QurAlis Corporation announces programme which targets UNC13A mis-splicing in neurodegenerative diseases

pharmafile | March 20, 2023 | News story | Research and Development  

US-based clinical-stage biotechnology QurAlis Corporation has announced the launch of its newest programme to treat neurodegenerative diseases like ALS and frontotemporal dementia (FTO).


UNC13A is an essential regulator of neurotransmitter release at synapses. In neurodegenerative disorders, TDP-43 accumulates in the cytoplasm, losing its ability to control RNA metabolism in the nucleus. Because of this, certain pre-mRNA transcripts are mis-spliced which results in an expression of a cryptic exon-containing transcript that interferes with appropriate protein generation.


58% of ALS patients and up to half of FTD patients carry a single nuclear polymorphism in the UNC13A gene.


QurAlis’ FlexASO Splice Modulator Platform uses antisense oligonucleotides (ASOs) which correct this mis-splicing, restoring UNC13A protein production and reducing cryptic exons ‒ segments of a DNA or RNA molecule which contain information coding for a protein or peptide sequence ‒ that may contribute to disease progression.


Kasper Roet, PhD, CEO of QurAlis, stated: “We are excited to bring our UNC13A programme out of stealth. UNC13A is the third QurAlis programme focused on a genetic target for the development of much-needed precision medicines for the sporadic ALS and FTD populations. This critical genetic alteration of UNC13A leading to mis-splicing is believed to occur in 58% of all ALS patients and up to half of all FTD cases. We look forward to advancing our UNC13A program along with our two lead clinical programmes in ALS and robust pipeline so that we can make a real difference in patients’ lives.”


James Spargo

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