Children diagnosed with TUBB4A leukodystrophy (Hypomyelination with Atrophy of Basal Ganglia and Cerebellum) experience a progressive and severe deterioration of recently acquired motor skills. Currently, there is no available cure for this condition. Evotec, in collaboration with SynaptixBio, is developing antisense oligonucleotides (ASOs) targeting TUBB4A mRNA to suppress gene expression, irrespective of specific mutations.
The research presented here represents a crucial segment of Evotec’s oligonucleotide-based drug discovery initiative. This study employs a combination of in vitro, in vivo, and bioinformatic methodologies to minimize the synthesis and testing of acutely toxic molecules in murine models.