Repeat Associated Diseases

Repeat Associated DiseasesTranslation malfunctions

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.

At Anima, we believe that the malfunction of the translation machinery is the common intervention point to find therapeutics for those devastating and yet untreatable diseases. Our unique approach allows us to visualize the different types of translation malfunctions and enables 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
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
Myotonic dystrophy type 1 coding DMPK
Myotonic dystrophy type 2 coding CNBP
Spinocerebellar Ataxia, type 10 coding Ataxin10