

The dysferlinopathies are a spectrum of autosomal recessive muscle diseases caused by mutations in the dysferlin gene (DYSF) gene. Clinical manifestations vary from asymptomatic hyperCKemia to severe...

Inverna Therapeutics is delighted to announce the formation of our new Scientific Advisory Board (SAB)🚀 We welcome world-renowned leaders in RNA therapeutics and neurodegenerative research to advance...

Poul Sørensen, CEO, is presenting Inverna’s platform for RNA splicing modulation at BIO-Europe Spring 2025 in Milan. #BioEuropeSpring #RNASplicingModulation…

Wada and colleagues identified a deep-intronic PCCA mutation causing near-complete 84-bp pseudoexon inclusion in propionic acidemia. A splice-switching ASO restored splicing and increased propionyl-CoA carboxylase activity to approximately 300% of wild-type in index-case fibroblasts, with modest yet potentially clinically meaningful increases in additional patient fibroblasts with residual propionyl-CoA carboxylase activity.

CRISPR/Cas12a-mediated removal of a pathogenic PCCA pseudoexon or its splice enhancer effectively restores normal splicing and increases PCC enzyme activity, providing proof of concept of a promising therapeutic strategy for propionic acidemia caused by pseudoexon activation or by variants with residual activity.

Duchenne muscular dystrophy (DMD) is caused by pathogenic variants of the DMD gene. Pseudoexons resulting from deep intronic mutations account for 1-7% of all DMD patients. Beljan and colleagues induced pseudoexon skipping in patient-derived cells by employing a modified U7 small nuclear RNA (U7snRNA), restoring WT mRNA and full-length dystrophin.

Collagen VI-related dystrophies (COL6-RDs) manifest with a spectrum of clinical phenotypes, ranging