Translation Malfunctions in Repeat Diseases

Indication Target
Discovery
Optimization
IND enabling
Neuroscience
Repeat Diseases Undisclosed  
Area Indication mRNA modulation  
Discovery
Optimization
IND enabling
Neuroscience Repeat Diseases Undisclosed  

Repeat associated diseases are a group of genetic diseases, also known as Trinucleotide expansion diseases. Some of the most challenging diseases belong to this group such as Huntington’s disease, Amyotrophic Lateral Sclerosis (ALS), Fragile X tremor ataxia and Myotonic dystrophy type 1/2.

Recent scientific discoveries point to malfunctions of the translation machinery as a common, underlying cause of many repeat associated diseases. As such, translation control is a novel intervention point and a new strategy against these devastating and yet untreatable diseases. Our unique approach enables us to visualize translation malfunctions and to discover molecules that selectively correct them, resulting in novel treatments.

We are in the process of running discovery programs around several diseases in this area.

Disease Repeat Target
Fragile X tremor ataxia non-coding FMR1
ALS non-coding C9orf72
Myotonic dystrophy type 1 non-coding DMPK
Myotonic dystrophy type 2 non-coding CNBP
Friedreich ataxia non-coding X25
Huntington disease coding HTT
Spinobulbar muscular atrophy coding AR
Spinocerebellar Ataxias (SCA types 1, 2, 3, 6, 7, 17) coding ATXN-1, 2, 3, 7, CACNA1A, TBP
Dentatorubro-Pallidoluysian Atrophy (DRPLA) coding ATN1
Spinocerebellar Ataxia, type 10 coding Ataxin10