Our programs
Parkinson’s disease
Parkinson’s disease affects an estimated 10 million people worldwide, a number projected to double by 2040. The LRRK2 gene plays a central role in regulating neuronal health, and mutations in LRRK2 are a frequent cause of inherited Parkinson’s disease. In addition, abnormal LRRK2 kinase activity is observed in patients with sporadic Parkinson’s disease. Excessive LRRK2 activity disrupts normal brain cell function, leading to the progressive movement and cognitive symptoms characteristic of the disease.
Inverna has identified pseudo-exon activator oligonucleotides to potently and selectively target LRRK2 RNA, thereby downregulating LRRK2 activity with the aim to halt Parkinson’s disease progression.
Huntington’s disease
HD is a dominantly inherited neurodegenerative disease, currently lacking disease-modifying treatment. It is caused by an expanded CAG trinucleotide repeat in the HTT gene, leading to expression of a mutant HTT protein and subsequent neuronal toxicity. Emerging research has demonstrated that CAG repeat expansion significantly accelerates HD onset and progression and may be required for pathogenesis of HD and other repeat expansion diseases.
Inverna’s precise oligonucleotide-mediated pseudo-exon activation approach enables a unique opportunity for targeting HD and other repeat expansion diseases at their root cause.